RU2701850C2 - Конструирование систем, способы и оптимизированные направляющие композиции для манипуляции с последовательностями - Google Patents
Конструирование систем, способы и оптимизированные направляющие композиции для манипуляции с последовательностями Download PDFInfo
- Publication number
- RU2701850C2 RU2701850C2 RU2015128098A RU2015128098A RU2701850C2 RU 2701850 C2 RU2701850 C2 RU 2701850C2 RU 2015128098 A RU2015128098 A RU 2015128098A RU 2015128098 A RU2015128098 A RU 2015128098A RU 2701850 C2 RU2701850 C2 RU 2701850C2
- Authority
- RU
- Russia
- Prior art keywords
- sequence
- crispr
- tracr
- cas system
- guide
- Prior art date
Links
- 238000000034 method Methods 0.000 title description 79
- 239000000203 mixture Substances 0.000 title description 12
- 108091033409 CRISPR Proteins 0.000 claims abstract description 135
- 239000013598 vector Substances 0.000 claims abstract description 114
- 230000014509 gene expression Effects 0.000 claims abstract description 109
- 125000003729 nucleotide group Chemical group 0.000 claims abstract description 104
- 239000002773 nucleotide Substances 0.000 claims abstract description 79
- 210000003527 eukaryotic cell Anatomy 0.000 claims abstract description 71
- 108091092236 Chimeric RNA Proteins 0.000 claims abstract description 70
- 230000001105 regulatory effect Effects 0.000 claims abstract description 58
- 230000030648 nucleus localization Effects 0.000 claims abstract description 24
- 102000040430 polynucleotide Human genes 0.000 claims description 101
- 108091033319 polynucleotide Proteins 0.000 claims description 101
- 239000002157 polynucleotide Substances 0.000 claims description 101
- 230000004048 modification Effects 0.000 claims description 75
- 238000012986 modification Methods 0.000 claims description 75
- 108091028113 Trans-activating crRNA Proteins 0.000 claims description 60
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 58
- 238000003776 cleavage reaction Methods 0.000 claims description 57
- 230000007017 scission Effects 0.000 claims description 57
- 241000193996 Streptococcus pyogenes Species 0.000 claims description 30
- 150000001413 amino acids Chemical class 0.000 claims description 26
- 238000005457 optimization Methods 0.000 claims description 19
- 101710163270 Nuclease Proteins 0.000 claims description 13
- 230000004927 fusion Effects 0.000 claims description 12
- 238000009396 hybridization Methods 0.000 claims description 12
- 230000002829 reductive effect Effects 0.000 claims description 8
- 238000004132 cross linking Methods 0.000 claims description 7
- MWBWWFOAEOYUST-UHFFFAOYSA-N 2-aminopurine Chemical compound NC1=NC=C2N=CNC2=N1 MWBWWFOAEOYUST-UHFFFAOYSA-N 0.000 claims description 5
- AGFIRQJZCNVMCW-UAKXSSHOSA-N 5-bromouridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C(Br)=C1 AGFIRQJZCNVMCW-UAKXSSHOSA-N 0.000 claims description 5
- OGHAROSJZRTIOK-KQYNXXCUSA-O 7-methylguanosine Chemical compound C1=2N=C(N)NC(=O)C=2[N+](C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OGHAROSJZRTIOK-KQYNXXCUSA-O 0.000 claims description 5
- 101710172824 CRISPR-associated endonuclease Cas9 Proteins 0.000 claims description 5
- HMFHBZSHGGEWLO-SOOFDHNKSA-N D-ribofuranose Chemical compound OC[C@H]1OC(O)[C@H](O)[C@@H]1O HMFHBZSHGGEWLO-SOOFDHNKSA-N 0.000 claims description 5
- UGQMRVRMYYASKQ-KQYNXXCUSA-N Inosine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(O)=C2N=C1 UGQMRVRMYYASKQ-KQYNXXCUSA-N 0.000 claims description 5
- 229930010555 Inosine Natural products 0.000 claims description 5
- 229910019142 PO4 Inorganic materials 0.000 claims description 5
- 229930185560 Pseudouridine Natural products 0.000 claims description 5
- PTJWIQPHWPFNBW-UHFFFAOYSA-N Pseudouridine C Natural products OC1C(O)C(CO)OC1C1=CNC(=O)NC1=O PTJWIQPHWPFNBW-UHFFFAOYSA-N 0.000 claims description 5
- PYMYPHUHKUWMLA-LMVFSUKVSA-N Ribose Natural products OC[C@@H](O)[C@@H](O)[C@@H](O)C=O PYMYPHUHKUWMLA-LMVFSUKVSA-N 0.000 claims description 5
- HMFHBZSHGGEWLO-UHFFFAOYSA-N alpha-D-Furanose-Ribose Natural products OCC1OC(O)C(O)C1O HMFHBZSHGGEWLO-UHFFFAOYSA-N 0.000 claims description 5
- WGDUUQDYDIIBKT-UHFFFAOYSA-N beta-Pseudouridine Natural products OC1OC(CN2C=CC(=O)NC2=O)C(O)C1O WGDUUQDYDIIBKT-UHFFFAOYSA-N 0.000 claims description 5
- 229960003786 inosine Drugs 0.000 claims description 5
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims description 5
- 239000010452 phosphate Substances 0.000 claims description 5
- PTJWIQPHWPFNBW-GBNDHIKLSA-N pseudouridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1C1=CNC(=O)NC1=O PTJWIQPHWPFNBW-GBNDHIKLSA-N 0.000 claims description 5
- 241000589876 Campylobacter Species 0.000 claims description 4
- 241000588653 Neisseria Species 0.000 claims description 3
- 230000000087 stabilizing effect Effects 0.000 claims description 3
- 241000589941 Azospirillum Species 0.000 claims description 2
- 241000032681 Gluconacetobacter Species 0.000 claims description 2
- 241000135938 Nitratifractor Species 0.000 claims description 2
- 241001386753 Parvibaculum Species 0.000 claims description 2
- 241000605947 Roseburia Species 0.000 claims description 2
- 241000191940 Staphylococcus Species 0.000 claims description 2
- 241000194017 Streptococcus Species 0.000 claims description 2
- 101000910035 Streptococcus pyogenes serotype M1 CRISPR-associated endonuclease Cas9/Csn1 Proteins 0.000 claims 1
- 230000000694 effects Effects 0.000 abstract description 60
- 239000000126 substance Substances 0.000 abstract 1
- 102000004190 Enzymes Human genes 0.000 description 163
- 108090000790 Enzymes Proteins 0.000 description 163
- 229940088598 enzyme Drugs 0.000 description 163
- 108090000623 proteins and genes Proteins 0.000 description 148
- 210000004027 cell Anatomy 0.000 description 146
- 102000004169 proteins and genes Human genes 0.000 description 71
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 68
- 235000018102 proteins Nutrition 0.000 description 68
- 150000007523 nucleic acids Chemical class 0.000 description 54
- 201000010099 disease Diseases 0.000 description 50
- 102000039446 nucleic acids Human genes 0.000 description 44
- 108020004707 nucleic acids Proteins 0.000 description 44
- 108020004414 DNA Proteins 0.000 description 41
- 230000035772 mutation Effects 0.000 description 39
- 239000012634 fragment Substances 0.000 description 29
- 241000196324 Embryophyta Species 0.000 description 28
- 230000000875 corresponding effect Effects 0.000 description 27
- 125000006850 spacer group Chemical group 0.000 description 27
- 241000282414 Homo sapiens Species 0.000 description 26
- 230000015572 biosynthetic process Effects 0.000 description 25
- 230000000295 complement effect Effects 0.000 description 25
- 238000004458 analytical method Methods 0.000 description 24
- 239000013604 expression vector Substances 0.000 description 24
- 229920002401 polyacrylamide Polymers 0.000 description 23
- 102100039087 Peptidyl-alpha-hydroxyglycine alpha-amidating lyase Human genes 0.000 description 22
- 241000194020 Streptococcus thermophilus Species 0.000 description 21
- 230000027455 binding Effects 0.000 description 21
- 238000003780 insertion Methods 0.000 description 21
- 230000037431 insertion Effects 0.000 description 21
- 235000001014 amino acid Nutrition 0.000 description 20
- 102100023823 Homeobox protein EMX1 Human genes 0.000 description 18
- 101001048956 Homo sapiens Homeobox protein EMX1 Proteins 0.000 description 18
- 229940024606 amino acid Drugs 0.000 description 18
- 208000035475 disorder Diseases 0.000 description 18
- 238000002744 homologous recombination Methods 0.000 description 18
- 230000006801 homologous recombination Effects 0.000 description 18
- 210000004940 nucleus Anatomy 0.000 description 18
- 238000004422 calculation algorithm Methods 0.000 description 17
- 238000005516 engineering process Methods 0.000 description 17
- 230000035897 transcription Effects 0.000 description 17
- 238000013518 transcription Methods 0.000 description 17
- 230000001404 mediated effect Effects 0.000 description 16
- 210000001519 tissue Anatomy 0.000 description 16
- 230000003612 virological effect Effects 0.000 description 16
- 108091026890 Coding region Proteins 0.000 description 15
- 108020004705 Codon Proteins 0.000 description 15
- 241000699666 Mus <mouse, genus> Species 0.000 description 15
- 241000320123 Streptococcus pyogenes M1 GAS Species 0.000 description 15
- 210000004962 mammalian cell Anatomy 0.000 description 15
- 239000013612 plasmid Substances 0.000 description 15
- 208000011580 syndromic disease Diseases 0.000 description 15
- 238000012217 deletion Methods 0.000 description 14
- 230000037430 deletion Effects 0.000 description 14
- 230000009870 specific binding Effects 0.000 description 14
- 241001465754 Metazoa Species 0.000 description 13
- 241000700605 Viruses Species 0.000 description 13
- 238000011161 development Methods 0.000 description 13
- 230000018109 developmental process Effects 0.000 description 13
- 239000011159 matrix material Substances 0.000 description 13
- 230000002068 genetic effect Effects 0.000 description 12
- 230000009261 transgenic effect Effects 0.000 description 12
- 241000702421 Dependoparvovirus Species 0.000 description 11
- 239000003814 drug Substances 0.000 description 11
- 238000000338 in vitro Methods 0.000 description 11
- 108090000765 processed proteins & peptides Proteins 0.000 description 11
- 102100030856 Myoglobin Human genes 0.000 description 10
- 108091028043 Nucleic acid sequence Proteins 0.000 description 10
- 238000009825 accumulation Methods 0.000 description 10
- 108010025678 empty spiracles homeobox proteins Proteins 0.000 description 10
- 102000004196 processed proteins & peptides Human genes 0.000 description 10
- 230000008685 targeting Effects 0.000 description 10
- 230000007018 DNA scission Effects 0.000 description 9
- 101000706121 Homo sapiens Parvalbumin alpha Proteins 0.000 description 9
- 241000124008 Mammalia Species 0.000 description 9
- 238000000636 Northern blotting Methods 0.000 description 9
- 239000000872 buffer Substances 0.000 description 9
- -1 for example Chemical group 0.000 description 9
- 230000001965 increasing effect Effects 0.000 description 9
- 241000701161 unidentified adenovirus Species 0.000 description 9
- 239000013603 viral vector Substances 0.000 description 9
- 238000003556 assay Methods 0.000 description 8
- 230000011559 double-strand break repair via nonhomologous end joining Effects 0.000 description 8
- 102000037865 fusion proteins Human genes 0.000 description 8
- 108020001507 fusion proteins Proteins 0.000 description 8
- 238000001415 gene therapy Methods 0.000 description 8
- 238000001727 in vivo Methods 0.000 description 8
- 238000004519 manufacturing process Methods 0.000 description 8
- 238000004806 packaging method and process Methods 0.000 description 8
- 230000037361 pathway Effects 0.000 description 8
- 230000008569 process Effects 0.000 description 8
- 238000001890 transfection Methods 0.000 description 8
- 108091093088 Amplicon Proteins 0.000 description 7
- 241000588724 Escherichia coli Species 0.000 description 7
- 108010008281 Recombinant Fusion Proteins Proteins 0.000 description 7
- 102000007056 Recombinant Fusion Proteins Human genes 0.000 description 7
- 230000001580 bacterial effect Effects 0.000 description 7
- 229940079593 drug Drugs 0.000 description 7
- 230000006870 function Effects 0.000 description 7
- 108020004999 messenger RNA Proteins 0.000 description 7
- 244000052769 pathogen Species 0.000 description 7
- 238000012545 processing Methods 0.000 description 7
- 210000001236 prokaryotic cell Anatomy 0.000 description 7
- 230000006798 recombination Effects 0.000 description 7
- 238000005215 recombination Methods 0.000 description 7
- 238000012163 sequencing technique Methods 0.000 description 7
- 241000894007 species Species 0.000 description 7
- 208000024891 symptom Diseases 0.000 description 7
- 241000894006 Bacteria Species 0.000 description 6
- 102000053602 DNA Human genes 0.000 description 6
- 241000238631 Hexapoda Species 0.000 description 6
- 101000635854 Homo sapiens Myoglobin Proteins 0.000 description 6
- 201000008266 amyotrophic lateral sclerosis type 2 Diseases 0.000 description 6
- 210000004899 c-terminal region Anatomy 0.000 description 6
- 239000003623 enhancer Substances 0.000 description 6
- 239000000499 gel Substances 0.000 description 6
- 208000032799 juvenile amyotrophic lateral sclerosis type 2 Diseases 0.000 description 6
- 230000008439 repair process Effects 0.000 description 6
- 230000010076 replication Effects 0.000 description 6
- 238000012360 testing method Methods 0.000 description 6
- 241001430294 unidentified retrovirus Species 0.000 description 6
- 102100033312 Alpha-2-macroglobulin Human genes 0.000 description 5
- 102220605874 Cytosolic arginine sensor for mTORC1 subunit 2_D10A_mutation Human genes 0.000 description 5
- 108010008532 Deoxyribonuclease I Proteins 0.000 description 5
- 102000007260 Deoxyribonuclease I Human genes 0.000 description 5
- 108091034117 Oligonucleotide Proteins 0.000 description 5
- 108010015078 Pregnancy-Associated alpha 2-Macroglobulins Proteins 0.000 description 5
- 102100030547 Serotonin N-acetyltransferase Human genes 0.000 description 5
- 108010021188 Superoxide Dismutase-1 Proteins 0.000 description 5
- 102100038836 Superoxide dismutase [Cu-Zn] Human genes 0.000 description 5
- 108010073925 Vascular Endothelial Growth Factor B Proteins 0.000 description 5
- 102100038217 Vascular endothelial growth factor B Human genes 0.000 description 5
- 208000021841 acute erythroid leukemia Diseases 0.000 description 5
- 239000003795 chemical substances by application Substances 0.000 description 5
- 230000000536 complexating effect Effects 0.000 description 5
- 229910052739 hydrogen Inorganic materials 0.000 description 5
- 239000001257 hydrogen Substances 0.000 description 5
- 230000005847 immunogenicity Effects 0.000 description 5
- 230000036961 partial effect Effects 0.000 description 5
- 229920001184 polypeptide Polymers 0.000 description 5
- 238000011160 research Methods 0.000 description 5
- 230000001177 retroviral effect Effects 0.000 description 5
- 230000003007 single stranded DNA break Effects 0.000 description 5
- 239000004055 small Interfering RNA Substances 0.000 description 5
- 230000006641 stabilisation Effects 0.000 description 5
- 238000011105 stabilization Methods 0.000 description 5
- 230000001225 therapeutic effect Effects 0.000 description 5
- 238000012546 transfer Methods 0.000 description 5
- 102100035923 4-aminobutyrate aminotransferase, mitochondrial Human genes 0.000 description 4
- 102100025064 Cellular tumor antigen p53 Human genes 0.000 description 4
- 241000282693 Cercopithecidae Species 0.000 description 4
- 241000195493 Cryptophyta Species 0.000 description 4
- 108010070675 Glutathione transferase Proteins 0.000 description 4
- 108010043121 Green Fluorescent Proteins Proteins 0.000 description 4
- 102000004144 Green Fluorescent Proteins Human genes 0.000 description 4
- 108020005004 Guide RNA Proteins 0.000 description 4
- 102100029100 Hematopoietic prostaglandin D synthase Human genes 0.000 description 4
- 101000891092 Homo sapiens TAR DNA-binding protein 43 Proteins 0.000 description 4
- 208000026350 Inborn Genetic disease Diseases 0.000 description 4
- 108700020796 Oncogene Proteins 0.000 description 4
- 102100033616 Phospholipid-transporting ATPase ABCA1 Human genes 0.000 description 4
- 241000700159 Rattus Species 0.000 description 4
- 108091081062 Repeated sequence (DNA) Proteins 0.000 description 4
- 102100040347 TAR DNA-binding protein 43 Human genes 0.000 description 4
- 108010073929 Vascular Endothelial Growth Factor A Proteins 0.000 description 4
- 102000009520 Vascular Endothelial Growth Factor C Human genes 0.000 description 4
- 108010073923 Vascular Endothelial Growth Factor C Proteins 0.000 description 4
- 102100039037 Vascular endothelial growth factor A Human genes 0.000 description 4
- 230000009471 action Effects 0.000 description 4
- 238000013459 approach Methods 0.000 description 4
- 230000009287 biochemical signal transduction Effects 0.000 description 4
- 239000003153 chemical reaction reagent Substances 0.000 description 4
- 230000001276 controlling effect Effects 0.000 description 4
- 230000007547 defect Effects 0.000 description 4
- 230000001419 dependent effect Effects 0.000 description 4
- 238000001514 detection method Methods 0.000 description 4
- 206010015037 epilepsy Diseases 0.000 description 4
- 238000002474 experimental method Methods 0.000 description 4
- 208000016361 genetic disease Diseases 0.000 description 4
- 238000010362 genome editing Methods 0.000 description 4
- 230000010354 integration Effects 0.000 description 4
- 238000001638 lipofection Methods 0.000 description 4
- 229920000642 polymer Polymers 0.000 description 4
- 238000004321 preservation Methods 0.000 description 4
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 description 4
- 238000003259 recombinant expression Methods 0.000 description 4
- 239000000523 sample Substances 0.000 description 4
- 238000006467 substitution reaction Methods 0.000 description 4
- 239000013607 AAV vector Substances 0.000 description 3
- 102100024378 AF4/FMR2 family member 2 Human genes 0.000 description 3
- 102100024401 Alpha-1D adrenergic receptor Human genes 0.000 description 3
- 208000024827 Alzheimer disease Diseases 0.000 description 3
- 108010074515 Arylalkylamine N-Acetyltransferase Proteins 0.000 description 3
- 102000007370 Ataxin2 Human genes 0.000 description 3
- 108010032951 Ataxin2 Proteins 0.000 description 3
- 108700010070 Codon Usage Proteins 0.000 description 3
- 101150074775 Csf1 gene Proteins 0.000 description 3
- 101710177611 DNA polymerase II large subunit Proteins 0.000 description 3
- 101710184669 DNA polymerase II small subunit Proteins 0.000 description 3
- 241000252212 Danio rerio Species 0.000 description 3
- LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 description 3
- 101000756842 Homo sapiens Alpha-2A adrenergic receptor Proteins 0.000 description 3
- 101000720032 Homo sapiens Alpha-2C adrenergic receptor Proteins 0.000 description 3
- 101000721661 Homo sapiens Cellular tumor antigen p53 Proteins 0.000 description 3
- 101000755630 Homo sapiens Peripheral-type benzodiazepine receptor-associated protein 1 Proteins 0.000 description 3
- 101001135385 Homo sapiens Prostacyclin synthase Proteins 0.000 description 3
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 description 3
- 102000004388 Interleukin-4 Human genes 0.000 description 3
- 108090000978 Interleukin-4 Proteins 0.000 description 3
- 241000713666 Lentivirus Species 0.000 description 3
- 101710175625 Maltose/maltodextrin-binding periplasmic protein Proteins 0.000 description 3
- 208000036626 Mental retardation Diseases 0.000 description 3
- OKKJLVBELUTLKV-UHFFFAOYSA-N Methanol Chemical compound OC OKKJLVBELUTLKV-UHFFFAOYSA-N 0.000 description 3
- 206010028980 Neoplasm Diseases 0.000 description 3
- 102100022397 Nitric oxide synthase, brain Human genes 0.000 description 3
- 102000002488 Nucleoplasmin Human genes 0.000 description 3
- 241000283973 Oryctolagus cuniculus Species 0.000 description 3
- 102100022033 Presenilin-1 Human genes 0.000 description 3
- 102100022036 Presenilin-2 Human genes 0.000 description 3
- 102100033075 Prostacyclin synthase Human genes 0.000 description 3
- 206010039491 Sarcoma Diseases 0.000 description 3
- 108020004459 Small interfering RNA Proteins 0.000 description 3
- 108091027544 Subgenomic mRNA Proteins 0.000 description 3
- 102100037346 Substance-P receptor Human genes 0.000 description 3
- 108700009124 Transcription Initiation Site Proteins 0.000 description 3
- 102100037160 Ubiquitin-like modifier-activating enzyme 1 Human genes 0.000 description 3
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 description 3
- 108020004102 alpha-1 Adrenergic Receptor Proteins 0.000 description 3
- 230000000692 anti-sense effect Effects 0.000 description 3
- 230000008901 benefit Effects 0.000 description 3
- 108091005948 blue fluorescent proteins Proteins 0.000 description 3
- 125000003178 carboxy group Chemical group [H]OC(*)=O 0.000 description 3
- 230000003197 catalytic effect Effects 0.000 description 3
- 238000004113 cell culture Methods 0.000 description 3
- 150000001875 compounds Chemical class 0.000 description 3
- 238000013461 design Methods 0.000 description 3
- 210000002950 fibroblast Anatomy 0.000 description 3
- 239000005090 green fluorescent protein Substances 0.000 description 3
- 230000036541 health Effects 0.000 description 3
- 230000001976 improved effect Effects 0.000 description 3
- 230000006872 improvement Effects 0.000 description 3
- 230000001939 inductive effect Effects 0.000 description 3
- 239000003446 ligand Substances 0.000 description 3
- 150000002632 lipids Chemical class 0.000 description 3
- 239000002502 liposome Substances 0.000 description 3
- 239000003550 marker Substances 0.000 description 3
- 230000007246 mechanism Effects 0.000 description 3
- 238000010369 molecular cloning Methods 0.000 description 3
- 108060005597 nucleoplasmin Proteins 0.000 description 3
- 230000001717 pathogenic effect Effects 0.000 description 3
- 238000002360 preparation method Methods 0.000 description 3
- 102000005962 receptors Human genes 0.000 description 3
- 108020003175 receptors Proteins 0.000 description 3
- 230000002441 reversible effect Effects 0.000 description 3
- 238000003786 synthesis reaction Methods 0.000 description 3
- 229940124597 therapeutic agent Drugs 0.000 description 3
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 description 3
- 230000005030 transcription termination Effects 0.000 description 3
- 230000014616 translation Effects 0.000 description 3
- 238000013519 translation Methods 0.000 description 3
- 241000701447 unidentified baculovirus Species 0.000 description 3
- 238000012800 visualization Methods 0.000 description 3
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 2
- GZCWLCBFPRFLKL-UHFFFAOYSA-N 1-prop-2-ynoxypropan-2-ol Chemical compound CC(O)COCC#C GZCWLCBFPRFLKL-UHFFFAOYSA-N 0.000 description 2
- 101710094518 4-aminobutyrate aminotransferase Proteins 0.000 description 2
- 101150092476 ABCA1 gene Proteins 0.000 description 2
- 108700005241 ATP Binding Cassette Transporter 1 Proteins 0.000 description 2
- 102100036618 ATP-binding cassette sub-family A member 13 Human genes 0.000 description 2
- 102100033618 ATP-binding cassette sub-family A member 2 Human genes 0.000 description 2
- 102100033092 ATP-binding cassette sub-family G member 8 Human genes 0.000 description 2
- 108010051457 Acid Phosphatase Proteins 0.000 description 2
- 102000013563 Acid Phosphatase Human genes 0.000 description 2
- 102100036732 Actin, aortic smooth muscle Human genes 0.000 description 2
- 241000589158 Agrobacterium Species 0.000 description 2
- 108700028369 Alleles Proteins 0.000 description 2
- 102100022815 Alpha-2A adrenergic receptor Human genes 0.000 description 2
- 102100025983 Alpha-2C adrenergic receptor Human genes 0.000 description 2
- 208000026833 Alzheimer disease 4 Diseases 0.000 description 2
- 102000002659 Amyloid Precursor Protein Secretases Human genes 0.000 description 2
- 108010043324 Amyloid Precursor Protein Secretases Proteins 0.000 description 2
- 102100022704 Amyloid-beta precursor protein Human genes 0.000 description 2
- 101710151993 Amyloid-beta precursor protein Proteins 0.000 description 2
- 102100032187 Androgen receptor Human genes 0.000 description 2
- 108010048154 Angiopoietin-1 Proteins 0.000 description 2
- 102000009088 Angiopoietin-1 Human genes 0.000 description 2
- 102100029470 Apolipoprotein E Human genes 0.000 description 2
- 241000283690 Bos taurus Species 0.000 description 2
- 208000014644 Brain disease Diseases 0.000 description 2
- 101710155857 C-C motif chemokine 2 Proteins 0.000 description 2
- 101000909256 Caldicellulosiruptor bescii (strain ATCC BAA-1888 / DSM 6725 / Z-1320) DNA polymerase I Proteins 0.000 description 2
- 241000589875 Campylobacter jejuni Species 0.000 description 2
- 108090000712 Cathepsin B Proteins 0.000 description 2
- 102000004225 Cathepsin B Human genes 0.000 description 2
- 108090000625 Cathepsin K Proteins 0.000 description 2
- 102000004171 Cathepsin K Human genes 0.000 description 2
- 102000000018 Chemokine CCL2 Human genes 0.000 description 2
- 102000019034 Chemokines Human genes 0.000 description 2
- 108010012236 Chemokines Proteins 0.000 description 2
- 108010035563 Chloramphenicol O-acetyltransferase Proteins 0.000 description 2
- 206010010904 Convulsion Diseases 0.000 description 2
- 241000701022 Cytomegalovirus Species 0.000 description 2
- 238000007400 DNA extraction Methods 0.000 description 2
- 241000450599 DNA viruses Species 0.000 description 2
- 230000004568 DNA-binding Effects 0.000 description 2
- 102100031939 Erythropoietin Human genes 0.000 description 2
- 108700024394 Exon Proteins 0.000 description 2
- 102000003869 Frataxin Human genes 0.000 description 2
- 108090000217 Frataxin Proteins 0.000 description 2
- 208000024412 Friedreich ataxia Diseases 0.000 description 2
- 241000233866 Fungi Species 0.000 description 2
- 101150106478 GPS1 gene Proteins 0.000 description 2
- 102100033713 Gamma-secretase subunit APH-1B Human genes 0.000 description 2
- 102100028260 Gamma-secretase subunit PEN-2 Human genes 0.000 description 2
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 description 2
- 108091027305 Heteroduplex Proteins 0.000 description 2
- 101001000686 Homo sapiens 4-aminobutyrate aminotransferase, mitochondrial Proteins 0.000 description 2
- 101000833172 Homo sapiens AF4/FMR2 family member 2 Proteins 0.000 description 2
- 101000929660 Homo sapiens ATP-binding cassette sub-family A member 13 Proteins 0.000 description 2
- 101000801645 Homo sapiens ATP-binding cassette sub-family A member 2 Proteins 0.000 description 2
- 101000733778 Homo sapiens Gamma-secretase subunit APH-1B Proteins 0.000 description 2
- 101000579663 Homo sapiens Gamma-secretase subunit PEN-2 Proteins 0.000 description 2
- 101000974009 Homo sapiens Nitric oxide synthase, brain Proteins 0.000 description 2
- 101000801684 Homo sapiens Phospholipid-transporting ATPase ABCA1 Proteins 0.000 description 2
- 101000801643 Homo sapiens Retinal-specific phospholipid-transporting ATPase ABCA4 Proteins 0.000 description 2
- 101000904787 Homo sapiens Serine/threonine-protein kinase ATR Proteins 0.000 description 2
- 101000652292 Homo sapiens Serotonin N-acetyltransferase Proteins 0.000 description 2
- 108010001336 Horseradish Peroxidase Proteins 0.000 description 2
- 208000023105 Huntington disease Diseases 0.000 description 2
- 108010073807 IgG Receptors Proteins 0.000 description 2
- 102000009490 IgG Receptors Human genes 0.000 description 2
- 206010061598 Immunodeficiency Diseases 0.000 description 2
- 208000029462 Immunodeficiency disease Diseases 0.000 description 2
- 206010061218 Inflammation Diseases 0.000 description 2
- 102000000589 Interleukin-1 Human genes 0.000 description 2
- 108010002352 Interleukin-1 Proteins 0.000 description 2
- XEEYBQQBJWHFJM-UHFFFAOYSA-N Iron Chemical compound [Fe] XEEYBQQBJWHFJM-UHFFFAOYSA-N 0.000 description 2
- 102100036600 Kynurenine/alpha-aminoadipate aminotransferase, mitochondrial Human genes 0.000 description 2
- QNAYBMKLOCPYGJ-REOHCLBHSA-N L-alanine Chemical compound C[C@H](N)C(O)=O QNAYBMKLOCPYGJ-REOHCLBHSA-N 0.000 description 2
- CKLJMWTZIZZHCS-REOHCLBHSA-N L-aspartic acid Chemical compound OC(=O)[C@@H](N)CC(O)=O CKLJMWTZIZZHCS-REOHCLBHSA-N 0.000 description 2
- 102100022745 Laminin subunit alpha-2 Human genes 0.000 description 2
- 101710128836 Large T antigen Proteins 0.000 description 2
- 208000009625 Lesch-Nyhan syndrome Diseases 0.000 description 2
- 235000007688 Lycopersicon esculentum Nutrition 0.000 description 2
- 101150083522 MECP2 gene Proteins 0.000 description 2
- 241001599018 Melanogaster Species 0.000 description 2
- 108700011259 MicroRNAs Proteins 0.000 description 2
- 108020005196 Mitochondrial DNA Proteins 0.000 description 2
- 101100219625 Mus musculus Casd1 gene Proteins 0.000 description 2
- 108010062374 Myoglobin Proteins 0.000 description 2
- 108010052185 Myotonin-Protein Kinase Proteins 0.000 description 2
- 102100022437 Myotonin-protein kinase Human genes 0.000 description 2
- 102100028719 NEDD8-activating enzyme E1 catalytic subunit Human genes 0.000 description 2
- 208000012902 Nervous system disease Diseases 0.000 description 2
- 108010040718 Neurokinin-1 Receptors Proteins 0.000 description 2
- 208000025966 Neurological disease Diseases 0.000 description 2
- 108020004711 Nucleic Acid Probes Proteins 0.000 description 2
- 208000018737 Parkinson disease Diseases 0.000 description 2
- 102100022369 Peripheral-type benzodiazepine receptor-associated protein 1 Human genes 0.000 description 2
- 108010036933 Presenilin-1 Proteins 0.000 description 2
- 108010036908 Presenilin-2 Proteins 0.000 description 2
- 208000024777 Prion disease Diseases 0.000 description 2
- 102100034180 Protein AATF Human genes 0.000 description 2
- 101710155502 Protein AATF Proteins 0.000 description 2
- 101000902592 Pyrococcus furiosus (strain ATCC 43587 / DSM 3638 / JCM 8422 / Vc1) DNA polymerase Proteins 0.000 description 2
- 102000003890 RNA-binding protein FUS Human genes 0.000 description 2
- 108090000292 RNA-binding protein FUS Proteins 0.000 description 2
- 108020004511 Recombinant DNA Proteins 0.000 description 2
- 108700008625 Reporter Genes Proteins 0.000 description 2
- 102100033617 Retinal-specific phospholipid-transporting ATPase ABCA4 Human genes 0.000 description 2
- 102000003661 Ribonuclease III Human genes 0.000 description 2
- 108010057163 Ribonuclease III Proteins 0.000 description 2
- 108010005173 SERPIN-B5 Proteins 0.000 description 2
- 240000004808 Saccharomyces cerevisiae Species 0.000 description 2
- 102100023921 Serine/threonine-protein kinase ATR Human genes 0.000 description 2
- 241000700584 Simplexvirus Species 0.000 description 2
- 102000039471 Small Nuclear RNA Human genes 0.000 description 2
- 108091027967 Small hairpin RNA Proteins 0.000 description 2
- CDBYLPFSWZWCQE-UHFFFAOYSA-L Sodium Carbonate Chemical compound [Na+].[Na+].[O-]C([O-])=O CDBYLPFSWZWCQE-UHFFFAOYSA-L 0.000 description 2
- UIIMBOGNXHQVGW-UHFFFAOYSA-M Sodium bicarbonate Chemical compound [Na+].OC([O-])=O UIIMBOGNXHQVGW-UHFFFAOYSA-M 0.000 description 2
- 240000003768 Solanum lycopersicum Species 0.000 description 2
- 102000005157 Somatostatin Human genes 0.000 description 2
- 108010056088 Somatostatin Proteins 0.000 description 2
- 241000205101 Sulfolobus Species 0.000 description 2
- 102100023532 Synaptic functional regulator FMR1 Human genes 0.000 description 2
- 102100036407 Thioredoxin Human genes 0.000 description 2
- 208000035317 Total hypoxanthine-guanine phosphoribosyl transferase deficiency Diseases 0.000 description 2
- 108700019146 Transgenes Proteins 0.000 description 2
- 101710191412 Ubiquitin-like modifier-activating enzyme 1 Proteins 0.000 description 2
- 102100039066 Very low-density lipoprotein receptor Human genes 0.000 description 2
- 108010091383 Xanthine dehydrogenase Proteins 0.000 description 2
- 102000005773 Xanthine dehydrogenase Human genes 0.000 description 2
- 108010093894 Xanthine oxidase Proteins 0.000 description 2
- 230000002159 abnormal effect Effects 0.000 description 2
- 239000013543 active substance Substances 0.000 description 2
- 230000006978 adaptation Effects 0.000 description 2
- 235000004279 alanine Nutrition 0.000 description 2
- 150000001298 alcohols Chemical class 0.000 description 2
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 description 2
- 108010080146 androgen receptors Proteins 0.000 description 2
- 210000004102 animal cell Anatomy 0.000 description 2
- 238000000137 annealing Methods 0.000 description 2
- 210000000709 aorta Anatomy 0.000 description 2
- 229940009098 aspartate Drugs 0.000 description 2
- 230000009286 beneficial effect Effects 0.000 description 2
- 230000002146 bilateral effect Effects 0.000 description 2
- 239000002551 biofuel Substances 0.000 description 2
- 230000008827 biological function Effects 0.000 description 2
- 210000000988 bone and bone Anatomy 0.000 description 2
- 238000004364 calculation method Methods 0.000 description 2
- 101150055766 cat gene Proteins 0.000 description 2
- 230000001413 cellular effect Effects 0.000 description 2
- 230000008859 change Effects 0.000 description 2
- 238000006243 chemical reaction Methods 0.000 description 2
- 201000006815 congenital muscular dystrophy Diseases 0.000 description 2
- 230000003247 decreasing effect Effects 0.000 description 2
- 230000002950 deficient Effects 0.000 description 2
- 241001493065 dsRNA viruses Species 0.000 description 2
- 230000002349 favourable effect Effects 0.000 description 2
- 238000001914 filtration Methods 0.000 description 2
- 108091006047 fluorescent proteins Proteins 0.000 description 2
- 238000010353 genetic engineering Methods 0.000 description 2
- 238000012268 genome sequencing Methods 0.000 description 2
- 210000002443 helper t lymphocyte Anatomy 0.000 description 2
- 125000004435 hydrogen atom Chemical class [H]* 0.000 description 2
- 230000007813 immunodeficiency Effects 0.000 description 2
- 230000004054 inflammatory process Effects 0.000 description 2
- 230000000977 initiatory effect Effects 0.000 description 2
- 230000003993 interaction Effects 0.000 description 2
- 229940028885 interleukin-4 Drugs 0.000 description 2
- 210000003292 kidney cell Anatomy 0.000 description 2
- 238000002372 labelling Methods 0.000 description 2
- 208000032839 leukemia Diseases 0.000 description 2
- 230000000670 limiting effect Effects 0.000 description 2
- 210000004185 liver Anatomy 0.000 description 2
- 208000002780 macular degeneration Diseases 0.000 description 2
- 239000000463 material Substances 0.000 description 2
- 239000002679 microRNA Substances 0.000 description 2
- 210000003205 muscle Anatomy 0.000 description 2
- 231100000350 mutagenesis Toxicity 0.000 description 2
- 239000003471 mutagenic agent Substances 0.000 description 2
- 230000009826 neoplastic cell growth Effects 0.000 description 2
- 230000004770 neurodegeneration Effects 0.000 description 2
- 210000002569 neuron Anatomy 0.000 description 2
- 108091027963 non-coding RNA Proteins 0.000 description 2
- 102000042567 non-coding RNA Human genes 0.000 description 2
- 239000002853 nucleic acid probe Substances 0.000 description 2
- 239000003921 oil Substances 0.000 description 2
- 235000019198 oils Nutrition 0.000 description 2
- 208000001749 optic atrophy Diseases 0.000 description 2
- 239000002245 particle Substances 0.000 description 2
- 230000001575 pathological effect Effects 0.000 description 2
- OXCMYAYHXIHQOA-UHFFFAOYSA-N potassium;[2-butyl-5-chloro-3-[[4-[2-(1,2,4-triaza-3-azanidacyclopenta-1,4-dien-5-yl)phenyl]phenyl]methyl]imidazol-4-yl]methanol Chemical compound [K+].CCCCC1=NC(Cl)=C(CO)N1CC1=CC=C(C=2C(=CC=CC=2)C2=N[N-]N=N2)C=C1 OXCMYAYHXIHQOA-UHFFFAOYSA-N 0.000 description 2
- 230000000069 prophylactic effect Effects 0.000 description 2
- 210000002307 prostate Anatomy 0.000 description 2
- 108020001580 protein domains Proteins 0.000 description 2
- 238000000746 purification Methods 0.000 description 2
- 238000004445 quantitative analysis Methods 0.000 description 2
- 230000004044 response Effects 0.000 description 2
- 201000000980 schizophrenia Diseases 0.000 description 2
- QZAYGJVTTNCVMB-UHFFFAOYSA-N serotonin Chemical compound C1=C(O)C=C2C(CCN)=CNC2=C1 QZAYGJVTTNCVMB-UHFFFAOYSA-N 0.000 description 2
- 108091029842 small nuclear ribonucleic acid Proteins 0.000 description 2
- 210000002460 smooth muscle Anatomy 0.000 description 2
- 239000000344 soap Substances 0.000 description 2
- 239000000243 solution Substances 0.000 description 2
- NHXLMOGPVYXJNR-ATOGVRKGSA-N somatostatin Chemical compound C([C@H]1C(=O)N[C@H](C(N[C@@H](CO)C(=O)N[C@@H](CSSC[C@@H](C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](CC=2C=CC=CC=2)C(=O)N[C@@H](CC=2C=CC=CC=2)C(=O)N[C@@H](CC=2C3=CC=CC=C3NC=2)C(=O)N[C@@H](CCCCN)C(=O)N[C@H](C(=O)N1)[C@@H](C)O)NC(=O)CNC(=O)[C@H](C)N)C(O)=O)=O)[C@H](O)C)C1=CC=CC=C1 NHXLMOGPVYXJNR-ATOGVRKGSA-N 0.000 description 2
- 229960000553 somatostatin Drugs 0.000 description 2
- 238000001228 spectrum Methods 0.000 description 2
- UCSJYZPVAKXKNQ-HZYVHMACSA-N streptomycin Chemical compound CN[C@H]1[C@H](O)[C@@H](O)[C@H](CO)O[C@H]1O[C@@H]1[C@](C=O)(O)[C@H](C)O[C@H]1O[C@@H]1[C@@H](NC(N)=N)[C@H](O)[C@@H](NC(N)=N)[C@H](O)[C@H]1O UCSJYZPVAKXKNQ-HZYVHMACSA-N 0.000 description 2
- WWJZWCUNLNYYAU-UHFFFAOYSA-N temephos Chemical compound C1=CC(OP(=S)(OC)OC)=CC=C1SC1=CC=C(OP(=S)(OC)OC)C=C1 WWJZWCUNLNYYAU-UHFFFAOYSA-N 0.000 description 2
- 108060008226 thioredoxin Proteins 0.000 description 2
- 238000010361 transduction Methods 0.000 description 2
- 230000026683 transduction Effects 0.000 description 2
- 238000011426 transformation method Methods 0.000 description 2
- 230000032258 transport Effects 0.000 description 2
- 241000712461 unidentified influenza virus Species 0.000 description 2
- 238000001262 western blot Methods 0.000 description 2
- 108091005957 yellow fluorescent proteins Proteins 0.000 description 2
- OPCHFPHZPIURNA-MFERNQICSA-N (2s)-2,5-bis(3-aminopropylamino)-n-[2-(dioctadecylamino)acetyl]pentanamide Chemical compound CCCCCCCCCCCCCCCCCCN(CC(=O)NC(=O)[C@H](CCCNCCCN)NCCCN)CCCCCCCCCCCCCCCCCC OPCHFPHZPIURNA-MFERNQICSA-N 0.000 description 1
- 101150028074 2 gene Proteins 0.000 description 1
- NHBKXEKEPDILRR-UHFFFAOYSA-N 2,3-bis(butanoylsulfanyl)propyl butanoate Chemical compound CCCC(=O)OCC(SC(=O)CCC)CSC(=O)CCC NHBKXEKEPDILRR-UHFFFAOYSA-N 0.000 description 1
- JKMHFZQWWAIEOD-UHFFFAOYSA-N 2-[4-(2-hydroxyethyl)piperazin-1-yl]ethanesulfonic acid Chemical compound OCC[NH+]1CCN(CCS([O-])(=O)=O)CC1 JKMHFZQWWAIEOD-UHFFFAOYSA-N 0.000 description 1
- FWBHETKCLVMNFS-UHFFFAOYSA-N 4',6-Diamino-2-phenylindol Chemical compound C1=CC(C(=N)N)=CC=C1C1=CC2=CC=C(C(N)=N)C=C2N1 FWBHETKCLVMNFS-UHFFFAOYSA-N 0.000 description 1
- UHPMCKVQTMMPCG-UHFFFAOYSA-N 5,8-dihydroxy-2-methoxy-6-methyl-7-(2-oxopropyl)naphthalene-1,4-dione Chemical compound CC1=C(CC(C)=O)C(O)=C2C(=O)C(OC)=CC(=O)C2=C1O UHPMCKVQTMMPCG-UHFFFAOYSA-N 0.000 description 1
- 102100024959 5-hydroxytryptamine receptor 2C Human genes 0.000 description 1
- 101150020052 AADAT gene Proteins 0.000 description 1
- 108091022885 ADAM Proteins 0.000 description 1
- 101150046889 ADORA3 gene Proteins 0.000 description 1
- 102000017907 ADRA1D Human genes 0.000 description 1
- 102000017906 ADRA2A Human genes 0.000 description 1
- 102000017904 ADRA2C Human genes 0.000 description 1
- 101710184468 AF4/FMR2 family member 2 Proteins 0.000 description 1
- 101150037123 APOE gene Proteins 0.000 description 1
- 102000000872 ATM Human genes 0.000 description 1
- 102100024643 ATP-binding cassette sub-family D member 1 Human genes 0.000 description 1
- 208000034012 Acid sphingomyelinase deficiency Diseases 0.000 description 1
- 241000589291 Acinetobacter Species 0.000 description 1
- 241001502050 Acis Species 0.000 description 1
- 101710192004 Actin, aortic smooth muscle Proteins 0.000 description 1
- 108010085238 Actins Proteins 0.000 description 1
- 102000007469 Actins Human genes 0.000 description 1
- ORILYTVJVMAKLC-UHFFFAOYSA-N Adamantane Natural products C1C(C2)CC3CC1CC2C3 ORILYTVJVMAKLC-UHFFFAOYSA-N 0.000 description 1
- 108010060261 Adenosine A3 Receptor Proteins 0.000 description 1
- 102000008161 Adenosine A3 Receptor Human genes 0.000 description 1
- 102100036006 Adenosine receptor A3 Human genes 0.000 description 1
- 201000011452 Adrenoleukodystrophy Diseases 0.000 description 1
- 241000567147 Aeropyrum Species 0.000 description 1
- 101150029691 Aff2 gene Proteins 0.000 description 1
- 235000001674 Agaricus brunnescens Nutrition 0.000 description 1
- 208000000363 Agenesis of Corpus Callosum Diseases 0.000 description 1
- HJCMDXDYPOUFDY-WHFBIAKZSA-N Ala-Gln Chemical compound C[C@H](N)C(=O)N[C@H](C(O)=O)CCC(N)=O HJCMDXDYPOUFDY-WHFBIAKZSA-N 0.000 description 1
- 108010088751 Albumins Proteins 0.000 description 1
- 208000011403 Alexander disease Diseases 0.000 description 1
- 208000006829 Allan-Herndon-Dudley syndrome Diseases 0.000 description 1
- 208000023434 Alpers-Huttenlocher syndrome Diseases 0.000 description 1
- 208000029602 Alpha-N-acetylgalactosaminidase deficiency Diseases 0.000 description 1
- 102100026882 Alpha-synuclein Human genes 0.000 description 1
- 206010068783 Alstroem syndrome Diseases 0.000 description 1
- 201000005932 Alstrom Syndrome Diseases 0.000 description 1
- 241000192542 Anabaena Species 0.000 description 1
- 208000009575 Angelman syndrome Diseases 0.000 description 1
- 102100034594 Angiopoietin-1 Human genes 0.000 description 1
- 101710095339 Apolipoprotein E Proteins 0.000 description 1
- 241000207208 Aquifex Species 0.000 description 1
- 241000205046 Archaeoglobus Species 0.000 description 1
- 239000004475 Arginine Substances 0.000 description 1
- 241000238421 Arthropoda Species 0.000 description 1
- 101150018431 Arx gene Proteins 0.000 description 1
- 206010003594 Ataxia telangiectasia Diseases 0.000 description 1
- 101150054444 Atp7a gene Proteins 0.000 description 1
- 206010003805 Autism Diseases 0.000 description 1
- 208000020706 Autistic disease Diseases 0.000 description 1
- 102100026189 Beta-galactosidase Human genes 0.000 description 1
- BTBUEUYNUDRHOZ-UHFFFAOYSA-N Borate Chemical compound [O-]B([O-])[O-] BTBUEUYNUDRHOZ-UHFFFAOYSA-N 0.000 description 1
- 108091003079 Bovine Serum Albumin Proteins 0.000 description 1
- 206010048409 Brain malformation Diseases 0.000 description 1
- 102000004219 Brain-derived neurotrophic factor Human genes 0.000 description 1
- 108090000715 Brain-derived neurotrophic factor Proteins 0.000 description 1
- 240000002791 Brassica napus Species 0.000 description 1
- 235000004977 Brassica sinapistrum Nutrition 0.000 description 1
- 102100021943 C-C motif chemokine 2 Human genes 0.000 description 1
- 208000010482 CADASIL Diseases 0.000 description 1
- 108091008927 CC chemokine receptors Proteins 0.000 description 1
- 102000005674 CCR Receptors Human genes 0.000 description 1
- 101150083327 CCR2 gene Proteins 0.000 description 1
- 101150017501 CCR5 gene Proteins 0.000 description 1
- 101150008656 COL1A1 gene Proteins 0.000 description 1
- 101150069031 CSN2 gene Proteins 0.000 description 1
- 101100205787 Caenorhabditis elegans timm-17B.1 gene Proteins 0.000 description 1
- 240000001432 Calendula officinalis Species 0.000 description 1
- 235000005881 Calendula officinalis Nutrition 0.000 description 1
- 208000022526 Canavan disease Diseases 0.000 description 1
- 244000025254 Cannabis sativa Species 0.000 description 1
- 241000283707 Capra Species 0.000 description 1
- 102000053642 Catalytic RNA Human genes 0.000 description 1
- 108090000994 Catalytic RNA Proteins 0.000 description 1
- 102100021633 Cathepsin B Human genes 0.000 description 1
- 102100024940 Cathepsin K Human genes 0.000 description 1
- 206010008025 Cerebellar ataxia Diseases 0.000 description 1
- 208000033221 Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy Diseases 0.000 description 1
- 208000033935 Cerebral autosomal dominant arteriopathy-subcortical infarcts-leukoencephalopathy Diseases 0.000 description 1
- 206010053684 Cerebrohepatorenal syndrome Diseases 0.000 description 1
- 108050000299 Chemokine receptor Proteins 0.000 description 1
- 102000009410 Chemokine receptor Human genes 0.000 description 1
- VYZAMTAEIAYCRO-UHFFFAOYSA-N Chromium Chemical compound [Cr] VYZAMTAEIAYCRO-UHFFFAOYSA-N 0.000 description 1
- 241000588881 Chromobacterium Species 0.000 description 1
- 101100007328 Cocos nucifera COS-1 gene Proteins 0.000 description 1
- 102000002734 Collagen Type VI Human genes 0.000 description 1
- 108010043741 Collagen Type VI Proteins 0.000 description 1
- 102100033601 Collagen alpha-1(I) chain Human genes 0.000 description 1
- 206010010099 Combined immunodeficiency Diseases 0.000 description 1
- 102100027826 Complexin-1 Human genes 0.000 description 1
- 206010010356 Congenital anomaly Diseases 0.000 description 1
- 206010010411 Congenital central nervous system anomaly Diseases 0.000 description 1
- 208000029011 Copper metabolism disease Diseases 0.000 description 1
- 201000009343 Cornelia de Lange syndrome Diseases 0.000 description 1
- 241000186216 Corynebacterium Species 0.000 description 1
- 101150074155 DHFR gene Proteins 0.000 description 1
- 230000005971 DNA damage repair Effects 0.000 description 1
- 102100022286 DNA repair-scaffolding protein Human genes 0.000 description 1
- 102000004163 DNA-directed RNA polymerases Human genes 0.000 description 1
- 108090000626 DNA-directed RNA polymerases Proteins 0.000 description 1
- 208000003471 De Lange Syndrome Diseases 0.000 description 1
- 206010012289 Dementia Diseases 0.000 description 1
- 241000605716 Desulfovibrio Species 0.000 description 1
- 208000004986 Diffuse Cerebral Sclerosis of Schilder Diseases 0.000 description 1
- 102100022825 Disintegrin and metalloproteinase domain-containing protein 22 Human genes 0.000 description 1
- 241000004297 Draba Species 0.000 description 1
- 101100118093 Drosophila melanogaster eEF1alpha2 gene Proteins 0.000 description 1
- 239000006144 Dulbecco’s modified Eagle's medium Substances 0.000 description 1
- 206010058314 Dysplasia Diseases 0.000 description 1
- 102000001301 EGF receptor Human genes 0.000 description 1
- 108060006698 EGF receptor Proteins 0.000 description 1
- 102100027723 Endogenous retrovirus group K member 6 Rec protein Human genes 0.000 description 1
- 108010041308 Endothelial Growth Factors Proteins 0.000 description 1
- 108010013369 Enteropeptidase Proteins 0.000 description 1
- 102100029727 Enteropeptidase Human genes 0.000 description 1
- 101710091045 Envelope protein Proteins 0.000 description 1
- 206010053177 Epidermolysis Diseases 0.000 description 1
- 102100033176 Epithelial membrane protein 2 Human genes 0.000 description 1
- 241000588698 Erwinia Species 0.000 description 1
- 108090000394 Erythropoietin Proteins 0.000 description 1
- 241000588722 Escherichia Species 0.000 description 1
- 241000186394 Eubacterium Species 0.000 description 1
- 241000206602 Eukaryota Species 0.000 description 1
- 101150107205 FCGR2 gene Proteins 0.000 description 1
- 101150061264 FXR1 gene Proteins 0.000 description 1
- 208000024720 Fabry Disease Diseases 0.000 description 1
- 208000035126 Facies Diseases 0.000 description 1
- 108010074860 Factor Xa Proteins 0.000 description 1
- 101150114401 Fcer1g gene Proteins 0.000 description 1
- 108010032606 Fragile X Mental Retardation Protein Proteins 0.000 description 1
- 102100036334 Fragile X mental retardation syndrome-related protein 1 Human genes 0.000 description 1
- 102100036336 Fragile X mental retardation syndrome-related protein 2 Human genes 0.000 description 1
- 241000223218 Fusarium Species 0.000 description 1
- 241000223221 Fusarium oxysporum Species 0.000 description 1
- 241000611205 Fusarium oxysporum f. sp. lycopersici Species 0.000 description 1
- 241000605909 Fusobacterium Species 0.000 description 1
- 208000017462 Galactosialidosis Diseases 0.000 description 1
- 102100039556 Galectin-4 Human genes 0.000 description 1
- 208000015872 Gaucher disease Diseases 0.000 description 1
- 108700028146 Genetic Enhancer Elements Proteins 0.000 description 1
- 108700007698 Genetic Terminator Regions Proteins 0.000 description 1
- 208000031448 Genomic Instability Diseases 0.000 description 1
- 241001135750 Geobacter Species 0.000 description 1
- 241000713813 Gibbon ape leukemia virus Species 0.000 description 1
- 108010060309 Glucuronidase Proteins 0.000 description 1
- 102000053187 Glucuronidase Human genes 0.000 description 1
- 239000004471 Glycine Substances 0.000 description 1
- 229920002527 Glycogen Polymers 0.000 description 1
- 108010051975 Glycogen Synthase Kinase 3 beta Proteins 0.000 description 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 description 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 description 1
- 102100022975 Glycogen synthase kinase-3 alpha Human genes 0.000 description 1
- 102100038104 Glycogen synthase kinase-3 beta Human genes 0.000 description 1
- HVLSXIKZNLPZJJ-TXZCQADKSA-N HA peptide Chemical compound C([C@@H](C(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](C(C)C)C(=O)N1[C@@H](CCC1)C(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](CC=1C=CC(O)=CC=1)C(=O)N[C@@H](C)C(O)=O)NC(=O)[C@H]1N(CCC1)C(=O)[C@@H](N)CC=1C=CC(O)=CC=1)C1=CC=C(O)C=C1 HVLSXIKZNLPZJJ-TXZCQADKSA-N 0.000 description 1
- 239000007995 HEPES buffer Substances 0.000 description 1
- 241000204988 Haloferax mediterranei Species 0.000 description 1
- 101710154606 Hemagglutinin Proteins 0.000 description 1
- 102100021519 Hemoglobin subunit beta Human genes 0.000 description 1
- 108091005904 Hemoglobin subunit beta Proteins 0.000 description 1
- 208000036066 Hemophagocytic Lymphohistiocytosis Diseases 0.000 description 1
- 101001023784 Heteractis crispa GFP-like non-fluorescent chromoprotein Proteins 0.000 description 1
- 102000016871 Hexosaminidase A Human genes 0.000 description 1
- 108010053317 Hexosaminidase A Proteins 0.000 description 1
- 208000032672 Histiocytosis haematophagic Diseases 0.000 description 1
- 108010033040 Histones Proteins 0.000 description 1
- 108700005087 Homeobox Genes Proteins 0.000 description 1
- 241000282412 Homo Species 0.000 description 1
- 101000761348 Homo sapiens 5-hydroxytryptamine receptor 2C Proteins 0.000 description 1
- 101000800430 Homo sapiens ATP-binding cassette sub-family G member 8 Proteins 0.000 description 1
- 101000929319 Homo sapiens Actin, aortic smooth muscle Proteins 0.000 description 1
- 101000783645 Homo sapiens Adenosine receptor A3 Proteins 0.000 description 1
- 101000775498 Homo sapiens Adenylate cyclase type 10 Proteins 0.000 description 1
- 101000689696 Homo sapiens Alpha-1D adrenergic receptor Proteins 0.000 description 1
- 101000924552 Homo sapiens Angiopoietin-1 Proteins 0.000 description 1
- 101000898449 Homo sapiens Cathepsin B Proteins 0.000 description 1
- 101000761509 Homo sapiens Cathepsin K Proteins 0.000 description 1
- 101000859600 Homo sapiens Complexin-1 Proteins 0.000 description 1
- 101000825159 Homo sapiens DNA repair-scaffolding protein Proteins 0.000 description 1
- 101000756722 Homo sapiens Disintegrin and metalloproteinase domain-containing protein 22 Proteins 0.000 description 1
- 101000619542 Homo sapiens E3 ubiquitin-protein ligase parkin Proteins 0.000 description 1
- 101000938351 Homo sapiens Ephrin type-A receptor 3 Proteins 0.000 description 1
- 101000851002 Homo sapiens Epithelial membrane protein 2 Proteins 0.000 description 1
- 101100335685 Homo sapiens FXR2 gene Proteins 0.000 description 1
- 101000930945 Homo sapiens Fragile X mental retardation syndrome-related protein 1 Proteins 0.000 description 1
- 101000930952 Homo sapiens Fragile X mental retardation syndrome-related protein 2 Proteins 0.000 description 1
- 101000608765 Homo sapiens Galectin-4 Proteins 0.000 description 1
- 101000903717 Homo sapiens Glycogen synthase kinase-3 alpha Proteins 0.000 description 1
- 101000843809 Homo sapiens Hydroxycarboxylic acid receptor 2 Proteins 0.000 description 1
- 101001033249 Homo sapiens Interleukin-1 beta Proteins 0.000 description 1
- 101000929733 Homo sapiens Kynurenine/alpha-aminoadipate aminotransferase, mitochondrial Proteins 0.000 description 1
- 101100127663 Homo sapiens LAMA2 gene Proteins 0.000 description 1
- 101000917826 Homo sapiens Low affinity immunoglobulin gamma Fc region receptor II-a Proteins 0.000 description 1
- 101000917824 Homo sapiens Low affinity immunoglobulin gamma Fc region receptor II-b Proteins 0.000 description 1
- 101001057159 Homo sapiens Melanoma-associated antigen C3 Proteins 0.000 description 1
- 101000615488 Homo sapiens Methyl-CpG-binding domain protein 2 Proteins 0.000 description 1
- 101000837517 Homo sapiens NEDD8-activating enzyme E1 catalytic subunit Proteins 0.000 description 1
- 101001108436 Homo sapiens Neurexin-1 Proteins 0.000 description 1
- 101001108433 Homo sapiens Neurexin-1-beta Proteins 0.000 description 1
- 101001109698 Homo sapiens Nuclear receptor subfamily 4 group A member 2 Proteins 0.000 description 1
- 101000801640 Homo sapiens Phospholipid-transporting ATPase ABCA3 Proteins 0.000 description 1
- 101000617536 Homo sapiens Presenilin-1 Proteins 0.000 description 1
- 101000617546 Homo sapiens Presenilin-2 Proteins 0.000 description 1
- 101001001272 Homo sapiens Prostatic acid phosphatase Proteins 0.000 description 1
- 101000600434 Homo sapiens Putative uncharacterized protein encoded by MIR7-3HG Proteins 0.000 description 1
- 101001012157 Homo sapiens Receptor tyrosine-protein kinase erbB-2 Proteins 0.000 description 1
- 101000785063 Homo sapiens Serine-protein kinase ATM Proteins 0.000 description 1
- 101000605835 Homo sapiens Serine/threonine-protein kinase PINK1, mitochondrial Proteins 0.000 description 1
- 101000600903 Homo sapiens Substance-P receptor Proteins 0.000 description 1
- 101000828537 Homo sapiens Synaptic functional regulator FMR1 Proteins 0.000 description 1
- 101000892398 Homo sapiens Tryptophan 2,3-dioxygenase Proteins 0.000 description 1
- 101000830742 Homo sapiens Tryptophan 5-hydroxylase 1 Proteins 0.000 description 1
- 101000851865 Homo sapiens Tryptophan 5-hydroxylase 2 Proteins 0.000 description 1
- 101000807306 Homo sapiens Ubiquitin-like modifier-activating enzyme 1 Proteins 0.000 description 1
- 206010020460 Human T-cell lymphotropic virus type I infection Diseases 0.000 description 1
- 241000714260 Human T-lymphotropic virus 1 Species 0.000 description 1
- 241000701109 Human adenovirus 2 Species 0.000 description 1
- 241000725303 Human immunodeficiency virus Species 0.000 description 1
- 208000025500 Hutchinson-Gilford progeria syndrome Diseases 0.000 description 1
- 102100030643 Hydroxycarboxylic acid receptor 2 Human genes 0.000 description 1
- 108060003951 Immunoglobulin Proteins 0.000 description 1
- 102100039065 Interleukin-1 beta Human genes 0.000 description 1
- 108091092195 Intron Proteins 0.000 description 1
- AHLPHDHHMVZTML-BYPYZUCNSA-N L-Ornithine Chemical compound NCCC[C@H](N)C(O)=O AHLPHDHHMVZTML-BYPYZUCNSA-N 0.000 description 1
- 101150089565 LAMA2 gene Proteins 0.000 description 1
- 241000186660 Lactobacillus Species 0.000 description 1
- 241000589248 Legionella Species 0.000 description 1
- 208000007764 Legionnaires' Disease Diseases 0.000 description 1
- 108010020246 Leucine-Rich Repeat Serine-Threonine Protein Kinase-2 Proteins 0.000 description 1
- 102100032693 Leucine-rich repeat serine/threonine-protein kinase 2 Human genes 0.000 description 1
- 239000012097 Lipofectamine 2000 Substances 0.000 description 1
- 101150034680 Lis-1 gene Proteins 0.000 description 1
- 206010048911 Lissencephaly Diseases 0.000 description 1
- 102100029204 Low affinity immunoglobulin gamma Fc region receptor II-a Human genes 0.000 description 1
- 108060001084 Luciferase Proteins 0.000 description 1
- 239000005089 Luciferase Substances 0.000 description 1
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 description 1
- 239000004472 Lysine Substances 0.000 description 1
- 102100026001 Lysosomal acid lipase/cholesteryl ester hydrolase Human genes 0.000 description 1
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 description 1
- 239000007993 MOPS buffer Substances 0.000 description 1
- 208000030162 Maple syrup disease Diseases 0.000 description 1
- 108010049137 Member 1 Subfamily D ATP Binding Cassette Transporter Proteins 0.000 description 1
- 108010090822 Member 8 Subfamily G ATP Binding Cassette Transporter Proteins 0.000 description 1
- 208000008948 Menkes Kinky Hair Syndrome Diseases 0.000 description 1
- 208000012583 Menkes disease Diseases 0.000 description 1
- 201000011442 Metachromatic leukodystrophy Diseases 0.000 description 1
- 102000005741 Metalloproteases Human genes 0.000 description 1
- 108010006035 Metalloproteases Proteins 0.000 description 1
- 241000202974 Methanobacterium Species 0.000 description 1
- 241000203353 Methanococcus Species 0.000 description 1
- 241000204675 Methanopyrus Species 0.000 description 1
- 241000205276 Methanosarcina Species 0.000 description 1
- 102100021299 Methyl-CpG-binding domain protein 2 Human genes 0.000 description 1
- 102100039124 Methyl-CpG-binding protein 2 Human genes 0.000 description 1
- 201000002169 Mitochondrial myopathy Diseases 0.000 description 1
- 208000034819 Mobility Limitation Diseases 0.000 description 1
- 208000033180 Monosomy 22q13.3 Diseases 0.000 description 1
- 208000008955 Mucolipidoses Diseases 0.000 description 1
- 206010072928 Mucolipidosis type II Diseases 0.000 description 1
- 208000002678 Mucopolysaccharidoses Diseases 0.000 description 1
- 101000981253 Mus musculus GPI-linked NAD(P)(+)-arginine ADP-ribosyltransferase 1 Proteins 0.000 description 1
- 241000699670 Mus sp. Species 0.000 description 1
- 208000007101 Muscle Cramp Diseases 0.000 description 1
- 101710135898 Myc proto-oncogene protein Proteins 0.000 description 1
- 102100038895 Myc proto-oncogene protein Human genes 0.000 description 1
- 241000186359 Mycobacterium Species 0.000 description 1
- 241000187479 Mycobacterium tuberculosis Species 0.000 description 1
- 241000204031 Mycoplasma Species 0.000 description 1
- 241000863420 Myxococcus Species 0.000 description 1
- PJKKQFAEFWCNAQ-UHFFFAOYSA-N N(4)-methylcytosine Chemical class CNC=1C=CNC(=O)N=1 PJKKQFAEFWCNAQ-UHFFFAOYSA-N 0.000 description 1
- 108700037255 NEDD8-activating enzyme E1 catalytic subunit Proteins 0.000 description 1
- 108091061960 Naked DNA Proteins 0.000 description 1
- 241000244206 Nematoda Species 0.000 description 1
- 102000048850 Neoplasm Genes Human genes 0.000 description 1
- 108700019961 Neoplasm Genes Proteins 0.000 description 1
- 102000048238 Neuregulin-1 Human genes 0.000 description 1
- 108090000556 Neuregulin-1 Proteins 0.000 description 1
- 102100021582 Neurexin-1-beta Human genes 0.000 description 1
- 108010088373 Neurofilament Proteins Proteins 0.000 description 1
- 101100058191 Neurospora crassa (strain ATCC 24698 / 74-OR23-1A / CBS 708.71 / DSM 1257 / FGSC 987) bcp-1 gene Proteins 0.000 description 1
- 101100385413 Neurospora crassa (strain ATCC 24698 / 74-OR23-1A / CBS 708.71 / DSM 1257 / FGSC 987) csm-3 gene Proteins 0.000 description 1
- 108010008858 Nitric Oxide Synthase Type I Proteins 0.000 description 1
- 241000605122 Nitrosomonas Species 0.000 description 1
- 208000035544 Nonketotic hyperglycinaemia Diseases 0.000 description 1
- 108010077850 Nuclear Localization Signals Proteins 0.000 description 1
- 102100022676 Nuclear receptor subfamily 4 group A member 2 Human genes 0.000 description 1
- 108091005461 Nucleic proteins Proteins 0.000 description 1
- 208000019851 Opitz G/BBB syndrome Diseases 0.000 description 1
- AHLPHDHHMVZTML-UHFFFAOYSA-N Orn-delta-NH2 Natural products NCCCC(N)C(O)=O AHLPHDHHMVZTML-UHFFFAOYSA-N 0.000 description 1
- UTJLXEIPEHZYQJ-UHFFFAOYSA-N Ornithine Natural products OC(=O)C(C)CCCN UTJLXEIPEHZYQJ-UHFFFAOYSA-N 0.000 description 1
- 101710093908 Outer capsid protein VP4 Proteins 0.000 description 1
- 101710135467 Outer capsid protein sigma-1 Proteins 0.000 description 1
- 238000012408 PCR amplification Methods 0.000 description 1
- 101150075879 PLA2G6 gene Proteins 0.000 description 1
- 101150082519 PLP1 gene Proteins 0.000 description 1
- 101150078890 POLG gene Proteins 0.000 description 1
- 108010011536 PTEN Phosphohydrolase Proteins 0.000 description 1
- 241001494479 Pecora Species 0.000 description 1
- 229930182555 Penicillin Natural products 0.000 description 1
- JGSARLDLIJGVTE-MBNYWOFBSA-N Penicillin G Chemical compound N([C@H]1[C@H]2SC([C@@H](N2C1=O)C(O)=O)(C)C)C(=O)CC1=CC=CC=C1 JGSARLDLIJGVTE-MBNYWOFBSA-N 0.000 description 1
- 241000009328 Perro Species 0.000 description 1
- 201000004316 Perry syndrome Diseases 0.000 description 1
- 201000006880 Phelan-McDermid syndrome Diseases 0.000 description 1
- 102100032543 Phosphatidylinositol 3,4,5-trisphosphate 3-phosphatase and dual-specificity protein phosphatase PTEN Human genes 0.000 description 1
- 102100033623 Phospholipid-transporting ATPase ABCA3 Human genes 0.000 description 1
- 108091000080 Phosphotransferase Proteins 0.000 description 1
- 241000204826 Picrophilus Species 0.000 description 1
- 102000012338 Poly(ADP-ribose) Polymerases Human genes 0.000 description 1
- 108010061844 Poly(ADP-ribose) Polymerases Proteins 0.000 description 1
- 229920000776 Poly(Adenosine diphosphate-ribose) polymerase Polymers 0.000 description 1
- 241001505332 Polyomavirus sp. Species 0.000 description 1
- 108010035004 Prephenate Dehydrogenase Proteins 0.000 description 1
- 102000029797 Prion Human genes 0.000 description 1
- 108091000054 Prion Proteins 0.000 description 1
- 208000007932 Progeria Diseases 0.000 description 1
- 208000033063 Progressive myoclonic epilepsy Diseases 0.000 description 1
- 102100035703 Prostatic acid phosphatase Human genes 0.000 description 1
- 101710176177 Protein A56 Proteins 0.000 description 1
- 102000007659 Protein Deglycase DJ-1 Human genes 0.000 description 1
- 108010032428 Protein Deglycase DJ-1 Proteins 0.000 description 1
- 108010029485 Protein Isoforms Proteins 0.000 description 1
- 102000001708 Protein Isoforms Human genes 0.000 description 1
- 102000001253 Protein Kinase Human genes 0.000 description 1
- 229940096437 Protein S Drugs 0.000 description 1
- 101710188315 Protein X Proteins 0.000 description 1
- 241000567197 Puccinia graminis f. sp. tritici Species 0.000 description 1
- 102100037401 Putative uncharacterized protein encoded by MIR7-3HG Human genes 0.000 description 1
- 241000205226 Pyrobaculum Species 0.000 description 1
- 241000205160 Pyrococcus Species 0.000 description 1
- 102000014450 RNA Polymerase III Human genes 0.000 description 1
- 108010078067 RNA Polymerase III Proteins 0.000 description 1
- 102100038188 RNA binding protein fox-1 homolog 1 Human genes 0.000 description 1
- 101710199540 RNA binding protein fox-1 homolog 1 Proteins 0.000 description 1
- 101100240886 Rattus norvegicus Nptx2 gene Proteins 0.000 description 1
- 101100047461 Rattus norvegicus Trpm8 gene Proteins 0.000 description 1
- 102100030086 Receptor tyrosine-protein kinase erbB-2 Human genes 0.000 description 1
- 101710100969 Receptor tyrosine-protein kinase erbB-3 Proteins 0.000 description 1
- 102100029986 Receptor tyrosine-protein kinase erbB-3 Human genes 0.000 description 1
- 102100029981 Receptor tyrosine-protein kinase erbB-4 Human genes 0.000 description 1
- 101710100963 Receptor tyrosine-protein kinase erbB-4 Proteins 0.000 description 1
- 201000000582 Retinoblastoma Diseases 0.000 description 1
- 108091028664 Ribonucleotide Proteins 0.000 description 1
- 201000001718 Roberts syndrome Diseases 0.000 description 1
- 101100170553 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) DLD2 gene Proteins 0.000 description 1
- 208000021811 Sandhoff disease Diseases 0.000 description 1
- 101100023124 Schizosaccharomyces pombe (strain 972 / ATCC 24843) mfr2 gene Proteins 0.000 description 1
- 102100020824 Serine-protein kinase ATM Human genes 0.000 description 1
- 102100038376 Serine/threonine-protein kinase PINK1, mitochondrial Human genes 0.000 description 1
- 102100030333 Serpin B5 Human genes 0.000 description 1
- 241000949716 Sphaerochaeta Species 0.000 description 1
- 108010055297 Sterol Esterase Proteins 0.000 description 1
- 241000187747 Streptomyces Species 0.000 description 1
- 241000282887 Suidae Species 0.000 description 1
- 108091008874 T cell receptors Proteins 0.000 description 1
- 102000016266 T-Cell Antigen Receptors Human genes 0.000 description 1
- 238000010459 TALEN Methods 0.000 description 1
- 241000255588 Tephritidae Species 0.000 description 1
- 241000186339 Thermoanaerobacter Species 0.000 description 1
- 241000204667 Thermoplasma Species 0.000 description 1
- 229910052776 Thorium Inorganic materials 0.000 description 1
- 108010022394 Threonine synthase Proteins 0.000 description 1
- 108090000190 Thrombin Proteins 0.000 description 1
- 241000283907 Tragelaphus oryx Species 0.000 description 1
- 108010043645 Transcription Activator-Like Effector Nucleases Proteins 0.000 description 1
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 description 1
- 101710150448 Transcriptional regulator Myc Proteins 0.000 description 1
- 239000007983 Tris buffer Substances 0.000 description 1
- 241000209140 Triticum Species 0.000 description 1
- 235000021307 Triticum Nutrition 0.000 description 1
- 102100040653 Tryptophan 2,3-dioxygenase Human genes 0.000 description 1
- 102100024971 Tryptophan 5-hydroxylase 1 Human genes 0.000 description 1
- 108010078814 Tumor Suppressor Protein p53 Proteins 0.000 description 1
- 101150044377 UBA1 gene Proteins 0.000 description 1
- 101150035006 UBA3 gene Proteins 0.000 description 1
- 108010005656 Ubiquitin Thiolesterase Proteins 0.000 description 1
- 102000005918 Ubiquitin Thiolesterase Human genes 0.000 description 1
- 241000251539 Vertebrata <Metazoa> Species 0.000 description 1
- 101710177612 Very low-density lipoprotein receptor Proteins 0.000 description 1
- 108020005202 Viral DNA Proteins 0.000 description 1
- 108700005077 Viral Genes Proteins 0.000 description 1
- 102100028885 Vitamin K-dependent protein S Human genes 0.000 description 1
- 101150115477 Vldlr gene Proteins 0.000 description 1
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 description 1
- 239000005862 Whey Substances 0.000 description 1
- 102000007544 Whey Proteins Human genes 0.000 description 1
- 108010046377 Whey Proteins Proteins 0.000 description 1
- 208000006254 Wolf-Hirschhorn Syndrome Diseases 0.000 description 1
- 241000605941 Wolinella Species 0.000 description 1
- 210000001766 X chromosome Anatomy 0.000 description 1
- 206010048215 Xanthomatosis Diseases 0.000 description 1
- 101000678336 Xenopus laevis Actin, alpha skeletal muscle 2 Proteins 0.000 description 1
- 101000678338 Xenopus tropicalis Actin, alpha cardiac muscle 2 Proteins 0.000 description 1
- 206010048218 Xeroderma Diseases 0.000 description 1
- 201000004525 Zellweger Syndrome Diseases 0.000 description 1
- 208000036813 Zellweger spectrum disease Diseases 0.000 description 1
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 description 1
- HMNZFMSWFCAGGW-XPWSMXQVSA-N [3-[hydroxy(2-hydroxyethoxy)phosphoryl]oxy-2-[(e)-octadec-9-enoyl]oxypropyl] (e)-octadec-9-enoate Chemical compound CCCCCCCC\C=C\CCCCCCCC(=O)OCC(COP(O)(=O)OCCO)OC(=O)CCCCCCC\C=C\CCCCCCCC HMNZFMSWFCAGGW-XPWSMXQVSA-N 0.000 description 1
- 230000021736 acetylation Effects 0.000 description 1
- 238000006640 acetylation reaction Methods 0.000 description 1
- 239000002253 acid Substances 0.000 description 1
- 150000007513 acids Chemical class 0.000 description 1
- 230000004913 activation Effects 0.000 description 1
- 201000009628 adenosine deaminase deficiency Diseases 0.000 description 1
- 238000001261 affinity purification Methods 0.000 description 1
- 206010064930 age-related macular degeneration Diseases 0.000 description 1
- 230000009418 agronomic effect Effects 0.000 description 1
- 102000009899 alpha Karyopherins Human genes 0.000 description 1
- 108010077099 alpha Karyopherins Proteins 0.000 description 1
- 108010026331 alpha-Fetoproteins Proteins 0.000 description 1
- 108090000185 alpha-Synuclein Proteins 0.000 description 1
- AWUCVROLDVIAJX-UHFFFAOYSA-N alpha-glycerophosphate Natural products OCC(O)COP(O)(O)=O AWUCVROLDVIAJX-UHFFFAOYSA-N 0.000 description 1
- 201000008333 alpha-mannosidosis Diseases 0.000 description 1
- 238000010171 animal model Methods 0.000 description 1
- 239000000427 antigen Substances 0.000 description 1
- 108091007433 antigens Proteins 0.000 description 1
- 102000036639 antigens Human genes 0.000 description 1
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 description 1
- 208000029560 autism spectrum disease Diseases 0.000 description 1
- 208000036556 autosomal recessive T cell-negative B cell-negative NK cell-negative due to adenosine deaminase deficiency severe combined immunodeficiency Diseases 0.000 description 1
- 208000005980 beta thalassemia Diseases 0.000 description 1
- 108010005774 beta-Galactosidase Proteins 0.000 description 1
- SQVRNKJHWKZAKO-UHFFFAOYSA-N beta-N-Acetyl-D-neuraminic acid Natural products CC(=O)NC1C(O)CC(O)(C(O)=O)OC1C(O)C(O)CO SQVRNKJHWKZAKO-UHFFFAOYSA-N 0.000 description 1
- 230000008436 biogenesis Effects 0.000 description 1
- 238000001369 bisulfite sequencing Methods 0.000 description 1
- 210000004369 blood Anatomy 0.000 description 1
- 239000008280 blood Substances 0.000 description 1
- 210000004556 brain Anatomy 0.000 description 1
- 210000000481 breast Anatomy 0.000 description 1
- 201000011510 cancer Diseases 0.000 description 1
- 150000001720 carbohydrates Chemical class 0.000 description 1
- 235000014633 carbohydrates Nutrition 0.000 description 1
- 229910002091 carbon monoxide Inorganic materials 0.000 description 1
- 210000000748 cardiovascular system Anatomy 0.000 description 1
- 108020001778 catalytic domains Proteins 0.000 description 1
- 125000002091 cationic group Chemical group 0.000 description 1
- 230000022131 cell cycle Effects 0.000 description 1
- 230000030833 cell death Effects 0.000 description 1
- 230000032823 cell division Effects 0.000 description 1
- 239000013592 cell lysate Substances 0.000 description 1
- 210000003855 cell nucleus Anatomy 0.000 description 1
- 230000005754 cellular signaling Effects 0.000 description 1
- 235000013339 cereals Nutrition 0.000 description 1
- 208000025434 cerebellar degeneration Diseases 0.000 description 1
- 208000016886 cerebral arteriopathy with subcortical infarcts and leukoencephalopathy Diseases 0.000 description 1
- 210000003710 cerebral cortex Anatomy 0.000 description 1
- 210000003763 chloroplast Anatomy 0.000 description 1
- 208000017568 chondrodysplasia Diseases 0.000 description 1
- 229910052804 chromium Inorganic materials 0.000 description 1
- 239000011651 chromium Substances 0.000 description 1
- 210000000349 chromosome Anatomy 0.000 description 1
- 238000010367 cloning Methods 0.000 description 1
- 238000012761 co-transfection Methods 0.000 description 1
- 239000003086 colorant Substances 0.000 description 1
- 208000003536 colpocephaly Diseases 0.000 description 1
- 239000002299 complementary DNA Substances 0.000 description 1
- 238000004590 computer program Methods 0.000 description 1
- 239000012141 concentrate Substances 0.000 description 1
- 235000008504 concentrate Nutrition 0.000 description 1
- 230000021615 conjugation Effects 0.000 description 1
- 238000010276 construction Methods 0.000 description 1
- 238000011109 contamination Methods 0.000 description 1
- 101150055601 cops2 gene Proteins 0.000 description 1
- 238000012937 correction Methods 0.000 description 1
- 230000002596 correlated effect Effects 0.000 description 1
- 239000013078 crystal Substances 0.000 description 1
- 210000000805 cytoplasm Anatomy 0.000 description 1
- 230000034994 death Effects 0.000 description 1
- 230000007812 deficiency Effects 0.000 description 1
- 230000006735 deficit Effects 0.000 description 1
- 239000005547 deoxyribonucleotide Substances 0.000 description 1
- 125000002637 deoxyribonucleotide group Chemical group 0.000 description 1
- 238000003745 diagnosis Methods 0.000 description 1
- 238000010586 diagram Methods 0.000 description 1
- 230000029087 digestion Effects 0.000 description 1
- 102000004419 dihydrofolate reductase Human genes 0.000 description 1
- 238000004821 distillation Methods 0.000 description 1
- 238000009826 distribution Methods 0.000 description 1
- 238000005315 distribution function Methods 0.000 description 1
- 238000009510 drug design Methods 0.000 description 1
- 238000007877 drug screening Methods 0.000 description 1
- 239000012636 effector Substances 0.000 description 1
- 210000001671 embryonic stem cell Anatomy 0.000 description 1
- 230000002255 enzymatic effect Effects 0.000 description 1
- 238000006911 enzymatic reaction Methods 0.000 description 1
- 102000052116 epidermal growth factor receptor activity proteins Human genes 0.000 description 1
- 108700015053 epidermal growth factor receptor activity proteins Proteins 0.000 description 1
- 208000028329 epileptic seizure Diseases 0.000 description 1
- 230000002449 erythroblastic effect Effects 0.000 description 1
- 229940105423 erythropoietin Drugs 0.000 description 1
- 238000011156 evaluation Methods 0.000 description 1
- 230000001747 exhibiting effect Effects 0.000 description 1
- 239000003925 fat Substances 0.000 description 1
- 239000012091 fetal bovine serum Substances 0.000 description 1
- 230000001605 fetal effect Effects 0.000 description 1
- 230000004992 fission Effects 0.000 description 1
- 108010021843 fluorescent protein 583 Proteins 0.000 description 1
- 102000034287 fluorescent proteins Human genes 0.000 description 1
- 235000013305 food Nutrition 0.000 description 1
- 201000008049 fucosidosis Diseases 0.000 description 1
- 238000001502 gel electrophoresis Methods 0.000 description 1
- 238000010363 gene targeting Methods 0.000 description 1
- 239000003862 glucocorticoid Substances 0.000 description 1
- 201000011205 glycine encephalopathy Diseases 0.000 description 1
- 229940096919 glycogen Drugs 0.000 description 1
- 201000004502 glycogen storage disease II Diseases 0.000 description 1
- 230000013595 glycosylation Effects 0.000 description 1
- 238000006206 glycosylation reaction Methods 0.000 description 1
- 239000003102 growth factor Substances 0.000 description 1
- 230000009931 harmful effect Effects 0.000 description 1
- 238000010438 heat treatment Methods 0.000 description 1
- 239000000185 hemagglutinin Substances 0.000 description 1
- 208000014752 hemophagocytic syndrome Diseases 0.000 description 1
- 208000006454 hepatitis Diseases 0.000 description 1
- 231100000283 hepatitis Toxicity 0.000 description 1
- 210000003494 hepatocyte Anatomy 0.000 description 1
- 102000057382 human EPHA3 Human genes 0.000 description 1
- 102000049240 human PVALB Human genes 0.000 description 1
- 210000005119 human aortic smooth muscle cell Anatomy 0.000 description 1
- 206010021198 ichthyosis Diseases 0.000 description 1
- 238000003384 imaging method Methods 0.000 description 1
- 230000028993 immune response Effects 0.000 description 1
- 102000018358 immunoglobulin Human genes 0.000 description 1
- 229940072221 immunoglobulins Drugs 0.000 description 1
- 238000003364 immunohistochemistry Methods 0.000 description 1
- 230000001771 impaired effect Effects 0.000 description 1
- 230000008676 import Effects 0.000 description 1
- 230000002779 inactivation Effects 0.000 description 1
- 208000023692 inborn mitochondrial myopathy Diseases 0.000 description 1
- 238000011534 incubation Methods 0.000 description 1
- 230000006698 induction Effects 0.000 description 1
- 208000019143 inherited prion disease Diseases 0.000 description 1
- 230000009878 intermolecular interaction Effects 0.000 description 1
- 229910052742 iron Inorganic materials 0.000 description 1
- 101150044508 key gene Proteins 0.000 description 1
- 210000001985 kidney epithelial cell Anatomy 0.000 description 1
- 238000011005 laboratory method Methods 0.000 description 1
- 229940039696 lactobacillus Drugs 0.000 description 1
- 201000010260 leiomyoma Diseases 0.000 description 1
- 208000014817 lissencephaly spectrum disease Diseases 0.000 description 1
- 238000011068 loading method Methods 0.000 description 1
- 230000007774 longterm Effects 0.000 description 1
- 210000004698 lymphocyte Anatomy 0.000 description 1
- 210000000415 mammalian chromosome Anatomy 0.000 description 1
- 210000001161 mammalian embryo Anatomy 0.000 description 1
- 230000013011 mating Effects 0.000 description 1
- 230000035800 maturation Effects 0.000 description 1
- 230000010534 mechanism of action Effects 0.000 description 1
- 230000011987 methylation Effects 0.000 description 1
- 238000007069 methylation reaction Methods 0.000 description 1
- 230000000813 microbial effect Effects 0.000 description 1
- 244000005700 microbiome Species 0.000 description 1
- 238000000520 microinjection Methods 0.000 description 1
- 235000013336 milk Nutrition 0.000 description 1
- 239000008267 milk Substances 0.000 description 1
- 210000004080 milk Anatomy 0.000 description 1
- 210000003470 mitochondria Anatomy 0.000 description 1
- 201000011540 mitochondrial DNA depletion syndrome 4a Diseases 0.000 description 1
- 238000012544 monitoring process Methods 0.000 description 1
- 208000020460 mucolipidosis II alpha/beta Diseases 0.000 description 1
- 206010028093 mucopolysaccharidosis Diseases 0.000 description 1
- 201000006938 muscular dystrophy Diseases 0.000 description 1
- 231100000219 mutagenic Toxicity 0.000 description 1
- 230000003505 mutagenic effect Effects 0.000 description 1
- YOHYSYJDKVYCJI-UHFFFAOYSA-N n-[3-[[6-[3-(trifluoromethyl)anilino]pyrimidin-4-yl]amino]phenyl]cyclopropanecarboxamide Chemical compound FC(F)(F)C1=CC=CC(NC=2N=CN=C(NC=3C=C(NC(=O)C4CC4)C=CC=3)C=2)=C1 YOHYSYJDKVYCJI-UHFFFAOYSA-N 0.000 description 1
- 230000001537 neural effect Effects 0.000 description 1
- 230000000626 neurodegenerative effect Effects 0.000 description 1
- 230000007472 neurodevelopment Effects 0.000 description 1
- 230000007935 neutral effect Effects 0.000 description 1
- 235000015097 nutrients Nutrition 0.000 description 1
- 210000000056 organ Anatomy 0.000 description 1
- 210000003463 organelle Anatomy 0.000 description 1
- 229960003104 ornithine Drugs 0.000 description 1
- 230000011164 ossification Effects 0.000 description 1
- 238000012856 packing Methods 0.000 description 1
- 210000000496 pancreas Anatomy 0.000 description 1
- 102000045222 parkin Human genes 0.000 description 1
- 229940049954 penicillin Drugs 0.000 description 1
- 239000000816 peptidomimetic Substances 0.000 description 1
- 230000002093 peripheral effect Effects 0.000 description 1
- 230000026731 phosphorylation Effects 0.000 description 1
- 238000006366 phosphorylation reaction Methods 0.000 description 1
- 102000020233 phosphotransferase Human genes 0.000 description 1
- 238000013081 phylogenetic analysis Methods 0.000 description 1
- 239000000049 pigment Substances 0.000 description 1
- 238000003976 plant breeding Methods 0.000 description 1
- 230000008488 polyadenylation Effects 0.000 description 1
- 238000006116 polymerization reaction Methods 0.000 description 1
- 230000029279 positive regulation of transcription, DNA-dependent Effects 0.000 description 1
- 244000144977 poultry Species 0.000 description 1
- 239000002243 precursor Substances 0.000 description 1
- 230000002028 premature Effects 0.000 description 1
- 238000004393 prognosis Methods 0.000 description 1
- 201000001204 progressive myoclonus epilepsy Diseases 0.000 description 1
- 230000002035 prolonged effect Effects 0.000 description 1
- 230000001737 promoting effect Effects 0.000 description 1
- 230000000644 propagated effect Effects 0.000 description 1
- 230000001681 protective effect Effects 0.000 description 1
- 108060006633 protein kinase Proteins 0.000 description 1
- 230000006337 proteolytic cleavage Effects 0.000 description 1
- 239000000700 radioactive tracer Substances 0.000 description 1
- 239000011535 reaction buffer Substances 0.000 description 1
- 230000009467 reduction Effects 0.000 description 1
- 108091008146 restriction endonucleases Proteins 0.000 description 1
- 238000007894 restriction fragment length polymorphism technique Methods 0.000 description 1
- 238000012552 review Methods 0.000 description 1
- 239000002336 ribonucleotide Substances 0.000 description 1
- 125000002652 ribonucleotide group Chemical group 0.000 description 1
- 108020004418 ribosomal RNA Proteins 0.000 description 1
- 108091092562 ribozyme Proteins 0.000 description 1
- 238000007480 sanger sequencing Methods 0.000 description 1
- 238000012216 screening Methods 0.000 description 1
- 238000000926 separation method Methods 0.000 description 1
- 229940076279 serotonin Drugs 0.000 description 1
- 238000004904 shortening Methods 0.000 description 1
- SQVRNKJHWKZAKO-OQPLDHBCSA-N sialic acid Chemical compound CC(=O)N[C@@H]1[C@@H](O)C[C@@](O)(C(O)=O)OC1[C@H](O)[C@H](O)CO SQVRNKJHWKZAKO-OQPLDHBCSA-N 0.000 description 1
- 230000008054 signal transmission Effects 0.000 description 1
- 230000011664 signaling Effects 0.000 description 1
- 210000003491 skin Anatomy 0.000 description 1
- AWUCVROLDVIAJX-GSVOUGTGSA-N sn-glycerol 3-phosphate Chemical compound OC[C@@H](O)COP(O)(O)=O AWUCVROLDVIAJX-GSVOUGTGSA-N 0.000 description 1
- 229910000030 sodium bicarbonate Inorganic materials 0.000 description 1
- 235000017557 sodium bicarbonate Nutrition 0.000 description 1
- 229910000029 sodium carbonate Inorganic materials 0.000 description 1
- 208000002320 spinal muscular atrophy Diseases 0.000 description 1
- 238000007619 statistical method Methods 0.000 description 1
- 108020003113 steroid hormone receptors Proteins 0.000 description 1
- 102000005969 steroid hormone receptors Human genes 0.000 description 1
- 238000003860 storage Methods 0.000 description 1
- 229960005322 streptomycin Drugs 0.000 description 1
- 239000000758 substrate Substances 0.000 description 1
- 230000009897 systematic effect Effects 0.000 description 1
- 229940037128 systemic glucocorticoids Drugs 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
- 229940094937 thioredoxin Drugs 0.000 description 1
- 229960004072 thrombin Drugs 0.000 description 1
- 229940113082 thymine Drugs 0.000 description 1
- 230000036962 time dependent Effects 0.000 description 1
- 230000005026 transcription initiation Effects 0.000 description 1
- 230000002103 transcriptional effect Effects 0.000 description 1
- 230000009466 transformation Effects 0.000 description 1
- 230000010474 transient expression Effects 0.000 description 1
- 238000003146 transient transfection Methods 0.000 description 1
- 230000005945 translocation Effects 0.000 description 1
- 230000018405 transmission of nerve impulse Effects 0.000 description 1
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 description 1
- 230000010415 tropism Effects 0.000 description 1
- 230000005760 tumorsuppression Effects 0.000 description 1
- 239000012498 ultrapure water Substances 0.000 description 1
- 210000001364 upper extremity Anatomy 0.000 description 1
- 210000000689 upper leg Anatomy 0.000 description 1
- 235000015112 vegetable and seed oil Nutrition 0.000 description 1
- 239000008158 vegetable oil Substances 0.000 description 1
- 239000003981 vehicle Substances 0.000 description 1
- 208000037997 venous disease Diseases 0.000 description 1
- 210000002845 virion Anatomy 0.000 description 1
- 239000000277 virosome Substances 0.000 description 1
- 238000007794 visualization technique Methods 0.000 description 1
- 235000013343 vitamin Nutrition 0.000 description 1
- 239000011782 vitamin Substances 0.000 description 1
- 229940088594 vitamin Drugs 0.000 description 1
- 229930003231 vitamin Natural products 0.000 description 1
- 230000003313 weakening effect Effects 0.000 description 1
- 210000005253 yeast cell Anatomy 0.000 description 1
- 239000011701 zinc Substances 0.000 description 1
- 229910052725 zinc Inorganic materials 0.000 description 1
Images
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/01—Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/1034—Isolating an individual clone by screening libraries
- C12N15/1082—Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/52—Genes encoding for enzymes or proenzymes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6806—Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
-
- G—PHYSICS
- G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
- G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
- G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
-
- G—PHYSICS
- G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
- G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
- G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
- G16B20/20—Allele or variant detection, e.g. single nucleotide polymorphism [SNP] detection
-
- G—PHYSICS
- G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
- G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
- G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
- G16B20/30—Detection of binding sites or motifs
-
- G—PHYSICS
- G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
- G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
- G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
- G16B20/50—Mutagenesis
-
- G—PHYSICS
- G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
- G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
- G16B30/00—ICT specially adapted for sequence analysis involving nucleotides or amino acids
- G16B30/10—Sequence alignment; Homology search
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
-
- G—PHYSICS
- G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
- G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
- G16B30/00—ICT specially adapted for sequence analysis involving nucleotides or amino acids
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Analytical Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Bioinformatics & Computational Biology (AREA)
- Spectroscopy & Molecular Physics (AREA)
- Theoretical Computer Science (AREA)
- Medical Informatics (AREA)
- Evolutionary Biology (AREA)
- Medicinal Chemistry (AREA)
- Mycology (AREA)
- Virology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Cell Biology (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (25)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261736527P | 2012-12-12 | 2012-12-12 | |
| US61/736,527 | 2012-12-12 | ||
| US201361748427P | 2013-01-02 | 2013-01-02 | |
| US61/748,427 | 2013-01-02 | ||
| US201361758468P | 2013-01-30 | 2013-01-30 | |
| US61/758,468 | 2013-01-30 | ||
| US201361769046P | 2013-02-25 | 2013-02-25 | |
| US61/769,046 | 2013-02-25 | ||
| US201361802174P | 2013-03-15 | 2013-03-15 | |
| US201361791409P | 2013-03-15 | 2013-03-15 | |
| US61/802,174 | 2013-03-15 | ||
| US61/791,409 | 2013-03-15 | ||
| US201361806375P | 2013-03-28 | 2013-03-28 | |
| US61/806,375 | 2013-03-28 | ||
| US201361814263P | 2013-04-20 | 2013-04-20 | |
| US61/814,263 | 2013-04-20 | ||
| US201361819803P | 2013-05-06 | 2013-05-06 | |
| US61/819,803 | 2013-05-06 | ||
| US201361828130P | 2013-05-28 | 2013-05-28 | |
| US61/828,130 | 2013-05-28 | ||
| US201361836127P | 2013-06-17 | 2013-06-17 | |
| US201361835931P | 2013-06-17 | 2013-06-17 | |
| US61/835,931 | 2013-06-17 | ||
| US61/836,127 | 2013-06-17 | ||
| PCT/US2013/074819 WO2014093712A1 (en) | 2012-12-12 | 2013-12-12 | Engineering of systems, methods and optimized guide compositions for sequence manipulation |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2019127300A Division RU2796017C2 (ru) | 2012-12-12 | 2013-12-12 | Конструирование систем, способы и оптимизированные направляющие композиции для манипуляции с последовательностями |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| RU2015128098A3 RU2015128098A3 (enExample) | 2019-03-28 |
| RU2015128098A RU2015128098A (ru) | 2019-03-28 |
| RU2701850C2 true RU2701850C2 (ru) | 2019-10-01 |
Family
ID=49920627
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2015128098A RU2701850C2 (ru) | 2012-12-12 | 2013-12-12 | Конструирование систем, способы и оптимизированные направляющие композиции для манипуляции с последовательностями |
Country Status (16)
| Country | Link |
|---|---|
| US (6) | US20140242664A1 (enExample) |
| EP (3) | EP2771468B1 (enExample) |
| JP (11) | JP2016504026A (enExample) |
| KR (1) | KR20150105633A (enExample) |
| CN (3) | CN113528577B (enExample) |
| AU (5) | AU2013359123B2 (enExample) |
| CA (1) | CA2894701A1 (enExample) |
| DK (1) | DK2771468T3 (enExample) |
| ES (4) | ES2536353T3 (enExample) |
| HK (1) | HK1210221A1 (enExample) |
| IL (2) | IL239344B2 (enExample) |
| MX (1) | MX380562B (enExample) |
| PL (2) | PL2771468T3 (enExample) |
| PT (4) | PT2896697E (enExample) |
| RU (1) | RU2701850C2 (enExample) |
| WO (1) | WO2014093712A1 (enExample) |
Cited By (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10597679B2 (en) | 2013-09-06 | 2020-03-24 | President And Fellows Of Harvard College | Switchable Cas9 nucleases and uses thereof |
| US10682410B2 (en) | 2013-09-06 | 2020-06-16 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US10704062B2 (en) | 2014-07-30 | 2020-07-07 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| US10858639B2 (en) | 2013-09-06 | 2020-12-08 | President And Fellows Of Harvard College | CAS9 variants and uses thereof |
| US10947530B2 (en) | 2016-08-03 | 2021-03-16 | President And Fellows Of Harvard College | Adenosine nucleobase editors and uses thereof |
| US10954548B2 (en) | 2013-08-09 | 2021-03-23 | President And Fellows Of Harvard College | Nuclease profiling system |
| US11046948B2 (en) | 2013-08-22 | 2021-06-29 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| US11214780B2 (en) | 2015-10-23 | 2022-01-04 | President And Fellows Of Harvard College | Nucleobase editors and uses thereof |
| US11268082B2 (en) | 2017-03-23 | 2022-03-08 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable DNA binding proteins |
| US11306324B2 (en) | 2016-10-14 | 2022-04-19 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
| US11447770B1 (en) | 2019-03-19 | 2022-09-20 | The Broad Institute, Inc. | Methods and compositions for prime editing nucleotide sequences |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| US11542496B2 (en) | 2017-03-10 | 2023-01-03 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| US11732274B2 (en) | 2017-07-28 | 2023-08-22 | President And Fellows Of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE) |
| US11795443B2 (en) | 2017-10-16 | 2023-10-24 | The Broad Institute, Inc. | Uses of adenosine base editors |
| US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| US11912985B2 (en) | 2020-05-08 | 2024-02-27 | The Broad Institute, Inc. | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| US12006520B2 (en) | 2011-07-22 | 2024-06-11 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
Families Citing this family (718)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011140441A2 (en) | 2010-05-06 | 2011-11-10 | Children's Hospital Medical Center | Methods and systems for converting precursor cells into intestinal tissues through directed differentiation |
| EP2689019A2 (en) | 2011-03-23 | 2014-01-29 | Pioneer Hi-Bred International Inc. | Methods for producing a complex transgenic trait locus |
| EP3604555B1 (en) | 2011-10-14 | 2024-12-25 | President and Fellows of Harvard College | Sequencing by structure assembly |
| WO2014163886A1 (en) | 2013-03-12 | 2014-10-09 | President And Fellows Of Harvard College | Method of generating a three-dimensional nucleic acid containing matrix |
| ES2991004T3 (es) | 2011-12-22 | 2024-12-02 | Harvard College | Métodos para la detección de analitos |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| US9637739B2 (en) * | 2012-03-20 | 2017-05-02 | Vilnius University | RNA-directed DNA cleavage by the Cas9-crRNA complex |
| RU2645475C2 (ru) | 2012-04-25 | 2018-02-21 | Регенерон Фармасьютикалз, Инк. | Опосредованное нуклеазой нацеливание с большими нацеливающими векторами |
| WO2013163628A2 (en) | 2012-04-27 | 2013-10-31 | Duke University | Genetic correction of mutated genes |
| LT3401400T (lt) | 2012-05-25 | 2019-06-10 | The Regents Of The University Of California | Būdai ir kompozicijos, skirtos rnr molekulės nukreipiamai tikslinės dnr modifikacijai ir rnr molekulės nukreipiamam transkripcijos moduliavimui |
| US20150225734A1 (en) | 2012-06-19 | 2015-08-13 | Regents Of The University Of Minnesota | Gene targeting in plants using dna viruses |
| CA2875618C (en) | 2012-07-11 | 2021-04-27 | Sangamo Biosciences, Inc. | Methods and compositions for the treatment of lysosomal storage diseases |
| US10648001B2 (en) | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
| JP2015527889A (ja) * | 2012-07-25 | 2015-09-24 | ザ ブロード インスティテュート, インコーポレイテッド | 誘導可能なdna結合タンパク質およびゲノム撹乱ツール、ならびにそれらの適用 |
| JP6367196B2 (ja) | 2012-08-14 | 2018-08-01 | テンエックス・ジェノミクス・インコーポレイテッド | マイクロカプセル組成物および方法 |
| US9951386B2 (en) | 2014-06-26 | 2018-04-24 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
| US10752949B2 (en) | 2012-08-14 | 2020-08-25 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
| US11591637B2 (en) | 2012-08-14 | 2023-02-28 | 10X Genomics, Inc. | Compositions and methods for sample processing |
| US9701998B2 (en) | 2012-12-14 | 2017-07-11 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
| US10584381B2 (en) | 2012-08-14 | 2020-03-10 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
| US10273541B2 (en) | 2012-08-14 | 2019-04-30 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
| US10323279B2 (en) | 2012-08-14 | 2019-06-18 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
| PL4397760T3 (pl) | 2012-10-23 | 2026-03-30 | Toolgen Incorporated | Kompozycja do rozszczepiania docelowego dna zawierająca prowadzące rna specyficzne dla docelowego dna i kwas nukleinowy kodujący białko cas lub białko cas oraz jej zastosowanie |
| AU2013352156B2 (en) | 2012-11-27 | 2018-12-06 | Children's Medical Center Corporation | Targeting BCL11A distal regulatory elements for fetal hemoglobin reinduction |
| EP3135765A1 (en) | 2012-12-06 | 2017-03-01 | Sigma-Aldrich Co. LLC | Crispr-based genome modification and regulation |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| EP2896697B1 (en) | 2012-12-12 | 2015-09-02 | The Broad Institute, Inc. | Engineering of systems, methods and optimized guide compositions for sequence manipulation |
| ES2576126T3 (es) | 2012-12-12 | 2016-07-05 | The Broad Institute, Inc. | Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias |
| JP2016504026A (ja) * | 2012-12-12 | 2016-02-12 | ザ・ブロード・インスティテュート・インコーポレイテッド | 配列操作のための系、方法および最適化ガイド組成物のエンジニアリング |
| IL239317B (en) | 2012-12-12 | 2022-07-01 | Broad Inst Inc | Providing, engineering and optimizing systems, methods and compositions for sequence manipulation and therapeutic applications |
| EP3434776A1 (en) | 2012-12-12 | 2019-01-30 | The Broad Institute, Inc. | Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| CN114634950A (zh) | 2012-12-12 | 2022-06-17 | 布罗德研究所有限公司 | 用于序列操纵的crispr-cas组分系统、方法以及组合物 |
| EP2931898B1 (en) | 2012-12-12 | 2016-03-09 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| WO2014093701A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| EP3567116A1 (en) | 2012-12-14 | 2019-11-13 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
| US10533221B2 (en) | 2012-12-14 | 2020-01-14 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
| SG10201912991WA (en) * | 2012-12-17 | 2020-03-30 | Harvard College | Rna-guided human genome engineering |
| EP3919505B1 (en) | 2013-01-16 | 2023-08-30 | Emory University | Uses of cas9-nucleic acid complexes |
| KR102190198B1 (ko) | 2013-02-08 | 2020-12-14 | 10엑스 제노믹스, 인크. | 폴리뉴클레오티드 바코드 생성 |
| SG11201507378UA (en) | 2013-03-14 | 2015-10-29 | Caribou Biosciences Inc | Compositions and methods of nucleic acid-targeting nucleic acids |
| US10760064B2 (en) | 2013-03-15 | 2020-09-01 | The General Hospital Corporation | RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci |
| WO2014204578A1 (en) | 2013-06-21 | 2014-12-24 | The General Hospital Corporation | Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing |
| US9234213B2 (en) | 2013-03-15 | 2016-01-12 | System Biosciences, Llc | Compositions and methods directed to CRISPR/Cas genomic engineering systems |
| US10378027B2 (en) | 2013-03-15 | 2019-08-13 | The General Hospital Corporation | RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci |
| US20140273235A1 (en) * | 2013-03-15 | 2014-09-18 | Regents Of The University Of Minnesota | ENGINEERING PLANT GENOMES USING CRISPR/Cas SYSTEMS |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| JP2016522679A (ja) | 2013-04-04 | 2016-08-04 | プレジデント アンド フェローズ オブ ハーバード カレッジ | CRISPR/Cas系を用いたゲノム編集の治療的使用 |
| DK2986729T3 (en) | 2013-04-16 | 2018-10-29 | Regeneron Pharma | TARGETED MODIFICATION OF ROOT THROUGH |
| SG10201710030QA (en) | 2013-06-04 | 2018-01-30 | Harvard College | Rna-guided transcriptional regulation |
| US20140356956A1 (en) | 2013-06-04 | 2014-12-04 | President And Fellows Of Harvard College | RNA-Guided Transcriptional Regulation |
| JP6665088B2 (ja) | 2013-06-17 | 2020-03-13 | ザ・ブロード・インスティテュート・インコーポレイテッド | 配列操作のための最適化されたCRISPR−Cas二重ニッカーゼ系、方法および組成物 |
| CN105793425B (zh) | 2013-06-17 | 2021-10-26 | 布罗德研究所有限公司 | 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用 |
| EP3725885A1 (en) | 2013-06-17 | 2020-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
| WO2014204724A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| MX2015017312A (es) | 2013-06-17 | 2017-04-10 | Broad Inst Inc | Suministro y uso de composiciones, vectores y sistemas crispr-cas para la modificación dirigida y terapia hepáticas. |
| EP3738545B1 (en) | 2013-06-19 | 2023-11-29 | Kolibree | Toothbrush system with sensors for a dental hygiene monitoring system |
| JP2016528890A (ja) | 2013-07-09 | 2016-09-23 | プレジデント アンド フェローズ オブ ハーバード カレッジ | CRISPR/Cas系を用いるゲノム編集の治療用の使用 |
| HUE056760T2 (hu) * | 2013-07-11 | 2022-03-28 | Modernatx Inc | A CRISPR-hez kapcsolódó fehérjéket és a szintetikus SGRNS-ket kódoló szintetikus polinukleotidokat tartalmazó készítmények és felhasználási módjaik |
| CA3221516A1 (en) | 2013-08-22 | 2015-02-26 | E. I. Du Pont De Nemours And Company | Plant genome modification using guide rna/cas endonuclease systems and methods of use |
| ES2844174T3 (es) | 2013-09-18 | 2021-07-21 | Kymab Ltd | Métodos, células y organismos |
| WO2015054507A1 (en) | 2013-10-10 | 2015-04-16 | Pronutria, Inc. | Nutritive polypeptide production systems, and methods of manufacture and use thereof |
| WO2015065964A1 (en) | 2013-10-28 | 2015-05-07 | The Broad Institute Inc. | Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof |
| CN106459995B (zh) | 2013-11-07 | 2020-02-21 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
| WO2015088643A1 (en) | 2013-12-11 | 2015-06-18 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for the targeted modification of a genome |
| WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
| BR112016013547A2 (pt) | 2013-12-12 | 2017-10-03 | Broad Inst Inc | Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos |
| RU2016128077A (ru) | 2013-12-12 | 2018-12-06 | Те Брод Инститьют Инк. | Доставка, применение и применения в терапии систем и композиций crispr-cas для лечения обусловленных hbv и вирусных заболеваний и нарушений |
| WO2015089427A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes |
| WO2015089486A2 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems |
| CN111206032B (zh) | 2013-12-12 | 2024-07-19 | 布罗德研究所有限公司 | 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用 |
| BR112016013213A2 (pt) | 2013-12-12 | 2017-12-05 | Massachusetts Inst Technology | administração, uso e aplicações terapêuticas dos sistemas crispr-cas e composições para visar distúrbios e doenças usando componentes de administração de partículas |
| WO2015089473A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation |
| EP4063503A1 (en) | 2014-02-11 | 2022-09-28 | The Regents of the University of Colorado, a body corporate | Crispr enabled multiplexed genome engineering |
| EP3107999A4 (en) | 2014-02-18 | 2017-10-04 | Duke University | Compositions for the inactivation of virus replication and methods of making and using the same |
| EP3971283A1 (en) | 2014-02-27 | 2022-03-23 | Monsanto Technology LLC | Compositions and methods for site directed genomic modification |
| WO2015130968A2 (en) | 2014-02-27 | 2015-09-03 | The Broad Institute Inc. | T cell balance gene expression, compositions of matters and methods of use thereof |
| EP3115457B1 (en) | 2014-03-05 | 2019-10-02 | National University Corporation Kobe University | Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same |
| US11028388B2 (en) | 2014-03-05 | 2021-06-08 | Editas Medicine, Inc. | CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa |
| US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| CA2948728A1 (en) | 2014-03-10 | 2015-09-17 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| JP6815986B2 (ja) | 2014-03-26 | 2021-01-20 | ユニバーシティ オブ メリーランド, カレッジ パーク | 大型家畜の接合体における標的化ゲノム編集 |
| WO2015148863A2 (en) | 2014-03-26 | 2015-10-01 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
| EP3498845B1 (en) * | 2014-04-01 | 2022-06-22 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating herpes simplex virus type 1 (hsv-1) |
| EP3126495A1 (en) | 2014-04-02 | 2017-02-08 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| CA2943624A1 (en) | 2014-04-10 | 2015-10-15 | 10X Genomics, Inc. | Fluidic devices, systems, and methods for encapsulating and partitioning reagents, and applications of same |
| ES2744186T3 (es) | 2014-04-25 | 2020-02-24 | Childrens Medical Center | Composiciones y métodos para tratar las hemoglobinopatías |
| EP3712254A1 (en) | 2014-05-28 | 2020-09-23 | Children's Hospital Medical Center | Methods and systems for converting precursor cells into gastric tissues through directed differentiation |
| KR20170005494A (ko) | 2014-05-30 | 2017-01-13 | 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 | 잠복 바이러스 감염에 대한 치료제를 전달하는 조성물 및 방법 |
| EP3154694B1 (en) | 2014-06-13 | 2025-01-29 | Children's Medical Center Corporation | Products and methods to isolate mitochondria |
| EP3919621A1 (en) | 2014-06-23 | 2021-12-08 | The General Hospital Corporation | Genomewide unbiased identification of dsbs evaluated by sequencing (guide-seq) |
| WO2015200555A2 (en) * | 2014-06-25 | 2015-12-30 | Caribou Biosciences, Inc. | Rna modification to engineer cas9 activity |
| WO2015200869A1 (en) | 2014-06-26 | 2015-12-30 | 10X Genomics, Inc. | Analysis of nucleic acid sequences |
| US12312640B2 (en) | 2014-06-26 | 2025-05-27 | 10X Genomics, Inc. | Analysis of nucleic acid sequences |
| EP4574974A3 (en) | 2014-06-26 | 2025-10-08 | 10x Genomics, Inc. | Methods of analyzing nucleic acids from individual cells or cell populations |
| CN106687594A (zh) | 2014-07-11 | 2017-05-17 | 纳幕尔杜邦公司 | 用于产生对草甘膦除草剂具有抗性的植物的组合物和方法 |
| US10179932B2 (en) | 2014-07-11 | 2019-01-15 | President And Fellows Of Harvard College | Methods for high-throughput labelling and detection of biological features in situ using microscopy |
| CN104195177B (zh) * | 2014-08-05 | 2017-06-09 | 南京大学 | 一种显著提高鱼类基因组编辑效率的方法 |
| DK3180426T3 (da) | 2014-08-17 | 2020-03-30 | Broad Inst Inc | Genomredigering ved anvendelse af cas9-nickaser |
| WO2016028887A1 (en) | 2014-08-19 | 2016-02-25 | Pacific Biosciences Of California, Inc. | Compositions and methods for enrichment of nucleic acids |
| CA2959070C (en) | 2014-08-27 | 2020-11-10 | Caribou Biosciences, Inc. | Methods for increasing cas9-mediated engineering efficiency |
| US10570418B2 (en) | 2014-09-02 | 2020-02-25 | The Regents Of The University Of California | Methods and compositions for RNA-directed target DNA modification |
| BR112017003757A2 (pt) | 2014-09-12 | 2017-12-26 | Du Pont | ?plantas de milho, partes de planta de milho ou sementes de milho? |
| WO2016049163A2 (en) | 2014-09-24 | 2016-03-31 | The Broad Institute Inc. | Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder |
| WO2016049024A2 (en) | 2014-09-24 | 2016-03-31 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo |
| WO2016049251A1 (en) | 2014-09-24 | 2016-03-31 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes |
| US10040048B1 (en) | 2014-09-25 | 2018-08-07 | Synthego Corporation | Automated modular system and method for production of biopolymers |
| WO2016049258A2 (en) | 2014-09-25 | 2016-03-31 | The Broad Institute Inc. | Functional screening with optimized functional crispr-cas systems |
| AU2015330699B2 (en) | 2014-10-10 | 2021-12-02 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| GB201418965D0 (enExample) | 2014-10-24 | 2014-12-10 | Ospedale San Raffaele And Fond Telethon | |
| WO2016069591A2 (en) | 2014-10-27 | 2016-05-06 | The Broad Institute Inc. | Compositions, methods and use of synthetic lethal screening |
| US9975122B2 (en) | 2014-11-05 | 2018-05-22 | 10X Genomics, Inc. | Instrument systems for integrated sample processing |
| EP3215623A4 (en) | 2014-11-06 | 2018-09-26 | President and Fellows of Harvard College | Cells lacking b2m surface expression and methods for allogeneic administration of such cells |
| WO2016073990A2 (en) | 2014-11-07 | 2016-05-12 | Editas Medicine, Inc. | Methods for improving crispr/cas-mediated genome-editing |
| EP3218508A4 (en) * | 2014-11-10 | 2018-04-18 | Modernatx, Inc. | Multiparametric nucleic acid optimization |
| KR101833433B1 (ko) * | 2014-11-25 | 2018-02-28 | 한국생명공학연구원 | 돼지 t 세포 및 b 세포 면역결핍 세포주 생산 및 그의 제조 방법 |
| US11168369B2 (en) | 2014-11-25 | 2021-11-09 | The Brigham And Women's Hospital, Inc. | Method of identifying and treating a person having a predisposition to or afflicted with a cardiometabolic disease |
| EP3224362B1 (en) | 2014-11-26 | 2024-09-25 | The Regents of The University of California | Therapeutic compositions comprising transcription factors and methods of making and using the same |
| GB201421096D0 (en) | 2014-11-27 | 2015-01-14 | Imp Innovations Ltd | Genome editing methods |
| WO2016089866A1 (en) | 2014-12-01 | 2016-06-09 | President And Fellows Of Harvard College | Rna-guided systems for in vivo gene editing |
| WO2016089433A1 (en) * | 2014-12-03 | 2016-06-09 | Agilent Technologies, Inc. | Guide rna with chemical modifications |
| EP3229586B1 (en) | 2014-12-10 | 2025-07-30 | Regents of the University of Minnesota | Genetically modified cells, tissues, and organs for treating disease |
| WO2016094874A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Escorted and functionalized guides for crispr-cas systems |
| EP3230451B1 (en) | 2014-12-12 | 2021-04-07 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
| WO2016094880A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs) |
| WO2016094872A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Dead guides for crispr transcription factors |
| WO2016100951A2 (en) * | 2014-12-18 | 2016-06-23 | Integrated Dna Technologies, Inc. | Crispr-based compositions and methods of use |
| WO2016100974A1 (en) | 2014-12-19 | 2016-06-23 | The Broad Institute Inc. | Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing |
| WO2016106236A1 (en) | 2014-12-23 | 2016-06-30 | The Broad Institute Inc. | Rna-targeting system |
| CA2970370A1 (en) | 2014-12-24 | 2016-06-30 | Massachusetts Institute Of Technology | Crispr having or associated with destabilization domains |
| WO2016108926A1 (en) | 2014-12-30 | 2016-07-07 | The Broad Institute Inc. | Crispr mediated in vivo modeling and genetic screening of tumor growth and metastasis |
| EP3245232B1 (en) | 2015-01-12 | 2021-04-21 | The Regents of The University of California | Heterodimeric cas9 and methods of use thereof |
| CN107427808B (zh) | 2015-01-12 | 2020-10-23 | 10X基因组学有限公司 | 用于制备核酸测序文库的方法和系统以及用其制备的文库 |
| US11180792B2 (en) | 2015-01-28 | 2021-11-23 | The Regents Of The University Of California | Methods and compositions for labeling a single-stranded target nucleic acid |
| SI3250691T1 (sl) | 2015-01-28 | 2023-10-30 | Caribou Biosciences, Inc. | Hibridni dna/rna-polinukleotidi crispr in postopki za uporabo |
| JP6929791B2 (ja) | 2015-02-09 | 2021-09-01 | デューク ユニバーシティ | エピゲノム編集のための組成物および方法 |
| EP3262173A2 (en) | 2015-02-23 | 2018-01-03 | Crispr Therapeutics AG | Materials and methods for treatment of human genetic diseases including hemoglobinopathies |
| AU2016225179C1 (en) | 2015-02-23 | 2022-11-03 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| EP3262407B1 (en) | 2015-02-24 | 2023-08-30 | 10X Genomics, Inc. | Partition processing methods and systems |
| WO2016138488A2 (en) | 2015-02-26 | 2016-09-01 | The Broad Institute Inc. | T cell balance gene expression, compositions of matters and methods of use thereof |
| JP6871169B2 (ja) | 2015-03-02 | 2021-05-12 | シナイ ヘルス システム | 相同組換え因子 |
| JP6817215B2 (ja) | 2015-03-03 | 2021-01-20 | ザ ジェネラル ホスピタル コーポレイション | 変更PAM特異性を有する遺伝子操作CRISPR−Cas9ヌクレアーゼ |
| EP4019635A1 (en) * | 2015-03-25 | 2022-06-29 | Editas Medicine, Inc. | Crispr/cas-related methods, compositions and components |
| EP3553178A1 (en) | 2015-03-27 | 2019-10-16 | E. I. du Pont de Nemours and Company | Soybean u6 small nuclear rna gene promoters and their use in constitutive expression of small rna genes in plants |
| KR102888521B1 (ko) * | 2015-04-06 | 2025-11-19 | 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 | Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna |
| GB201506509D0 (en) | 2015-04-16 | 2015-06-03 | Univ Wageningen | Nuclease-mediated genome editing |
| EP3286571B1 (en) | 2015-04-24 | 2021-08-18 | Editas Medicine, Inc. | Evaluation of cas9 molecule/guide rna molecule complexes |
| ES2835861T5 (en) | 2015-05-08 | 2025-02-18 | Childrens Medical Ct Corp | Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction |
| CA2988854A1 (en) | 2015-05-08 | 2016-11-17 | President And Fellows Of Harvard College | Universal donor stem cells and related methods |
| WO2016182893A1 (en) | 2015-05-08 | 2016-11-17 | Teh Broad Institute Inc. | Functional genomics using crispr-cas systems for saturating mutagenesis of non-coding elements, compositions, methods, libraries and applications thereof |
| EP3294896A1 (en) | 2015-05-11 | 2018-03-21 | Editas Medicine, Inc. | Optimized crispr/cas9 systems and methods for gene editing in stem cells |
| US10117911B2 (en) | 2015-05-29 | 2018-11-06 | Agenovir Corporation | Compositions and methods to treat herpes simplex virus infections |
| EP3919622A1 (en) | 2015-06-01 | 2021-12-08 | Temple University - Of The Commonwealth System of Higher Education | Methods and compositions for rna-guided treatment of hiv infection |
| EP3303585A4 (en) | 2015-06-03 | 2018-10-31 | Board of Regents of the University of Nebraska | Dna editing using single-stranded dna |
| EP3303634B1 (en) | 2015-06-03 | 2023-08-30 | The Regents of The University of California | Cas9 variants and methods of use thereof |
| EP3302525A2 (en) | 2015-06-05 | 2018-04-11 | Novartis AG | Methods and compositions for diagnosing, treating, and monitoring treatment of shank3 deficiency associated disorders |
| KR102796744B1 (ko) | 2015-06-09 | 2025-04-15 | 에디타스 메디신, 인코포레이티드 | 이식의 개선을 위한 crispr/cas-관련 방법 및 조성물 |
| US20160362667A1 (en) * | 2015-06-10 | 2016-12-15 | Caribou Biosciences, Inc. | CRISPR-Cas Compositions and Methods |
| JP2018518182A (ja) | 2015-06-17 | 2018-07-12 | ザ ユーエービー リサーチ ファンデーション | ゲノム編集のためのcrispr/cas9複合体 |
| CA2989858A1 (en) | 2015-06-17 | 2016-12-22 | The Uab Research Foundation | Crispr/cas9 complex for introducing a functional polypeptide into cells of blood cell lineage |
| WO2016205728A1 (en) | 2015-06-17 | 2016-12-22 | Massachusetts Institute Of Technology | Crispr mediated recording of cellular events |
| EP3436575A1 (en) | 2015-06-18 | 2019-02-06 | The Broad Institute Inc. | Novel crispr enzymes and systems |
| AU2016279077A1 (en) | 2015-06-18 | 2019-03-28 | Omar O. Abudayyeh | Novel CRISPR enzymes and systems |
| IL293323B2 (en) | 2015-06-18 | 2024-01-01 | Massachusetts Inst Technology | Crispr enzyme mutations reducing off-target effects |
| US10648020B2 (en) | 2015-06-18 | 2020-05-12 | The Broad Institute, Inc. | CRISPR enzymes and systems |
| WO2016205759A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation |
| US9790490B2 (en) | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| WO2016205745A2 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Cell sorting |
| CA2990699A1 (en) | 2015-06-29 | 2017-01-05 | Ionis Pharmaceuticals, Inc. | Modified crispr rna and modified single crispr rna and uses thereof |
| WO2017004279A2 (en) * | 2015-06-29 | 2017-01-05 | Massachusetts Institute Of Technology | Compositions comprising nucleic acids and methods of using the same |
| GB2557123B (en) | 2015-07-31 | 2021-11-03 | Univ Minnesota | Modified cells and methods of therapy |
| US9580727B1 (en) | 2015-08-07 | 2017-02-28 | Caribou Biosciences, Inc. | Compositions and methods of engineered CRISPR-Cas9 systems using split-nexus Cas9-associated polynucleotides |
| WO2017027810A2 (en) | 2015-08-12 | 2017-02-16 | The General Hospital Corporation | Compositions and methods that promote hypoxia or the hypoxia response for treatment and prevention of mitochondrial dysfunction and oxidative stress disorders |
| US11214800B2 (en) | 2015-08-18 | 2022-01-04 | The Broad Institute, Inc. | Methods and compositions for altering function and structure of chromatin loops and/or domains |
| WO2017035416A2 (en) * | 2015-08-25 | 2017-03-02 | Duke University | Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases |
| US9512446B1 (en) | 2015-08-28 | 2016-12-06 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| US9926546B2 (en) | 2015-08-28 | 2018-03-27 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| AU2016316845B2 (en) | 2015-08-28 | 2022-03-10 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| CN107922949A (zh) * | 2015-08-31 | 2018-04-17 | 安捷伦科技有限公司 | 用于通过同源重组的基于crispr/cas的基因组编辑的化合物和方法 |
| DK3348638T3 (da) | 2015-09-09 | 2023-02-13 | Univ Kobe Nat Univ Corp | Fremgangsmåde til at konvertere genomsekvens fra gram-positiv bakterie ved specifikt at konvertere nukleinsyrebase i tilsigtet dna-sekvens, og molekylkompleks anvendt dertil |
| CN108350453A (zh) | 2015-09-11 | 2018-07-31 | 通用医疗公司 | 核酸酶dsb的完全查询和测序(find-seq) |
| SI3350333T1 (sl) | 2015-09-17 | 2022-04-29 | ModernaTX,Inc. | Polinukleotidi, ki zajemajo stabilizacijsko repno regijo |
| WO2017048969A1 (en) * | 2015-09-17 | 2017-03-23 | The Regents Of The University Of California | Variant cas9 polypeptides comprising internal insertions |
| EP3353298B1 (en) * | 2015-09-21 | 2023-09-13 | Arcturus Therapeutics, Inc. | Allele selective gene editing and uses thereof |
| EP3353296B1 (en) | 2015-09-24 | 2020-11-04 | Editas Medicine, Inc. | Use of exonucleases to improve crispr/cas-mediated genome editing |
| JP2018529353A (ja) | 2015-09-30 | 2018-10-11 | ザ ジェネラル ホスピタル コーポレイション | 配列決定法による切断事象の包括的生体外報告(CIRCLE−seq) |
| US12241053B2 (en) | 2015-10-09 | 2025-03-04 | The Brigham And Women's Hospital, Inc. | Modulation of novel immune checkpoint targets |
| EP4089175A1 (en) | 2015-10-13 | 2022-11-16 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| CN109153980B (zh) | 2015-10-22 | 2023-04-14 | 布罗德研究所有限公司 | Vi-b型crispr酶和系统 |
| EP3350327B1 (en) | 2015-10-23 | 2018-09-26 | Caribou Biosciences, Inc. | Engineered crispr class 2 cross-type nucleic-acid targeting nucleic acids |
| EP3368687B1 (en) | 2015-10-27 | 2021-09-29 | The Broad Institute, Inc. | Compositions and methods for targeting cancer-specific sequence variations |
| WO2017075451A1 (en) | 2015-10-28 | 2017-05-04 | The Broad Institute Inc. | Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1 |
| WO2017075294A1 (en) | 2015-10-28 | 2017-05-04 | The Board Institute Inc. | Assays for massively combinatorial perturbation profiling and cellular circuit reconstruction |
| WO2017075465A1 (en) | 2015-10-28 | 2017-05-04 | The Broad Institute Inc. | Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3 |
| EP3368689B1 (en) | 2015-10-28 | 2020-06-17 | The Broad Institute, Inc. | Composition for modulating immune responses by use of immune cell gene signature |
| WO2017075265A1 (en) | 2015-10-28 | 2017-05-04 | The Broad Institute, Inc. | Multiplex analysis of single cell constituents |
| WO2017079406A1 (en) | 2015-11-03 | 2017-05-11 | President And Fellows Of Harvard College | Method and apparatus for volumetric imaging of a three-dimensional nucleic acid containing matrix |
| US12043843B2 (en) | 2015-11-04 | 2024-07-23 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| WO2017083852A1 (en) | 2015-11-13 | 2017-05-18 | MOORE, Tara | Methods for the treatment of corneal dystrophies |
| EP3377042A4 (en) | 2015-11-16 | 2019-05-29 | The Research Institute at Nationwide Children's Hospital | MATERIALS AND METHOD FOR THE TREATMENT OF TITIN-BASED MYOPATHIES AND OTHER TITINOPATHIES |
| WO2017087910A1 (en) * | 2015-11-19 | 2017-05-26 | 10X Genomics, Inc. | Transformable tagging compositions, methods, and processes incorporating same |
| CA3005878A1 (en) | 2015-11-19 | 2017-05-26 | The Brigham And Women's Hospital, Inc. | Lymphocyte antigen cd5-like (cd5l)-interleukin 12b (p40) heterodimers in immunity |
| US11371094B2 (en) | 2015-11-19 | 2022-06-28 | 10X Genomics, Inc. | Systems and methods for nucleic acid processing using degenerate nucleotides |
| WO2017090761A1 (ja) * | 2015-11-27 | 2017-06-01 | 国立大学法人神戸大学 | 標的化したdna配列の核酸塩基を特異的に変換する、単子葉植物のゲノム配列の変換方法、及びそれに用いる分子複合体 |
| WO2017095967A2 (en) | 2015-11-30 | 2017-06-08 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| EP3383411B1 (en) | 2015-11-30 | 2025-07-09 | Sana Biotechnology, Inc. | Sub-mitochondrial particles for use in treating metabolic conditions |
| SG10202108763UA (en) | 2015-12-04 | 2021-09-29 | 10X Genomics Inc | Methods and compositions for nucleic acid analysis |
| DK3371310T3 (da) * | 2015-12-04 | 2019-08-05 | Caribou Biosciences Inc | Nucleinsyrekonstruerede targetnucleinsyrer |
| US9988624B2 (en) | 2015-12-07 | 2018-06-05 | Zymergen Inc. | Microbial strain improvement by a HTP genomic engineering platform |
| US11208649B2 (en) | 2015-12-07 | 2021-12-28 | Zymergen Inc. | HTP genomic engineering platform |
| CA3007635A1 (en) | 2015-12-07 | 2017-06-15 | Zymergen Inc. | Promoters from corynebacterium glutamicum |
| US12110490B2 (en) | 2015-12-18 | 2024-10-08 | The Broad Institute, Inc. | CRISPR enzymes and systems |
| WO2017106767A1 (en) * | 2015-12-18 | 2017-06-22 | The Scripps Research Institute | Production of unnatural nucleotides using a crispr/cas9 system |
| WO2017106569A1 (en) * | 2015-12-18 | 2017-06-22 | The Regents Of The University Of California | Modified site-directed modifying polypeptides and methods of use thereof |
| BR112018013074A2 (en) | 2015-12-30 | 2018-12-11 | Novartis Ag | immune effector cell therapies with enhanced efficacy |
| MX2018008345A (es) | 2016-01-11 | 2018-12-06 | Univ Leland Stanford Junior | Proteínas quiméricas y métodos de inmunoterapia. |
| IL304088A (en) | 2016-01-11 | 2023-08-01 | Univ Leland Stanford Junior | Systems containing chimeric proteins and their uses for controlling gene expression |
| ES3057779T3 (en) | 2016-01-15 | 2026-03-04 | Childrens Medical Ct Corp | Therapeutic use of mitochondria and combined mitochondrial agents |
| US11845933B2 (en) * | 2016-02-03 | 2023-12-19 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide RNA and its applications |
| WO2017139309A1 (en) | 2016-02-12 | 2017-08-17 | Ceres, Inc. | Methods and materials for high throughput testing of mutagenized allele combinations |
| EP3417061B1 (en) | 2016-02-18 | 2022-10-26 | The Regents of the University of California | Methods and compositions for gene editing in stem cells |
| EP3420102B1 (en) | 2016-02-22 | 2024-04-03 | Massachusetts Institute of Technology | Methods for identifying and modulating immune phenotypes |
| CA3015665C (en) * | 2016-02-26 | 2020-09-22 | Lanzatech New Zealand Limited | Crispr/cas systems for c1-fixing bacteria |
| WO2017152023A1 (en) | 2016-03-04 | 2017-09-08 | Indoor Biotechnologies Inc. | Fel d 1 knockouts and associated compositions and methods based on crispr-cas9 genomic editing |
| WO2017161043A1 (en) | 2016-03-16 | 2017-09-21 | The J. David Gladstone Institutes | Methods and compositions for treating obesity and/or diabetes and for identifying candidate treatment agents |
| EP3219799A1 (en) | 2016-03-17 | 2017-09-20 | IMBA-Institut für Molekulare Biotechnologie GmbH | Conditional crispr sgrna expression |
| US11427861B2 (en) | 2016-03-17 | 2022-08-30 | Massachusetts Institute Of Technology | Methods for identifying and modulating co-occurant cellular phenotypes |
| US12012436B2 (en) | 2016-03-23 | 2024-06-18 | The Regents Of The University Of California | Methods of treating mitochondrial disorders |
| US12011488B2 (en) | 2016-03-23 | 2024-06-18 | The Regents Of The University Of California | Methods of treating mitochondrial disorders |
| WO2017165859A1 (en) * | 2016-03-24 | 2017-09-28 | Research Institute At Nationwide Children's Hospital | Modified viral capsid proteins |
| US11597924B2 (en) | 2016-03-25 | 2023-03-07 | Editas Medicine, Inc. | Genome editing systems comprising repair-modulating enzyme molecules and methods of their use |
| WO2017165862A1 (en) | 2016-03-25 | 2017-09-28 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| EP3443081A4 (en) | 2016-04-13 | 2019-10-30 | Duke University | CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE |
| WO2017180694A1 (en) | 2016-04-13 | 2017-10-19 | Editas Medicine, Inc. | Cas9 fusion molecules gene editing systems, and methods of use thereof |
| US20170298348A1 (en) * | 2016-04-14 | 2017-10-19 | The Board Of Trustees Of The Leland Stanford Junior University | Genome editing of human neural stem cells using nucleases |
| SG11201810179RA (en) * | 2016-04-19 | 2018-12-28 | Broad Inst Inc | Novel crispr enzymes and systems |
| AU2017253107B2 (en) | 2016-04-19 | 2023-07-20 | Massachusetts Institute Of Technology | CPF1 complexes with reduced indel activity |
| AU2017257624B2 (en) | 2016-04-25 | 2023-05-25 | President And Fellows Of Harvard College | Hybridization chain reaction methods for in situ molecular detection |
| WO2017197343A2 (en) | 2016-05-12 | 2017-11-16 | 10X Genomics, Inc. | Microfluidic on-chip filters |
| WO2017197338A1 (en) | 2016-05-13 | 2017-11-16 | 10X Genomics, Inc. | Microfluidic systems and methods of use |
| US10767175B2 (en) * | 2016-06-08 | 2020-09-08 | Agilent Technologies, Inc. | High specificity genome editing using chemically modified guide RNAs |
| US11788083B2 (en) * | 2016-06-17 | 2023-10-17 | The Broad Institute, Inc. | Type VI CRISPR orthologs and systems |
| ES2915562T3 (es) | 2016-06-24 | 2022-06-23 | Univ Colorado Regents | Métodos para generar bibliotecas combinatorias con código de barras |
| US12385034B2 (en) | 2016-06-24 | 2025-08-12 | Modernatx, Inc. | Methods and apparatus for filtration |
| WO2018005445A1 (en) | 2016-06-27 | 2018-01-04 | The Broad Institute, Inc. | Compositions and methods for detecting and treating diabetes |
| WO2018005589A1 (en) | 2016-06-28 | 2018-01-04 | Cellectis | Altering expression of gene products in plants through targeted insertion of nucleic acid sequences |
| US12595478B2 (en) | 2016-06-29 | 2026-04-07 | The Broad Institute, Inc. | Crispr-Cas systems having destabilization domain |
| WO2018005793A1 (en) | 2016-06-30 | 2018-01-04 | Zymergen Inc. | Methods for generating a glucose permease library and uses thereof |
| JP2019519242A (ja) | 2016-06-30 | 2019-07-11 | ザイマージェン インコーポレイテッド | 細菌ヘモグロビンライブラリーを生成するための方法およびその使用 |
| EP3484870B1 (en) | 2016-07-13 | 2022-11-16 | Vertex Pharmaceuticals Incorporated | Methods, compositions and kits for increasing genome editing efficiency |
| EP4275747A3 (en) | 2016-07-19 | 2024-01-24 | Duke University | Therapeutic applications of cpf1-based genome editing |
| CA3032822A1 (en) | 2016-08-02 | 2018-02-08 | Editas Medicine, Inc. | Compositions and methods for treating cep290 associated disease |
| US11078481B1 (en) | 2016-08-03 | 2021-08-03 | KSQ Therapeutics, Inc. | Methods for screening for cancer targets |
| JP7215808B2 (ja) | 2016-08-12 | 2023-01-31 | ツールゲン インコーポレイテッド | 操作された免疫調節エレメントおよび変更された免疫 |
| WO2018035364A1 (en) | 2016-08-17 | 2018-02-22 | The Broad Institute Inc. | Product and methods useful for modulating and evaluating immune responses |
| WO2018035387A1 (en) | 2016-08-17 | 2018-02-22 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
| US11810649B2 (en) | 2016-08-17 | 2023-11-07 | The Broad Institute, Inc. | Methods for identifying novel gene editing elements |
| WO2018035388A1 (en) | 2016-08-17 | 2018-02-22 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
| US12431216B2 (en) | 2016-08-17 | 2025-09-30 | Broad Institute, Inc. | Methods for identifying class 2 crispr-cas systems |
| EP3500677A4 (en) | 2016-08-20 | 2020-04-01 | Avellino Lab USA, Inc. | Single guide rna, crispr/cas9 systems, and methods of use thereof |
| WO2020225754A1 (en) | 2019-05-06 | 2020-11-12 | Mcmullen Tara | Crispr gene editing for autosomal dominant diseases |
| GB2569252A (en) | 2016-08-31 | 2019-06-12 | Harvard College | Methods of combining the detection of biomolecules into a single assay using fluorescent in situ sequencing |
| US11078483B1 (en) | 2016-09-02 | 2021-08-03 | KSQ Therapeutics, Inc. | Methods for measuring and improving CRISPR reagent function |
| JP7215994B2 (ja) | 2016-09-06 | 2023-01-31 | ザ チルドレンズ メディカル センター コーポレーション | 人工多能性幹細胞由来の免疫細胞 |
| US20190262399A1 (en) | 2016-09-07 | 2019-08-29 | The Broad Institute, Inc. | Compositions and methods for evaluating and modulating immune responses |
| US20190225974A1 (en) | 2016-09-23 | 2019-07-25 | BASF Agricultural Solutions Seed US LLC | Targeted genome optimization in plants |
| US11306305B2 (en) | 2016-09-23 | 2022-04-19 | Board Of Trustees Of Southern Illinois University | Tuning CRISPR/Cas9 activity with chemically modified nucleotide substitutions |
| WO2018064208A1 (en) | 2016-09-28 | 2018-04-05 | The Broad Institute, Inc. | Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof |
| JP2019532644A (ja) | 2016-09-30 | 2019-11-14 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | Rna誘導型核酸修飾酵素及びその使用方法 |
| US10669539B2 (en) | 2016-10-06 | 2020-06-02 | Pioneer Biolabs, Llc | Methods and compositions for generating CRISPR guide RNA libraries |
| CA3039409A1 (en) | 2016-10-07 | 2018-04-12 | Integrated Dna Technologies, Inc. | S. pyogenes cas9 mutant genes and polypeptides encoded by same |
| WO2018067991A1 (en) | 2016-10-07 | 2018-04-12 | The Brigham And Women's Hospital, Inc. | Modulation of novel immune checkpoint targets |
| US11242542B2 (en) | 2016-10-07 | 2022-02-08 | Integrated Dna Technologies, Inc. | S. pyogenes Cas9 mutant genes and polypeptides encoded by same |
| GB201617559D0 (en) | 2016-10-17 | 2016-11-30 | University Court Of The University Of Edinburgh The | Swine comprising modified cd163 and associated methods |
| GB2573406B (en) | 2016-10-18 | 2021-11-10 | Univ Minnesota | Tumor infiltrating lymphocytes and methods of therapy |
| US20180245065A1 (en) | 2016-11-01 | 2018-08-30 | Novartis Ag | Methods and compositions for enhancing gene editing |
| SG11201903697WA (en) | 2016-11-04 | 2019-05-30 | Childrens Hospital Med Ct | Liver organoid compositions and methods of making and using same |
| CA3042259A1 (en) | 2016-11-04 | 2018-05-11 | Flagship Pioneering Innovations V. Inc. | Novel plant cells, plants, and seeds |
| WO2018089386A1 (en) | 2016-11-11 | 2018-05-17 | The Broad Institute, Inc. | Modulation of intestinal epithelial cell differentiation, maintenance and/or function through t cell action |
| AU2017364084B2 (en) | 2016-11-22 | 2024-07-04 | Integrated Dna Technologies, Inc. | CRISPR/Cpf1 systems and methods |
| US9816093B1 (en) | 2016-12-06 | 2017-11-14 | Caribou Biosciences, Inc. | Engineered nucleic acid-targeting nucleic acids |
| MY206324A (en) | 2016-12-08 | 2024-12-10 | Intellia Therapeutics Inc | Modified guide rnas |
| US12404514B2 (en) | 2016-12-09 | 2025-09-02 | The Broad Institute, Inc. | CRISPR-systems for modifying a trait of interest in a plant |
| US11293022B2 (en) | 2016-12-12 | 2022-04-05 | Integrated Dna Technologies, Inc. | Genome editing enhancement |
| US20180179553A1 (en) | 2016-12-14 | 2018-06-28 | Ligandal, Inc. | Compositions and methods for nucleic acid and/or protein payload delivery |
| WO2018119010A1 (en) | 2016-12-19 | 2018-06-28 | Editas Medicine, Inc. | Assessing nuclease cleavage |
| US10550429B2 (en) | 2016-12-22 | 2020-02-04 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
| CA3047163A1 (en) | 2016-12-22 | 2018-06-28 | Monsanto Technology Llc | Genome editing-based crop engineering and production of brachytic plants |
| US10815525B2 (en) | 2016-12-22 | 2020-10-27 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
| EP3565907B1 (en) | 2017-01-06 | 2022-05-04 | Editas Medicine, Inc. | Methods of assessing nuclease cleavage |
| SG11201906795SA (en) | 2017-01-28 | 2019-08-27 | Inari Agriculture Inc | Novel plant cells, plants, and seeds |
| EP4310183B1 (en) | 2017-01-30 | 2025-07-09 | 10X Genomics, Inc. | Methods and systems for droplet-based single cell barcoding |
| TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
| US10995333B2 (en) | 2017-02-06 | 2021-05-04 | 10X Genomics, Inc. | Systems and methods for nucleic acid preparation |
| CA3049989A1 (en) | 2017-02-10 | 2018-08-16 | Zymergen Inc. | A modular universal plasmid design strategy for the assembly and editing of multiple dna constructs for multiple hosts |
| EP3596217A1 (en) | 2017-03-14 | 2020-01-22 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| EP3596207B1 (en) | 2017-03-15 | 2023-12-20 | The Broad Institute, Inc. | Novel cas13b orthologues crispr enzymes and systems |
| US12180546B2 (en) | 2017-03-17 | 2024-12-31 | Massachusetts Institute Of Technology | Methods for identifying and modulating co-occurant cellular phenotypes |
| WO2018175924A1 (en) | 2017-03-24 | 2018-09-27 | The Broad Institute, Inc. | Methods and compositions for regulating innate lymphoid cell inflammatory responses |
| US11963966B2 (en) | 2017-03-31 | 2024-04-23 | Dana-Farber Cancer Institute, Inc. | Compositions and methods for treating ovarian tumors |
| US11913075B2 (en) | 2017-04-01 | 2024-02-27 | The Broad Institute, Inc. | Methods and compositions for detecting and modulating an immunotherapy resistance gene signature in cancer |
| WO2018191558A1 (en) | 2017-04-12 | 2018-10-18 | The Broad Institute, Inc. | Modulation of epithelial cell differentiation, maintenance and/or function through t cell action, and markers and methods of use thereof |
| US20200071773A1 (en) | 2017-04-12 | 2020-03-05 | Massachusetts Eye And Ear Infirmary | Tumor signature for metastasis, compositions of matter methods of use thereof |
| WO2018191520A1 (en) | 2017-04-12 | 2018-10-18 | The Broad Institute, Inc. | Respiratory and sweat gland ionocytes |
| EP3610009A1 (en) | 2017-04-12 | 2020-02-19 | The Broad Institute, Inc. | Novel type vi crispr orthologs and systems |
| EP3609997A4 (en) | 2017-04-14 | 2021-03-03 | Children's Hospital Medical Center | COMPOSITIONS OF STEM CELLS FROM MULTIPLE DONOR CELLS AND THEIR PREPARATION PROCEDURES |
| US12350368B2 (en) | 2017-04-14 | 2025-07-08 | The Broad Institute, Inc. | Delivery of large payloads |
| EP3612629A1 (en) | 2017-04-18 | 2020-02-26 | The Broad Institute, Inc. | Compositions for detecting secretion and methods of use |
| EP3612023A4 (en) | 2017-04-20 | 2021-05-12 | Egenesis, Inc. | METHOD FOR PRODUCING GENETICALLY MODIFIED ANIMALS |
| US12208140B2 (en) | 2017-04-21 | 2025-01-28 | The Broad Institute, Inc. | Targeted delivery to beta cells |
| ES2992912T3 (en) | 2017-04-21 | 2024-12-19 | Massachusetts Gen Hospital | Variants of cpf1 (cas12a) with altered pam specificity |
| US11499151B2 (en) | 2017-04-28 | 2022-11-15 | Editas Medicine, Inc. | Methods and systems for analyzing guide RNA molecules |
| WO2018204777A2 (en) | 2017-05-05 | 2018-11-08 | The Broad Institute, Inc. | Methods for identification and modification of lncrna associated with target genotypes and phenotypes |
| CN110914431B (zh) * | 2017-05-08 | 2024-04-19 | 株式会社图尔金 | 经人工操纵的免疫细胞 |
| EP3622070A2 (en) | 2017-05-10 | 2020-03-18 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| WO2019103857A1 (en) | 2017-11-22 | 2019-05-31 | Iovance Biotherapeutics, Inc. | Expansion of peripheral blood lymphocytes (pbls) from peripheral blood |
| WO2018209324A2 (en) | 2017-05-11 | 2018-11-15 | The Broad Institute, Inc. | Methods and compositions of use of cd8+ tumor infiltrating lymphocyte subtypes and gene signatures thereof |
| WO2018213708A1 (en) | 2017-05-18 | 2018-11-22 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| US12297436B2 (en) | 2017-05-18 | 2025-05-13 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| AU2018273986A1 (en) | 2017-05-25 | 2019-12-12 | The General Hospital Corporation | Bipartite base editor (BBE) architectures and type-II-C-Cas9 zinc finger editing |
| US11788087B2 (en) | 2017-05-25 | 2023-10-17 | The Children's Medical Center Corporation | BCL11A guide delivery |
| CN109526228B (zh) | 2017-05-26 | 2022-11-25 | 10X基因组学有限公司 | 转座酶可接近性染色质的单细胞分析 |
| US10844372B2 (en) | 2017-05-26 | 2020-11-24 | 10X Genomics, Inc. | Single cell analysis of transposase accessible chromatin |
| EP3409104A1 (en) | 2017-05-31 | 2018-12-05 | Vilmorin et Cie | Tomato plant resistant to tomato yellow leaf curl virus, powdery mildew, and nematodes |
| WO2020249996A1 (en) | 2019-06-14 | 2020-12-17 | Vilmorin & Cie | Resistance in plants of solanum lycopersicum to the tobamovirus tomato brown rugose fruit virus |
| EP3409106A1 (en) | 2017-06-01 | 2018-12-05 | Vilmorin et Cie | Tolerance in plants of solanum lycopersicum to the tobamovirus tomato brown rugose fruit virus (tbrfv) |
| AU2018280116B2 (en) | 2017-06-05 | 2025-03-27 | Research Institute At Nationwide Children's Hospital | Enhanced modified viral capsid proteins |
| US11603544B2 (en) | 2017-06-05 | 2023-03-14 | Fred Hutchinson Cancer Center | Genomic safe harbors for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies |
| KR20200026874A (ko) | 2017-06-06 | 2020-03-11 | 지머젠 인코포레이티드 | 대장균 개량을 위한 htp 게놈 공학 플랫폼 |
| KR20200026878A (ko) | 2017-06-06 | 2020-03-11 | 지머젠 인코포레이티드 | 균류 균주를 개량하기 위한 htp 게놈 공학 플랫폼 |
| JP7518620B2 (ja) | 2017-06-09 | 2024-07-18 | エディタス・メディシン,インコーポレイテッド | 操作されたcas9ヌクレアーゼ |
| IL271275B1 (en) | 2017-06-13 | 2026-04-01 | Flagship Pioneering Innovations V Inc | Preparations containing croons and their uses |
| WO2018232195A1 (en) | 2017-06-14 | 2018-12-20 | The Broad Institute, Inc. | Compositions and methods targeting complement component 3 for inhibiting tumor growth |
| US10011849B1 (en) | 2017-06-23 | 2018-07-03 | Inscripta, Inc. | Nucleic acid-guided nucleases |
| US9982279B1 (en) | 2017-06-23 | 2018-05-29 | Inscripta, Inc. | Nucleic acid-guided nucleases |
| CA3064601A1 (en) | 2017-06-26 | 2019-01-03 | President And Fellows Of Harvard College | Crispr/cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing |
| EP3645721A1 (en) | 2017-06-30 | 2020-05-06 | Novartis AG | Methods for the treatment of disease with gene editing systems |
| JP2020530307A (ja) | 2017-06-30 | 2020-10-22 | インティマ・バイオサイエンス,インコーポレーテッド | 遺伝子治療のためのアデノ随伴ウイルスベクター |
| WO2019014564A1 (en) | 2017-07-14 | 2019-01-17 | Editas Medicine, Inc. | SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES |
| WO2019011118A1 (zh) * | 2017-07-14 | 2019-01-17 | 苏州克睿基因生物科技有限公司 | 一种基因编辑系统及基因编辑的方法 |
| US12049643B2 (en) | 2017-07-14 | 2024-07-30 | The Broad Institute, Inc. | Methods and compositions for modulating cytotoxic lymphocyte activity |
| US12105089B2 (en) | 2017-07-17 | 2024-10-01 | The Broad Institute, Inc. | Cell atlas of the healthy and ulcerative colitis human colon |
| US12239646B2 (en) | 2017-07-19 | 2025-03-04 | Dana-Farber Cancer Institute, Inc. | Cancer diagnostic and treatment |
| CA3073448A1 (en) | 2017-08-23 | 2019-02-28 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
| US20190076814A1 (en) | 2017-09-11 | 2019-03-14 | Synthego Corporation | Biopolymer synthesis system and method |
| WO2019060759A1 (en) | 2017-09-21 | 2019-03-28 | Dana-Farber Cancer Institute, Inc. | ISOLATION, PRESERVATION, COMPOSITIONS AND USES OF PLANT EXTRACTS JUSTICIA |
| AU2018338318B2 (en) | 2017-09-21 | 2022-12-22 | Massachusetts Institute Of Technology | Systems, methods, and compositions for targeted nucleic acid editing |
| BR112020005803A2 (pt) | 2017-09-25 | 2020-12-01 | Agrospheres, Inc. | composições e métodos para produção e administração moduláveis de produtos biológicos |
| AU2018339164B2 (en) * | 2017-09-28 | 2022-06-16 | Toolgen Incorporated | Artificial genome manipulation for gene expression regulation |
| US11572574B2 (en) | 2017-09-28 | 2023-02-07 | Toolgen Incorporated | Artificial genome manipulation for gene expression regulation |
| WO2019070755A1 (en) | 2017-10-02 | 2019-04-11 | The Broad Institute, Inc. | METHODS AND COMPOSITIONS FOR DETECTING AND MODULATING A GENETIC SIGNATURE OF IMMUNOTHERAPY RESISTANCE IN CANCER |
| WO2019071054A1 (en) | 2017-10-04 | 2019-04-11 | The Broad Institute, Inc. | METHODS AND COMPOSITIONS FOR MODIFYING THE FUNCTION AND STRUCTURE OF BUCKLES AND / OR CHROMATIN DOMAINS |
| US12297457B2 (en) | 2017-10-10 | 2025-05-13 | Children's Hospital Medical Center | Esophageal tissue and/or organoid compositions and methods of making same |
| WO2019075197A1 (en) | 2017-10-11 | 2019-04-18 | The General Hospital Corporation | METHODS OF DETECTION OF INDIVIDUAL SITE-SPECIFIC PARASITE GENOMIC DEAMINATION BY BASE EDITING TECHNOLOGIES |
| WO2019079362A1 (en) | 2017-10-16 | 2019-04-25 | Massachusetts Institute Of Technology | HOST-PATHOGENIC INTERACTION OF MYCOBACTERIUM TUBERCULOSIS |
| SG11202003024WA (en) | 2017-10-20 | 2020-05-28 | Hutchinson Fred Cancer Res | Systems and methods to produce b cells genetically modified to express selected antibodies |
| WO2019135816A2 (en) | 2017-10-23 | 2019-07-11 | The Broad Institute, Inc. | Novel nucleic acid modifiers |
| WO2019089803A1 (en) | 2017-10-31 | 2019-05-09 | The Broad Institute, Inc. | Methods and compositions for studying cell evolution |
| US20210180053A1 (en) | 2017-11-01 | 2021-06-17 | Novartis Ag | Synthetic rnas and methods of use |
| WO2019084664A1 (en) * | 2017-11-02 | 2019-05-09 | The Governors Of The University Of Alberta | Chemically-modified guide rnas to improve crispr-cas protein specificity |
| WO2019094955A1 (en) | 2017-11-13 | 2019-05-16 | The Broad Institute, Inc. | Methods and compositions for targeting developmental and oncogenic programs in h3k27m gliomas |
| EP3710039A4 (en) | 2017-11-13 | 2021-08-04 | The Broad Institute, Inc. | METHODS AND COMPOSITIONS FOR TREATMENT OF CANCER BY TARGETING THE CLEC2D-KLRB1 PATH |
| US12221720B2 (en) | 2017-11-13 | 2025-02-11 | The Broad Institute, Inc. | Methods for determining spatial and temporal gene expression dynamics during adult neurogenesis in single cells |
| CN111051523B (zh) | 2017-11-15 | 2024-03-19 | 10X基因组学有限公司 | 功能化凝胶珠 |
| WO2019099943A1 (en) * | 2017-11-16 | 2019-05-23 | Astrazeneca Ab | Compositions and methods for improving the efficacy of cas9-based knock-in strategies |
| US10829815B2 (en) | 2017-11-17 | 2020-11-10 | 10X Genomics, Inc. | Methods and systems for associating physical and genetic properties of biological particles |
| CN111601883B (zh) | 2017-11-17 | 2024-06-21 | 艾欧凡斯生物治疗公司 | 由细针抽吸物和小活检物扩增til |
| SG11202005147WA (en) | 2017-12-05 | 2020-06-29 | Vertex Pharma | Crispr-cas9 modified cd34+ human hematopoietic stem and progenitor cells and uses thereof |
| WO2019113506A1 (en) | 2017-12-07 | 2019-06-13 | The Broad Institute, Inc. | Methods and compositions for multiplexing single cell and single nuclei sequencing |
| WO2019126037A1 (en) | 2017-12-19 | 2019-06-27 | City Of Hope | Modified tracrrnas grnas, and uses thereof |
| EP3727394A4 (en) | 2017-12-21 | 2021-09-08 | Children's Hospital Medical Center | DIGITALIZED HUMAN ORGANOIDS AND METHOD OF USING THEREOF |
| RU2652899C1 (ru) * | 2017-12-28 | 2018-05-03 | Федеральное бюджетное учреждение науки "Центральный научно-исследовательский институт эпидемиологии" Федеральной службы по надзору в сфере защиты прав потребителей и благополучия человека (ФБУН ЦНИИ Эпидемиологии Роспотребнадзора) | РНК-проводники для подавления репликации вируса гепатита B и для элиминации вируса гепатита B из клетки-хозяина |
| US11994512B2 (en) | 2018-01-04 | 2024-05-28 | Massachusetts Institute Of Technology | Single-cell genomic methods to generate ex vivo cell systems that recapitulate in vivo biology with improved fidelity |
| US20200362366A1 (en) | 2018-01-12 | 2020-11-19 | Basf Se | Gene underlying the number of spikelets per spike qtl in wheat on chromosome 7a |
| JP7391854B2 (ja) | 2018-01-17 | 2023-12-05 | バーテックス ファーマシューティカルズ インコーポレイテッド | Dna-pk阻害剤 |
| AU2019209292B2 (en) | 2018-01-17 | 2023-10-05 | Vertex Pharmaceuticals Incorporated | Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency |
| BR112020014140A2 (pt) | 2018-01-17 | 2020-12-01 | Vertex Pharmaceuticals Incorporated | inibidores de dna-pk |
| US12509492B2 (en) | 2018-01-19 | 2025-12-30 | Duke University | Genome engineering with CRISPR-Cas systems in eukaryotes |
| WO2019147743A1 (en) | 2018-01-26 | 2019-08-01 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
| US11926835B1 (en) | 2018-01-29 | 2024-03-12 | Inari Agriculture Technology, Inc. | Methods for efficient tomato genome editing |
| AR114350A1 (es) | 2018-01-31 | 2020-08-26 | Res Institute At Nationwide ChildrenS Hospital | Terapia genética para la distrofia muscular de cinturas del tipo 2c |
| US11268077B2 (en) | 2018-02-05 | 2022-03-08 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| WO2019150196A1 (en) | 2018-02-05 | 2019-08-08 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| WO2019157326A1 (en) | 2018-02-08 | 2019-08-15 | Zymergen Inc. | Genome editing using crispr in corynebacterium |
| SG11202007686VA (en) | 2018-02-12 | 2020-09-29 | 10X Genomics Inc | Methods characterizing multiple analytes from individual cells or cell populations |
| EP3755792A4 (en) | 2018-02-23 | 2021-12-08 | Pioneer Hi-Bred International, Inc. | NEW CAS9 ORTHOLOGIST |
| KR102907245B1 (ko) | 2018-03-14 | 2026-01-05 | 에디타스 메디신, 인코포레이티드 | 혈색소병증 치료를 위한 시스템 및 방법 |
| PL235163B1 (pl) * | 2018-04-05 | 2020-06-01 | Inst Hodowli I Aklimatyzacji Roslin Panstwowy Inst Badawczy | Sekwencja nukleotydowa syntetycznego genu Cas9, kaseta kierująca sgRNA do edytowania genomu roślinnego i wydajny system do ukierunkowanej mutagenezy wybranego regionu genomu roślinnego |
| CN116536272A (zh) | 2018-04-06 | 2023-08-04 | 儿童医疗中心有限公司 | 用于体细胞重新编程和调整印记的组合物和方法 |
| WO2019195166A1 (en) | 2018-04-06 | 2019-10-10 | 10X Genomics, Inc. | Systems and methods for quality control in single cell processing |
| EP3781585A4 (en) | 2018-04-17 | 2022-01-26 | The General Hospital Corporation | SENSITIVE IN VITRO TESTS FOR SUBSTRATE PREFERENCES AND SITE FOR NUCLEIC ACID BINDERS, MODIFIERS AND CLEAVAGE AGENTS |
| GB2587970B (en) | 2018-04-19 | 2023-02-08 | Univ California | Compositions and methods for gene editing |
| WO2019204585A1 (en) | 2018-04-19 | 2019-10-24 | Massachusetts Institute Of Technology | Single-stranded break detection in double-stranded dna |
| DK3560330T3 (da) | 2018-04-24 | 2022-07-11 | Kws Saat Se & Co Kgaa | Planter med forbedret fordøjelighed og markørhaplotyper |
| US11957695B2 (en) | 2018-04-26 | 2024-04-16 | The Broad Institute, Inc. | Methods and compositions targeting glucocorticoid signaling for modulating immune responses |
| EP3784254A1 (en) | 2018-04-27 | 2021-03-03 | Iovance Biotherapeutics, Inc. | Closed process for expansion and gene editing of tumor infiltrating lymphocytes and uses of same in immunotherapy |
| WO2019210268A2 (en) | 2018-04-27 | 2019-10-31 | The Broad Institute, Inc. | Sequencing-based proteomics |
| JP7568511B2 (ja) | 2018-04-27 | 2024-10-16 | ジーンエディット インコーポレイテッド | カチオン性ポリマーおよびその生体分子送達のための使用 |
| US12522811B2 (en) | 2018-05-01 | 2026-01-13 | The Children's Medical Center Corporation | Enhanced BCL11A RNP / CRISPR delivery and editing using a 3XNLS-CAS9 |
| WO2019213013A1 (en) | 2018-05-02 | 2019-11-07 | The Children's Medical Center Corporation | Improved bcl11a micrornas for treating hemoglobinopathies |
| WO2019213660A2 (en) | 2018-05-04 | 2019-11-07 | The Broad Institute, Inc. | Compositions and methods for modulating cgrp signaling to regulate innate lymphoid cell inflammatory responses |
| WO2019217294A1 (en) | 2018-05-06 | 2019-11-14 | Emendobio Inc. | Differential knockout of an allele of a heterozygous elane gene |
| WO2019217758A1 (en) | 2018-05-10 | 2019-11-14 | 10X Genomics, Inc. | Methods and systems for molecular library generation |
| EP3794130A4 (en) | 2018-05-16 | 2022-07-27 | Synthego Corporation | METHODS AND SYSTEMS FOR DESIGNING AND USING GUIDE RNA |
| CN108707628B (zh) * | 2018-05-28 | 2021-11-23 | 上海海洋大学 | 斑马鱼notch2基因突变体的制备方法 |
| US20210371932A1 (en) | 2018-06-01 | 2021-12-02 | Massachusetts Institute Of Technology | Methods and compositions for detecting and modulating microenvironment gene signatures from the csf of metastasis patients |
| US11866719B1 (en) | 2018-06-04 | 2024-01-09 | Inari Agriculture Technology, Inc. | Heterologous integration of regulatory elements to alter gene expression in wheat cells and wheat plants |
| KR20210018437A (ko) | 2018-06-07 | 2021-02-17 | 더 브리검 앤드 우먼즈 하스피털, 인크. | 조혈 줄기 세포를 생산하기 위한 방법 |
| US20220403001A1 (en) | 2018-06-12 | 2022-12-22 | Obsidian Therapeutics, Inc. | Pde5 derived regulatory constructs and methods of use in immunotherapy |
| US12036240B2 (en) | 2018-06-14 | 2024-07-16 | The Broad Institute, Inc. | Compositions and methods targeting complement component 3 for inhibiting tumor growth |
| BR112020025349A2 (pt) | 2018-06-15 | 2021-03-09 | KWS SAAT SE & Co. KGaA | Métodos para melhorar a engenharia e regeneração do genoma em planta |
| CN112585269B (zh) | 2018-06-15 | 2025-01-07 | 科沃施种子欧洲股份两合公司 | 改善植物中基因组工程化和再生的方法ii |
| EP3814527B1 (en) | 2018-06-26 | 2023-02-22 | Massachusetts Institute of Technology | Crispr effector system based amplification methods, systems, and diagnostics |
| BR112020025319A2 (pt) | 2018-06-26 | 2021-03-09 | The Broad Institute Inc. | Composições, sistemas e métodos de amplificação baseada em crispr/cas e transposase |
| WO2020002579A1 (en) | 2018-06-29 | 2020-01-02 | Stichting Het Nederlands Kanker Instituut - Antoni Van Leeuwenhoek Ziekenhuis | Tweak-receptor agonists for use in combination with immunotherapy of a cancer |
| WO2020006458A1 (en) | 2018-06-29 | 2020-01-02 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
| AU2019291918B2 (en) | 2018-06-29 | 2025-06-12 | Editas Medicine, Inc. | Synthetic guide molecules, compositions and methods relating thereto |
| JP2021530212A (ja) | 2018-07-13 | 2021-11-11 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California | レトロトランスポゾンベースの送達媒体及びその使用方法 |
| US12421500B2 (en) | 2018-07-26 | 2025-09-23 | Children's Hospital Medical Center | Hepato-biliary-pancreatic tissues and methods of making same |
| US20200032335A1 (en) | 2018-07-27 | 2020-01-30 | 10X Genomics, Inc. | Systems and methods for metabolome analysis |
| KR20210053898A (ko) | 2018-07-31 | 2021-05-12 | 더 브로드 인스티튜트, 인코퍼레이티드 | 신규 crispr 효소 및 시스템 |
| WO2020028729A1 (en) | 2018-08-01 | 2020-02-06 | Mammoth Biosciences, Inc. | Programmable nuclease compositions and methods of use thereof |
| WO2020033601A1 (en) | 2018-08-07 | 2020-02-13 | The Broad Institute, Inc. | Novel cas12b enzymes and systems |
| EP3607819A1 (en) | 2018-08-10 | 2020-02-12 | Vilmorin et Cie | Resistance to xanthomonas campestris pv. campestris (xcc) in cauliflower |
| EP3821020A4 (en) | 2018-08-15 | 2022-05-04 | Zymergen Inc. | APPLICATIONS OF CRISPRI IN HIGH-PERFORMANCE METABOLIC ENGINEERING |
| US12421507B2 (en) | 2018-08-20 | 2025-09-23 | The Broad Institute, Inc. | Methods and compositions for optochemical control of CRISPR-CAS9 |
| US20210324357A1 (en) | 2018-08-20 | 2021-10-21 | The Brigham And Women's Hospital, Inc. | Degradation domain modifications for spatio-temporal control of rna-guided nucleases |
| EP3841205A4 (en) | 2018-08-22 | 2022-08-17 | The Regents of The University of California | Variant type v crispr/cas effector polypeptides and methods of use thereof |
| US20230021641A1 (en) * | 2018-08-23 | 2023-01-26 | The Broad Institute, Inc. | Cas9 variants having non-canonical pam specificities and uses thereof |
| EP3841204A4 (en) | 2018-08-23 | 2022-05-18 | Sangamo Therapeutics, Inc. | CHANGED TARGET SPECIFIC BASE EDITORS |
| US12545910B2 (en) | 2018-08-31 | 2026-02-10 | Inari Agriculture Technology, Inc. | Compositions, systems, and methods for genome editing |
| US12534743B2 (en) | 2018-08-31 | 2026-01-27 | Inari Agriculture Technology, Inc. | Compositions, systems, and methods for genome editing |
| US11459551B1 (en) | 2018-08-31 | 2022-10-04 | Inari Agriculture Technology, Inc. | Compositions, systems, and methods for genome editing |
| US20210317479A1 (en) | 2018-09-06 | 2021-10-14 | The Broad Institute, Inc. | Nucleic acid assemblies for use in targeted delivery |
| US10724052B2 (en) | 2018-09-07 | 2020-07-28 | Crispr Therapeutics Ag | Universal donor cells |
| JP2021535753A (ja) | 2018-09-12 | 2021-12-23 | チルドレンズ ホスピタル メディカル センター | 造血幹細胞およびその派生体の生成のためのオルガノイド組成物 |
| US12398402B2 (en) | 2018-09-12 | 2025-08-26 | Fred Hutchinson Cancer Center | Reducing CD33 expression to selectively protect therapeutic cells |
| EP3852813A4 (en) | 2018-09-18 | 2022-06-22 | VNV Newco Inc. | ARC-BASED CAPSIDS AND THEIR USES |
| US20210403907A1 (en) | 2018-09-18 | 2021-12-30 | Vnv Newco Inc. | Arc-based capsids and uses thereof |
| WO2020069029A1 (en) | 2018-09-26 | 2020-04-02 | Emendobio Inc. | Novel crispr nucleases |
| WO2020076976A1 (en) | 2018-10-10 | 2020-04-16 | Readcoor, Inc. | Three-dimensional spatial molecular indexing |
| US20220411783A1 (en) | 2018-10-12 | 2022-12-29 | The Broad Institute, Inc. | Method for extracting nuclei or whole cells from formalin-fixed paraffin-embedded tissues |
| WO2020081730A2 (en) | 2018-10-16 | 2020-04-23 | Massachusetts Institute Of Technology | Methods and compositions for modulating microenvironment |
| US12116458B2 (en) | 2018-10-24 | 2024-10-15 | Genedit Inc. | Cationic polymer with alkyl side chains and use for biomolecule delivery |
| WO2020086742A1 (en) | 2018-10-24 | 2020-04-30 | Obsidian Therapeutics, Inc. | Er tunable protein regulation |
| US11407995B1 (en) | 2018-10-26 | 2022-08-09 | Inari Agriculture Technology, Inc. | RNA-guided nucleases and DNA binding proteins |
| SG11202104409YA (en) | 2018-10-31 | 2021-05-28 | Zymergen Inc | Multiplexed deterministic assembly of dna libraries |
| US20220010321A1 (en) * | 2018-11-01 | 2022-01-13 | Keygene N.V. | Dual guide rna for crispr/cas genome editing in plants cells |
| US11434477B1 (en) | 2018-11-02 | 2022-09-06 | Inari Agriculture Technology, Inc. | RNA-guided nucleases and DNA binding proteins |
| TW202039831A (zh) | 2018-11-05 | 2020-11-01 | 美商艾歐凡斯生物治療公司 | 對抗pd-1抗體呈現難治性之非小細胞肺癌(nsclc)病患之治療 |
| CA3118493A1 (en) | 2018-11-05 | 2020-05-14 | Iovance Biotherapeutics, Inc. | Expansion of tils utilizing akt pathway inhibitors |
| TW202039829A (zh) | 2018-11-05 | 2020-11-01 | 美商艾歐凡斯生物治療公司 | 改善之腫瘤反應性t細胞的選擇 |
| CR20210295A (es) | 2018-11-05 | 2021-07-09 | Iovance Biotherapeutics Inc | Procesos para la producción de linfocitos infiltrantes de tumor y usos de estos en inmunoterapia |
| MX2021005444A (es) | 2018-11-08 | 2021-06-15 | Triton Algae Innovations Inc | Procedimientos para la superproduccion de protoporfirina ix en algas y composiciones de la misma. |
| US12402610B2 (en) | 2018-11-09 | 2025-09-02 | The Broad Institute, Inc. | Methods and compositions for modulating innate lymphoid cell pathogenic effectors |
| US12165743B2 (en) | 2018-11-09 | 2024-12-10 | The Broad Institute, Inc. | Compressed sensing for screening and tissue imaging |
| EP3877517A4 (en) | 2018-11-09 | 2022-09-07 | Inari Agriculture, Inc. | RNA-DRIVEN NUCLEASES AND DNA-BINDING PROTEINS |
| US11459607B1 (en) | 2018-12-10 | 2022-10-04 | 10X Genomics, Inc. | Systems and methods for processing-nucleic acid molecules from a single cell using sequential co-partitioning and composite barcodes |
| US11166996B2 (en) | 2018-12-12 | 2021-11-09 | Flagship Pioneering Innovations V, Inc. | Anellovirus compositions and methods of use |
| CN113166744B (zh) | 2018-12-14 | 2025-02-07 | 先锋国际良种公司 | 用于基因组编辑的新颖crispr-cas系统 |
| US12264323B2 (en) | 2018-12-17 | 2025-04-01 | The Broad Institute, Inc. | CRISPR CPF1 direct repeat variants |
| US20220062394A1 (en) | 2018-12-17 | 2022-03-03 | The Broad Institute, Inc. | Methods for identifying neoantigens |
| AU2019406778A1 (en) | 2018-12-17 | 2021-07-22 | Massachusetts Institute Of Technology | Crispr-associated transposase systems and methods of use thereof |
| WO2020131547A1 (en) | 2018-12-19 | 2020-06-25 | Iovance Biotherapeutics, Inc. | Methods of expanding tumor infiltrating lymphocytes using engineered cytokine receptor pairs and uses thereof |
| EP3931313A2 (en) | 2019-01-04 | 2022-01-05 | Mammoth Biosciences, Inc. | Programmable nuclease improvements and compositions and methods for nucleic acid amplification and detection |
| US11739156B2 (en) | 2019-01-06 | 2023-08-29 | The Broad Institute, Inc. Massachusetts Institute of Technology | Methods and compositions for overcoming immunosuppression |
| US11845983B1 (en) | 2019-01-09 | 2023-12-19 | 10X Genomics, Inc. | Methods and systems for multiplexing of droplet based assays |
| WO2020160044A1 (en) | 2019-01-28 | 2020-08-06 | The Broad Institute, Inc. | In-situ spatial transcriptomics |
| EP3918080A1 (en) | 2019-01-29 | 2021-12-08 | The University Of Warwick | Methods for enhancing genome engineering efficiency |
| WO2020160371A1 (en) | 2019-02-01 | 2020-08-06 | The University Of Hong Kong | Innervated organoid compositions and methods of making same |
| EP3921417A4 (en) | 2019-02-04 | 2022-11-09 | The General Hospital Corporation | VARIANTS OF ADENINE DNA BASES WITH REDUCED OFF-TARGET RNA EDIT |
| WO2020163379A1 (en) | 2019-02-05 | 2020-08-13 | Emendobio Inc. | Crispr compositions and methods for promoting gene editing of ribosomal protein s19 (rps19) gene |
| WO2020163856A1 (en) | 2019-02-10 | 2020-08-13 | The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone | Modified mitochondrion and methods of use thereof |
| US11851683B1 (en) | 2019-02-12 | 2023-12-26 | 10X Genomics, Inc. | Methods and systems for selective analysis of cellular samples |
| CA3131390A1 (en) | 2019-02-26 | 2020-09-03 | Research Institute Of Nationwide Children's Hospital | Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy |
| KR20210136050A (ko) | 2019-03-01 | 2021-11-16 | 이오반스 바이오테라퓨틱스, 인크. | 액상 종양으로부터의 종양 침윤 림프구의 확장 및 그의 치료 용도 |
| EP4219700A1 (en) | 2019-03-07 | 2023-08-02 | The Regents of the University of California | Crispr-cas effector polypeptides and methods of use thereof |
| KR20210149251A (ko) | 2019-03-08 | 2021-12-08 | 옵시디안 테라퓨틱스, 인크. | 조율가능한 조절을 위한 인간 탄산 무수화효소 2 조성물 및 방법 |
| CA3129871A1 (en) | 2019-03-08 | 2020-09-17 | Zymergen Inc. | Iterative genome editing in microbes |
| US11053515B2 (en) | 2019-03-08 | 2021-07-06 | Zymergen Inc. | Pooled genome editing in microbes |
| WO2020191102A1 (en) | 2019-03-18 | 2020-09-24 | The Broad Institute, Inc. | Type vii crispr proteins and systems |
| US20220162649A1 (en) | 2019-04-01 | 2022-05-26 | The Broad Institute, Inc. | Novel nucleic acid modifiers |
| CA3136061A1 (en) | 2019-04-05 | 2020-10-08 | Osaka University | Method for producing knock-in cell |
| WO2020219776A1 (en) | 2019-04-23 | 2020-10-29 | Genedit Inc. | Cationic polymer with alkyl side chains |
| US11162079B2 (en) | 2019-05-10 | 2021-11-02 | The Regents Of The University Of California | Blood type O Rh-hypo-immunogenic pluripotent cells |
| WO2020232029A1 (en) | 2019-05-13 | 2020-11-19 | Iovance Biotherapeutics, Inc. | Methods and compositions for selecting tumor infiltrating lymphocytes and uses of the same in immunotherapy |
| US20220243287A1 (en) | 2019-05-13 | 2022-08-04 | KWS SAAT SE & Co. KGaA | Drought tolerance in corn |
| US12116583B2 (en) | 2019-05-14 | 2024-10-15 | Yield10 Bioscience, Inc. | Modified plants comprising a polynucleotide comprising a non-cognate promoter operably linked to a coding sequence that encodes a transcription factor |
| US20220235340A1 (en) | 2019-05-20 | 2022-07-28 | The Broad Institute, Inc. | Novel crispr-cas systems and uses thereof |
| WO2020236972A2 (en) | 2019-05-20 | 2020-11-26 | The Broad Institute, Inc. | Non-class i multi-component nucleic acid targeting systems |
| JP2022533713A (ja) | 2019-05-21 | 2022-07-25 | サンガモ セラピューティクス, インコーポレイテッド | 調節性t細胞における制御された導入遺伝子発現 |
| AR118995A1 (es) | 2019-05-25 | 2021-11-17 | Kws Saat Se & Co Kgaa | Mejorador de la inducción de haploides |
| JP7805789B2 (ja) | 2019-05-28 | 2026-01-26 | ジーンエディット インコーポレイテッド | 生体分子送達のための複数の機能化側鎖を含むポリマー |
| MX2021014528A (es) | 2019-05-29 | 2022-01-06 | Monsanto Technology Llc | Metodos y composiciones para generar alelos dominantes usando edicion del genoma. |
| CA3141814A1 (en) | 2019-05-31 | 2020-12-03 | Children's Hospital Medical Center | Shaped organoid compositions and methods of making same |
| US20220243178A1 (en) | 2019-05-31 | 2022-08-04 | The Broad Institute, Inc. | Methods for treating metabolic disorders by targeting adcy5 |
| CN114174494A (zh) | 2019-05-31 | 2022-03-11 | 儿童医院医学中心 | 产生和扩增造血干细胞的方法 |
| WO2020254850A1 (en) | 2019-06-21 | 2020-12-24 | Vilmorin & Cie | Improvement of quality and permanence of green color of peppers at maturity and over-maturity |
| CN120665955A (zh) | 2019-06-25 | 2025-09-19 | 伊纳瑞农业技术有限公司 | 改善的同源依赖修复基因组编辑 |
| JP2022539248A (ja) | 2019-07-02 | 2022-09-07 | フレッド ハッチンソン キャンサー リサーチ センター | 組換えad35ベクター及び関連遺伝子治療改善 |
| US11746354B2 (en) | 2019-07-19 | 2023-09-05 | Inari Agriculture Technology, Inc. | Homology dependent repair genome editing |
| WO2021019272A1 (en) | 2019-07-31 | 2021-02-04 | Vilmorin & Cie | Tolerance to tolcndv in cucumber |
| WO2021026294A1 (en) | 2019-08-06 | 2021-02-11 | Children's Medical Center Corporation | Methods and compositions for reconstituting microglia |
| EP3772542A1 (en) | 2019-08-07 | 2021-02-10 | KWS SAAT SE & Co. KGaA | Modifying genetic variation in crops by modulating the pachytene checkpoint protein 2 |
| WO2021028359A1 (en) | 2019-08-09 | 2021-02-18 | Sangamo Therapeutics France | Controlled expression of chimeric antigen receptors in t cells |
| EP4488287A3 (en) | 2019-08-21 | 2025-03-05 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy |
| WO2021041922A1 (en) | 2019-08-30 | 2021-03-04 | The Broad Institute, Inc. | Crispr-associated mu transposase systems |
| AU2020340622A1 (en) | 2019-09-05 | 2022-03-03 | Crispr Therapeutics Ag | Universal donor cells |
| US11118195B2 (en) | 2019-09-05 | 2021-09-14 | Crispr Therapeutics Ag | Universal donor cells |
| WO2021046451A1 (en) | 2019-09-06 | 2021-03-11 | Obsidian Therapeutics, Inc. | Compositions and methods for dhfr tunable protein regulation |
| US12235262B1 (en) | 2019-09-09 | 2025-02-25 | 10X Genomics, Inc. | Methods and systems for single cell protein analysis |
| US20230025039A1 (en) | 2019-09-20 | 2023-01-26 | The Broad Institute, Inc. | Novel type vi crispr enzymes and systems |
| CN114391040A (zh) | 2019-09-23 | 2022-04-22 | 欧米茄治疗公司 | 用于调节载脂蛋白b(apob)基因表达的组合物和方法 |
| AU2020352552A1 (en) | 2019-09-23 | 2022-03-17 | Omega Therapeutics, Inc. | Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (HNF4α) gene expression |
| US11981922B2 (en) | 2019-10-03 | 2024-05-14 | Dana-Farber Cancer Institute, Inc. | Methods and compositions for the modulation of cell interactions and signaling in the tumor microenvironment |
| WO2021074367A1 (en) | 2019-10-17 | 2021-04-22 | KWS SAAT SE & Co. KGaA | Enhanced disease resistance of crops by downregulation of repressor genes |
| WO2021081378A1 (en) | 2019-10-25 | 2021-04-29 | Iovance Biotherapeutics, Inc. | Gene editing of tumor infiltrating lymphocytes and uses of same in immunotherapy |
| GB201916020D0 (en) | 2019-11-04 | 2019-12-18 | Univ Of Essex Enterprise Limited | Crispr-mediated identification of biotinylated proteins and chromatin regions |
| WO2021094805A1 (en) | 2019-11-14 | 2021-05-20 | Vilmorin & Cie | Resistance to acidovorax valerianellae in corn salad |
| WO2021118990A1 (en) | 2019-12-11 | 2021-06-17 | Iovance Biotherapeutics, Inc. | Processes for the production of tumor infiltrating lymphocytes (tils) and methods of using the same |
| EP4706689A2 (en) | 2019-12-19 | 2026-03-11 | Entrada Therapeutics, Inc. | Compositions for delivery of antisense compounds |
| CN116234906A (zh) | 2020-01-13 | 2023-06-06 | 萨那生物技术股份有限公司 | 血型抗原的修饰 |
| IL294715A (en) | 2020-01-23 | 2022-09-01 | Childrens Medical Ct Corp | Stroma-free t cell differentiation from human pluripotent stem cells |
| WO2021151085A2 (en) | 2020-01-24 | 2021-07-29 | The General Hospital Corporation | Crispr-cas enzymes with enhanced on-target activity |
| WO2021151073A2 (en) | 2020-01-24 | 2021-07-29 | The General Hospital Corporation | Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants |
| CN115362264A (zh) | 2020-01-29 | 2022-11-18 | 10X基因组学有限公司 | 用于分析物检测的组合物和方法 |
| EP3872190A1 (en) | 2020-02-26 | 2021-09-01 | Antibodies-Online GmbH | A method of using cut&run or cut&tag to validate crispr-cas targeting |
| KR102676313B1 (ko) * | 2020-02-27 | 2024-06-17 | 경희대학교 산학협력단 | 유전자 선별 방법 및 장치 |
| US20240191248A1 (en) | 2020-02-28 | 2024-06-13 | KWS SAAT SE & Co. KGaA | Method for rapid genome modification in recalcitrant plants |
| US12473563B2 (en) | 2020-02-28 | 2025-11-18 | KWS SAAT SE & Co. KGaA | Immature inflorescence meristem editing |
| WO2021183720A1 (en) | 2020-03-11 | 2021-09-16 | Omega Therapeutics, Inc. | Compositions and methods for modulating forkhead box p3 (foxp3) gene expression |
| US12057197B2 (en) | 2020-04-03 | 2024-08-06 | Creyon Bio, Inc. | Oligonucleotide-based machine learning |
| US20230165909A1 (en) | 2020-04-21 | 2023-06-01 | Aspen Neuroscience, Inc. | Gene editing of lrrk2 in stem cells and method of use of cells differentiated therefrom |
| US20250263752A1 (en) | 2020-04-21 | 2025-08-21 | Aspen Neuroscience, Inc. | Gene editing of gba1 in stem cells and method of use of cells differentiated therefrom |
| WO2021217082A1 (en) | 2020-04-23 | 2021-10-28 | Genedit Inc. | Polymer with cationic and hydrophobic side chains |
| CA3176826A1 (en) | 2020-05-04 | 2021-11-11 | Iovance Biotherapeutics, Inc. | Processes for production of tumor infiltrating lymphocytes and uses of the same in immunotherapy |
| EP4146797A1 (en) | 2020-05-06 | 2023-03-15 | Orchard Therapeutics (Europe) Limited | Treatment for neurodegenerative diseases |
| CN121884957A (zh) | 2020-05-22 | 2026-04-17 | 因斯特罗公司 | 使用机器学习模型预测疾病结果 |
| WO2021239986A1 (en) | 2020-05-29 | 2021-12-02 | KWS SAAT SE & Co. KGaA | Plant haploid induction |
| WO2021245435A1 (en) | 2020-06-03 | 2021-12-09 | Vilmorin & Cie | Melon plants resistant to scab disease, aphids and powdery mildew |
| BR112022024738A2 (pt) | 2020-06-05 | 2023-03-07 | Vilmorin & Cie | Planta, célula de planta, cultura de tecidos de células, parte de planta, semente, uso de uma planta, métodos para detectar e/ou selecionar plantas, conferir resistência ao tobrfv, melhorar o rendimento de tomateiros e reduzir a perda na produção de tomate e usos de um marcador |
| EP4161552A1 (en) | 2020-06-05 | 2023-04-12 | The Broad Institute, Inc. | Compositions and methods for treating neoplasia |
| EP4172340A1 (en) | 2020-06-29 | 2023-05-03 | KWS SAAT SE & Co. KGaA | Boosting homology directed repair in plants |
| CN116096378A (zh) | 2020-08-10 | 2023-05-09 | 诺华股份有限公司 | 视网膜变性疾病的治疗 |
| US20220049303A1 (en) | 2020-08-17 | 2022-02-17 | Readcoor, Llc | Methods and systems for spatial mapping of genetic variants |
| JP7830442B2 (ja) | 2020-10-02 | 2026-03-16 | ビルモラン エ コンパニー | 貯蔵寿命が延長されたメロン |
| EP4225330A1 (en) | 2020-10-06 | 2023-08-16 | Iovance Biotherapeutics, Inc. | Treatment of nsclc patients with tumor infiltrating lymphocyte therapies |
| WO2022076606A1 (en) | 2020-10-06 | 2022-04-14 | Iovance Biotherapeutics, Inc. | Treatment of nsclc patients with tumor infiltrating lymphocyte therapies |
| US20240026369A1 (en) | 2020-10-27 | 2024-01-25 | KWS SAAT SE & Co. KGaA | Use of enhanced pol theta activity for eukaryotic genome engineering |
| EP4242237A4 (en) | 2020-11-06 | 2025-01-01 | Editforce, Inc. | FOKI NUCLEASE DOMAIN VARIANT |
| EP4243608A1 (en) | 2020-11-11 | 2023-09-20 | Leibniz-Institut für Pflanzenbiochemie (IPB) | Fusion protein for editing endogenous dna of a eukaryotic cell |
| EP4001429A1 (en) | 2020-11-16 | 2022-05-25 | Antibodies-Online GmbH | Analysis of crispr-cas binding and cleavage sites followed by high-throughput sequencing (abc-seq) |
| EP4255172A1 (en) | 2020-12-03 | 2023-10-11 | Vilmorin & Cie | Tomato plants resistant to tobrfv, tmv, tomv and tommv and corresponding resistance genes |
| CA3202483A1 (en) | 2020-12-17 | 2022-06-23 | Maria Fardis | Treatment with tumor infiltrating lymphocyte therapies in combination with ctla-4 and pd-1 inhibitors |
| US20240123067A1 (en) | 2020-12-17 | 2024-04-18 | Iovance Biotherapeutics, Inc. | Treatment of cancers with tumor infiltrating lymphocyte therapies |
| EP4019639A1 (en) | 2020-12-22 | 2022-06-29 | KWS SAAT SE & Co. KGaA | Promoting regeneration and transformation in beta vulgaris |
| IL303901A (en) * | 2020-12-22 | 2023-08-01 | Ensoma Inc | Adenoviral serotype 35 helper vectors |
| KR20230124682A (ko) | 2020-12-23 | 2023-08-25 | 플래그쉽 파이어니어링 이노베이션스 브이, 인크. | Rna를 봉입하는 아넬로바이러스 캡시드의 시험관 내어셈블리 |
| KR20230146007A (ko) | 2020-12-31 | 2023-10-18 | 크리스퍼 테라퓨틱스 아게 | 범용 공여자 세포 |
| WO2022165260A1 (en) | 2021-01-29 | 2022-08-04 | Iovance Biotherapeutics, Inc. | Methods of making modified tumor infiltrating lymphocytes and their use in adoptive cell therapy |
| JP2024510505A (ja) | 2021-03-19 | 2024-03-07 | アイオバンス バイオセラピューティクス,インコーポレイテッド | Cd39/cd69選択に関連した腫瘍浸潤リンパ球(til)拡張及びtilにおける遺伝子ノックアウトのための方法 |
| WO2022204564A2 (en) | 2021-03-25 | 2022-09-29 | Iovance Biotherapeutics, Inc. | Methods and compositions for t-cell coculture potency assays and use with cell therapy products |
| US20250243244A1 (en) | 2021-03-31 | 2025-07-31 | Entrada Therapeutics, Inc. | Cyclic cell penetrating peptides |
| WO2022208489A1 (en) | 2021-04-02 | 2022-10-06 | Vilmorin & Cie | Semi-determinate or determinate growth habit trait in cucurbita |
| KR20240037185A (ko) | 2021-04-19 | 2024-03-21 | 이오반스 바이오테라퓨틱스, 인크. | 키메라 공동자극 수용체, 케모카인 수용체, 및 세포 면역치료에서의 이의 용도 |
| CA3217862A1 (en) | 2021-05-05 | 2022-11-10 | Radius Pharmaceuticals, Inc. | Animal model having homologous recombination of mouse pth1 receptor |
| EP4337261A2 (en) | 2021-05-10 | 2024-03-20 | Entrada Therapeutics, Inc. | Compositions and methods for modulating mrna splicing |
| AU2022271873A1 (en) | 2021-05-10 | 2024-01-04 | Entrada Therapeutics, Inc. | Compositions and methods for intracellular therapeutics |
| WO2022240721A1 (en) | 2021-05-10 | 2022-11-17 | Entrada Therapeutics, Inc. | Compositions and methods for modulating interferon regulatory factor-5 (irf-5) activity |
| JP2024519029A (ja) | 2021-05-17 | 2024-05-08 | アイオバンス バイオセラピューティクス,インコーポレイテッド | Pd-1遺伝子編集された腫瘍浸潤リンパ球及び免疫療法におけるその使用 |
| AU2022276078A1 (en) | 2021-05-17 | 2024-01-04 | Nogra Pharma Limited | Il-34 antisense agents and methods of using same |
| GB202107057D0 (en) | 2021-05-18 | 2021-06-30 | Univ York | Glycosylation method |
| WO2022243437A1 (en) | 2021-05-19 | 2022-11-24 | KWS SAAT SE & Co. KGaA | Sample preparation with oppositely oriented guide polynucleotides |
| EP4419672A2 (en) | 2021-06-01 | 2024-08-28 | Artisan Development Labs, Inc. | Compositions and methods for targeting, editing, or modifying genes |
| WO2022256440A2 (en) | 2021-06-01 | 2022-12-08 | Arbor Biotechnologies, Inc. | Gene editing systems comprising a crispr nuclease and uses thereof |
| EP4352131A1 (en) | 2021-06-11 | 2024-04-17 | Genedit Inc. | Biodegradable polymer comprising side chains with polyamine and polyalkylene oxide groups |
| EP4370676A2 (en) | 2021-06-18 | 2024-05-22 | Artisan Development Labs, Inc. | Compositions and methods for targeting, editing or modifying human genes |
| GEAP202416440A (en) | 2021-06-23 | 2024-04-25 | Entrada Therapeutics Inc | Antisense compounds and methods for targeting cug repeats |
| WO2023283359A2 (en) | 2021-07-07 | 2023-01-12 | Omega Therapeutics, Inc. | Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression |
| JP2024526898A (ja) | 2021-07-22 | 2024-07-19 | アイオバンス バイオセラピューティクス,インコーポレイテッド | 固形腫瘍断片の凍結保存のための方法 |
| WO2023009716A1 (en) | 2021-07-28 | 2023-02-02 | Iovance Biotherapeutics, Inc. | Treatment of cancer patients with tumor infiltrating lymphocyte therapies in combination with kras inhibitors |
| CA3221517A1 (en) | 2021-07-30 | 2023-02-02 | Monika KLOIBER-MAITZ | Plants with improved digestibility and marker haplotypes |
| MX2024001624A (es) | 2021-08-06 | 2024-04-30 | Vilmorin & Cie | Resistencia a leveillula taurica en el pimiento. |
| WO2023029532A1 (en) * | 2021-08-30 | 2023-03-09 | Huigene Therapeutics Co., Ltd. | Engineered cas6 protein and uses thereof |
| WO2023039586A1 (en) | 2021-09-10 | 2023-03-16 | Agilent Technologies, Inc. | Guide rnas with chemical modification for prime editing |
| EP4596697A3 (en) | 2021-09-28 | 2025-08-27 | Acrigen Biosciences | Compositions and methods for nucleic acid modifications |
| US12285497B2 (en) | 2021-10-15 | 2025-04-29 | Research Institute At Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| EP4166670A1 (en) | 2021-10-18 | 2023-04-19 | KWS SAAT SE & Co. KGaA | Plant-tag-based weeding control |
| CA3235824A1 (en) | 2021-10-27 | 2023-05-04 | Frederick G. Vogt | Systems and methods for coordinating manufacturing of cells for patient-specific immunotherapy |
| WO2023077148A1 (en) | 2021-11-01 | 2023-05-04 | Tome Biosciences, Inc. | Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo |
| EP4426832A1 (en) | 2021-11-03 | 2024-09-11 | The J. David Gladstone Institutes, A Testamentary Trust Established under The Will of J. David Gladstone | Precise genome editing using retrons |
| US20250032618A1 (en) | 2021-11-10 | 2025-01-30 | Iovance Biotherapeutics, Inc. | Methods of expansion treatment utilizing cd8 tumor infiltrating lymphocytes |
| US20240408236A1 (en) | 2021-11-26 | 2024-12-12 | Epigenic Therapeutics, Inc. | Method of modulating pcsk9 and uses thereof |
| GB202118058D0 (en) | 2021-12-14 | 2022-01-26 | Univ Warwick | Methods to increase yields in crops |
| US20230279442A1 (en) | 2021-12-15 | 2023-09-07 | Versitech Limited | Engineered cas9-nucleases and method of use thereof |
| US20250059239A1 (en) | 2021-12-17 | 2025-02-20 | Sana Biotechnology, Inc. | Modified paramyxoviridae fusion glycoproteins |
| WO2023115041A1 (en) | 2021-12-17 | 2023-06-22 | Sana Biotechnology, Inc. | Modified paramyxoviridae attachment glycoproteins |
| CA3243006A1 (en) | 2021-12-21 | 2025-02-27 | Alia Therapeutics Srl | TYPE II CAS PROTEINS AND THEIR APPLICATIONS |
| EP4452335A1 (en) | 2021-12-22 | 2024-10-30 | Tome Biosciences, Inc. | Co-delivery of a gene editor construct and a donor template |
| KR20240133725A (ko) | 2021-12-23 | 2024-09-04 | 유니버시티 오브 매사추세츠 | 취약 x 관련 장애의 치료용 치료제 |
| WO2023133595A2 (en) | 2022-01-10 | 2023-07-13 | Sana Biotechnology, Inc. | Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses |
| WO2023141602A2 (en) | 2022-01-21 | 2023-07-27 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
| WO2023147488A1 (en) | 2022-01-28 | 2023-08-03 | Iovance Biotherapeutics, Inc. | Cytokine associated tumor infiltrating lymphocytes compositions and methods |
| EP4472646A1 (en) | 2022-02-01 | 2024-12-11 | Sana Biotechnology, Inc. | Cd3-targeted lentiviral vectors and uses thereof |
| WO2023150647A1 (en) | 2022-02-02 | 2023-08-10 | Sana Biotechnology, Inc. | Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses |
| EP4486881A1 (en) | 2022-03-01 | 2025-01-08 | Celyntra Therapeutics SA | Composition and methods for transgene insertion |
| WO2023194359A1 (en) | 2022-04-04 | 2023-10-12 | Alia Therapeutics Srl | Compositions and methods for treatment of usher syndrome type 2a |
| EP4256950A1 (en) | 2022-04-06 | 2023-10-11 | Vilmorin et Cie | Tolerance to cgmmv in cucumber |
| CA3247638A1 (en) | 2022-04-06 | 2023-10-12 | Maria Fardis | TREATMENT OF PATIENTS WITH NON-SMALL CELL LUNG CARCINOMA (NSCLC) WITH TUMOR-INFILTRATING LYMPHOCYTE THERAPIES |
| WO2023201369A1 (en) | 2022-04-15 | 2023-10-19 | Iovance Biotherapeutics, Inc. | Til expansion processes using specific cytokine combinations and/or akti treatment |
| WO2023205744A1 (en) | 2022-04-20 | 2023-10-26 | Tome Biosciences, Inc. | Programmable gene insertion compositions |
| WO2023215831A1 (en) | 2022-05-04 | 2023-11-09 | Tome Biosciences, Inc. | Guide rna compositions for programmable gene insertion |
| WO2023219933A1 (en) | 2022-05-09 | 2023-11-16 | Entrada Therapeutics, Inc. | Compositions and methods for delivery of nucleic acid therapeutics |
| US20250281537A1 (en) | 2022-05-10 | 2025-09-11 | Iovance Biotherapeutics, Inc. | Treatment of cancer patients with tumor infiltrating lymphocyte therapies in combination with an il-15r agonist |
| US20250308637A1 (en) | 2022-05-20 | 2025-10-02 | Celyntra Therapeutics Sa | Systems and methods for assessing risk of genome editing events |
| WO2023225670A2 (en) | 2022-05-20 | 2023-11-23 | Tome Biosciences, Inc. | Ex vivo programmable gene insertion |
| WO2023230570A2 (en) | 2022-05-25 | 2023-11-30 | Flagship Pioneering Innovations Vii, Llc | Compositions and methods for modulating genetic drivers |
| CA3254044A1 (en) | 2022-05-25 | 2023-11-30 | Flagship Pioneering Innovations Vii, Llc | COMPOSITIONS AND MODULATION METHODS OF TUMOR SUPPRESSANTS AND ONCOGENS |
| CN119563038A (zh) | 2022-05-25 | 2025-03-04 | 旗舰创业创新七公司 | 用于调节免疫应答的组合物和方法 |
| EP4532768A2 (en) | 2022-05-25 | 2025-04-09 | Flagship Pioneering Innovations VII, LLC | Compositions and methods for modulating circulating factors |
| JP2025517967A (ja) | 2022-05-25 | 2025-06-12 | フラッグシップ パイオニアリング イノベーションズ セブン,エルエルシー | サイトカインを調節するための組成物及び方法 |
| KR20250044484A (ko) | 2022-06-24 | 2025-03-31 | 튠 쎄라퓨틱스, 인코포레이티드 | 표적 유전자 억제를 통해 저밀도 지단백질을 감소시키기 위한 조성물, 시스템, 및 방법 |
| WO2024005863A1 (en) | 2022-06-30 | 2024-01-04 | Inari Agriculture Technology, Inc. | Compositions, systems, and methods for genome editing |
| EP4299739A1 (en) | 2022-06-30 | 2024-01-03 | Inari Agriculture Technology, Inc. | Compositions, systems, and methods for genome editing |
| GB2621813A (en) | 2022-06-30 | 2024-02-28 | Univ Newcastle | Preventing disease recurrence in Mitochondrial replacement therapy |
| JP7152094B1 (ja) * | 2022-06-30 | 2022-10-12 | リージョナルフィッシュ株式会社 | tracrRNAユニット、及びゲノム編集方法 |
| WO2024005864A1 (en) | 2022-06-30 | 2024-01-04 | Inari Agriculture Technology, Inc. | Compositions, systems, and methods for genome editing |
| EP4299733A1 (en) | 2022-06-30 | 2024-01-03 | Inari Agriculture Technology, Inc. | Compositions, systems, and methods for genome editing |
| CN120344660A (zh) | 2022-07-18 | 2025-07-18 | 雷纳嘉德医疗管理公司 | 基因编辑组分、系统和使用方法 |
| WO2024020587A2 (en) | 2022-07-22 | 2024-01-25 | Tome Biosciences, Inc. | Pleiopluripotent stem cell programmable gene insertion |
| US20260055146A1 (en) | 2022-08-24 | 2026-02-26 | Sana Biotechnology, Inc. | Delivery of heterologous proteins |
| WO2024044723A1 (en) | 2022-08-25 | 2024-02-29 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
| US20250386788A1 (en) | 2022-08-26 | 2025-12-25 | KWS SAAT SE & Co. KGaA | Use of paired genes in hybrid breeding |
| KR20240034661A (ko) * | 2022-09-06 | 2024-03-14 | 주식회사 툴젠 | 캄필로박터 제주니 유래 Cas9의 가이드 RNA 구조변화를 통한 유전자교정 향상 시스템 |
| CA3267752A1 (en) | 2022-09-16 | 2024-03-21 | Alia Therapeutics Srl | ENQP TYPE II CAS PROTEINS AND THEIR APPLICATIONS |
| WO2024064838A1 (en) | 2022-09-21 | 2024-03-28 | Sana Biotechnology, Inc. | Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof |
| WO2024081820A1 (en) | 2022-10-13 | 2024-04-18 | Sana Biotechnology, Inc. | Viral particles targeting hematopoietic stem cells |
| EP4612277A1 (en) | 2022-11-04 | 2025-09-10 | Iovance Biotherapeutics, Inc. | Methods for tumor infiltrating lymphocyte (til) expansion related to cd39/cd103 selection |
| WO2024098024A1 (en) | 2022-11-04 | 2024-05-10 | Iovance Biotherapeutics, Inc. | Expansion of tumor infiltrating lymphocytes from liquid tumors and therapeutic uses thereof |
| WO2024102434A1 (en) | 2022-11-10 | 2024-05-16 | Senda Biosciences, Inc. | Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs |
| WO2024105162A1 (en) | 2022-11-16 | 2024-05-23 | Alia Therapeutics Srl | Type ii cas proteins and applications thereof |
| KR20250122544A (ko) | 2022-11-21 | 2025-08-13 | 이오반스 바이오테라퓨틱스, 인크. | 종양 침윤 림프구의 확장을 위한 2차원 공정 및 이를 통한 요법 |
| WO2024118836A1 (en) | 2022-11-30 | 2024-06-06 | Iovance Biotherapeutics, Inc. | Processes for production of tumor infiltrating lymphocytes with shortened rep step |
| WO2024119157A1 (en) | 2022-12-02 | 2024-06-06 | Sana Biotechnology, Inc. | Lipid particles with cofusogens and methods of producing and using the same |
| KR20250153183A (ko) | 2022-12-19 | 2025-10-24 | 카마우 테라퓨틱스, 인크. | P53 결합 단백질인 53bp1의 개선된 펩타이드 저해제 |
| WO2024138194A1 (en) | 2022-12-22 | 2024-06-27 | Tome Biosciences, Inc. | Platforms, compositions, and methods for in vivo programmable gene insertion |
| WO2024141599A1 (en) | 2022-12-29 | 2024-07-04 | Vilmorin & Cie | Tomato plants resistant to resistance-breaking tswv strains and corresponding resistance genes |
| CN115982034B (zh) * | 2022-12-30 | 2023-11-28 | 云舟生物科技(广州)股份有限公司 | 载体构建系统虚拟终端的测试方法、存储介质及电子设备 |
| JP2026502531A (ja) | 2023-01-11 | 2026-01-23 | アリア セラピューティクス エスアールエル | Ii型casタンパク質およびその適用 |
| WO2024168265A1 (en) | 2023-02-10 | 2024-08-15 | Possible Medicines Llc | Aav delivery of rna guided recombination system |
| WO2024168253A1 (en) | 2023-02-10 | 2024-08-15 | Possible Medicines Llc | Delivery of an rna guided recombination system |
| CN120712355A (zh) | 2023-02-15 | 2025-09-26 | 阿伯生物技术公司 | 用于抑制抑微管装配蛋白2(stmn2)转录物中的异常剪接的基因编辑方法 |
| WO2024175308A1 (en) | 2023-02-22 | 2024-08-29 | KWS SAAT SE & Co. KGaA | Plants with improved performance |
| US12225874B2 (en) | 2023-03-27 | 2025-02-18 | Redsea Science And Technology Inc. | Tomato plant designated ‘X22-31’ |
| WO2024206911A2 (en) | 2023-03-30 | 2024-10-03 | Children's Hospital Medical Center | Clinical-grade organoids |
| WO2024211287A1 (en) | 2023-04-03 | 2024-10-10 | Seagen Inc. | Production cell lines with targeted integration sites |
| WO2024220560A1 (en) | 2023-04-18 | 2024-10-24 | Sana Biotechnology, Inc. | Engineered protein g fusogens and related lipid particles and methods thereof |
| WO2024220598A2 (en) | 2023-04-18 | 2024-10-24 | Sana Biotechnology, Inc. | Lentiviral vectors with two or more genomes |
| WO2024220574A1 (en) | 2023-04-18 | 2024-10-24 | Sana Biotechnology, Inc. | Universal protein g fusogens and adapter systems thereof and related lipid particles and uses |
| TW202449159A (zh) | 2023-04-19 | 2024-12-16 | 美商旗艦先鋒創新有限責任(Vii)公司 | 用於產生文庫之組合物及方法 |
| CN121443140A (zh) | 2023-04-21 | 2026-01-30 | 非营利性组织佛兰芒综合大学生物技术研究所 | 用于作物产量提升的等位基因组合 |
| WO2024226499A1 (en) | 2023-04-24 | 2024-10-31 | The Broad Institute, Inc. | Compositions and methods for modifying fertility |
| WO2024223900A1 (en) | 2023-04-28 | 2024-10-31 | Vilmorin & Cie | Genetic determinants conferring improved tobrfv resistance |
| WO2024234006A1 (en) | 2023-05-11 | 2024-11-14 | Tome Biosciences, Inc. | Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs) |
| IL324634A (en) | 2023-05-15 | 2026-01-01 | Nchroma Bio Inc | Compositions and methods for epigenetic regulation of hbv gene expression |
| EP4713433A2 (en) | 2023-05-18 | 2026-03-25 | Children's Hospital Medical Center | Liver organoids with intrahepatic sympathetic nerves, and methods of use thereof |
| WO2024236547A1 (en) | 2023-05-18 | 2024-11-21 | Inceptor Bio, Llc | Modified phagocytic cells expressing chimeric antigen receptors comprising a herpes virus entry mediator (hvem) co-stimulatory domain and uses thereof |
| WO2024243438A2 (en) | 2023-05-23 | 2024-11-28 | Omega Therapeutics, Inc. | Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression |
| AU2024274792A1 (en) | 2023-05-23 | 2025-10-23 | Sana Biotechnology, Inc. | Tandem fusogens and related lipid particles |
| WO2024253957A1 (en) | 2023-06-07 | 2024-12-12 | Northwestern University | Targeting autoimmunity-associated t cells through regulation of transcription factors |
| EP4731750A2 (en) | 2023-06-23 | 2026-04-29 | Children's Hospital Medical Center | Methods of matrix-free suspension culture |
| WO2025006419A1 (en) | 2023-06-26 | 2025-01-02 | Flagship Pioneering Innovations Vii, Llc | Engineered plasmodia and related methods |
| EP4734755A1 (en) | 2023-06-28 | 2026-05-06 | Vilmorin & Cie | Pepper resistance to meloidogyne enterolobii |
| CN121548346A (zh) | 2023-07-24 | 2026-02-17 | 维尔莫林公司 | 甜瓜对cysdv的抗性 |
| IL326289A (en) | 2023-08-01 | 2026-04-01 | Kws Saat Se & Co Kgaa | A method for reducing off-flavors in alternative proteins |
| WO2025027133A1 (en) | 2023-08-01 | 2025-02-06 | KWS SAAT SE & Co. KGaA | Method of improving gelation properties of alternative proteins |
| WO2025046513A1 (en) | 2023-08-29 | 2025-03-06 | Inceptor Bio, Llc | Methods of manufacturing myeloid-derived cells from hematopoietic stem cells and compositions and uses thereof |
| WO2025050069A1 (en) | 2023-09-01 | 2025-03-06 | Tome Biosciences, Inc. | Programmable gene insertion using engineered integration enzymes |
| WO2025049959A2 (en) | 2023-09-01 | 2025-03-06 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
| WO2025054202A1 (en) | 2023-09-05 | 2025-03-13 | Sana Biotechnology, Inc. | Method of screening a sample comprising a transgene with a unique barcode |
| WO2025054540A1 (en) | 2023-09-08 | 2025-03-13 | Iovance Biotherapeutics, Inc. | Methods of gene-editing using programmable nucleases |
| WO2025064408A1 (en) | 2023-09-18 | 2025-03-27 | The Broad Institute, Inc. | Compositions and methods for treating cardiovascular disease |
| GB202314578D0 (en) | 2023-09-22 | 2023-11-08 | Univ Manchester | Methods of producing homoplasmic modified plants or parts thereof |
| WO2025072803A1 (en) | 2023-09-29 | 2025-04-03 | Children's Hospital Medical Center | Ntrk2 signaling-mediated alveolar capillary injury and repair |
| EP4554371A4 (en) | 2023-10-03 | 2026-04-22 | Inari Agriculture Tech Inc | GRNA VIRAL ADMINISTRATION IN GRAFTS |
| AU2024357861A1 (en) | 2023-10-12 | 2026-04-23 | Renagade Therapeutics Management Inc. | Nickase-retron template-based precision editing system and methods of use |
| WO2025101484A1 (en) | 2023-11-06 | 2025-05-15 | Iovance Biotherapeutics, Inc. | Treatment of endometrial cancers with tumor infiltrating lymphocyte therapies |
| WO2025132866A1 (en) | 2023-12-21 | 2025-06-26 | KWS SAAT SE & Co. KGaA | Maize plants with improved disease resistance |
| WO2025151838A1 (en) | 2024-01-12 | 2025-07-17 | Sana Biotechnology, Inc. | Safety switches to control in vitro and in vivo proliferation of cell therapy products |
| WO2025155753A2 (en) | 2024-01-17 | 2025-07-24 | Renagade Therapeutics Management Inc. | Improved gene editing system, guides, and methods |
| WO2025153582A1 (en) | 2024-01-17 | 2025-07-24 | KWS SAAT SE & Co. KGaA | Maize plants with improved disease resistance |
| WO2025168705A1 (en) | 2024-02-08 | 2025-08-14 | Vib Vzw | Means and methods for the production of saponins with endosomal escape-enhancing properties |
| WO2025171210A1 (en) | 2024-02-09 | 2025-08-14 | Arbor Biotechnologies, Inc. | Compositions and methods for gene editing via homology-mediated end joining |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025184529A1 (en) | 2024-03-01 | 2025-09-04 | Sana Biotechnology, Inc. | Viral particles with fusogen display and related compositions and methods |
| WO2025186284A1 (en) | 2024-03-05 | 2025-09-12 | Vilmorin & Cie | Increased yield in cucumber |
| WO2025196502A1 (en) | 2024-03-20 | 2025-09-25 | North Carolina Agricultural & Technical State University | Choline kinase inhibitors as a therapeutic treatment for obesity |
| WO2025212920A1 (en) | 2024-04-03 | 2025-10-09 | Children's Hospital Medical Center | Multi-zonal liver organoids |
| WO2025217202A1 (en) | 2024-04-08 | 2025-10-16 | Children's Hospital Medical Center | Bile duct organoid |
| US20250332260A1 (en) | 2024-04-10 | 2025-10-30 | Garuda Therapeutics, Inc. | Immune compatible cells for allogeneic cell therapies to cover global, ethnic, or disease-specific populations |
| EP4677108A1 (en) | 2024-04-22 | 2026-01-14 | Basecamp Research Ltd | Method and compositions for detecting off-target editing |
| WO2025224182A2 (en) | 2024-04-23 | 2025-10-30 | Basecamp Research Ltd | Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo |
| WO2025240947A1 (en) | 2024-05-17 | 2025-11-20 | University Of Massachusetts | Therapeutic treatment for fragile x-associated disorder |
| WO2025259610A1 (en) | 2024-06-10 | 2025-12-18 | Northwestern University | Genetic modulators of kras protein expression and their uses |
| WO2025259554A1 (en) | 2024-06-13 | 2025-12-18 | J.R. Simplot Company | Engineering remontant flowering in rosaceae |
| WO2026006542A2 (en) | 2024-06-26 | 2026-01-02 | Yale University | Compositions and methods for crispr/cas9 based reactivation of human angelman syndrome |
| WO2026015829A2 (en) | 2024-07-12 | 2026-01-15 | Arbor Biotechnologies, Inc. | Small reverse transcriptases and gene editing systems comprising such |
| WO2026015832A2 (en) | 2024-07-12 | 2026-01-15 | Arbor Biotechnologies, Inc. | Reverse transcriptases and gene editing systems comprising such |
| WO2026017833A1 (en) | 2024-07-17 | 2026-01-22 | Vilmorin & Cie | Tswv-resistant pepper |
| EP4686414A1 (en) | 2024-08-01 | 2026-02-04 | KWS SAAT SE & Co. KGaA | Method of improving gelation properties of pea proteins |
| FR3165152A3 (fr) | 2024-08-01 | 2026-02-06 | KWS SAAT SE & Co. KGaA | Lignées de pois au profil gustatif amélioré |
| FR3165153A3 (fr) | 2024-08-01 | 2026-02-06 | KWS SAAT SE & Co. KGaA | Lignées de pois au profil gustatif amélioré des ingrédients à base de pois |
| WO2026036000A1 (en) | 2024-08-08 | 2026-02-12 | Invaio Sciences, Inc. | Nature-derived lipid particles for use in agriculture |
| WO2026047626A1 (en) | 2024-08-30 | 2026-03-05 | Inceptor Bio, Llc | Compositions and methods involving immune cells engineered for binding bispecific antibodies for use in cell therapy |
| WO2026060399A2 (en) | 2024-09-16 | 2026-03-19 | Northwestern University | T cell lymphoma mutations, compositions, and uses to enhance engineered t cell therapies |
| WO2026060398A2 (en) | 2024-09-16 | 2026-03-19 | Northwestern University | B cell lymphoma mutations, compositions, and uses to enhance engineered t cell therapies |
| WO2026062237A1 (en) | 2024-09-20 | 2026-03-26 | KWS SAAT SE & Co. KGaA | Improved color-coded male sterile system for targeted selection of cereal plants |
| WO2026096383A1 (en) | 2024-10-28 | 2026-05-07 | The Broad Institute, Inc. | Compositions and methods for inducing phagocytosis |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2009136452A (ru) * | 2007-03-02 | 2011-04-10 | Даниско А/С (Dk) | Культуры с повышенной устойчивостью к фагам |
Family Cites Families (49)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4217344A (en) | 1976-06-23 | 1980-08-12 | L'oreal | Compositions containing aqueous dispersions of lipid spheres |
| US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
| US4186183A (en) | 1978-03-29 | 1980-01-29 | The United States Of America As Represented By The Secretary Of The Army | Liposome carriers in chemotherapy of leishmaniasis |
| US4261975A (en) | 1979-09-19 | 1981-04-14 | Merck & Co., Inc. | Viral liposome particle |
| US4485054A (en) | 1982-10-04 | 1984-11-27 | Lipoderm Pharmaceuticals Limited | Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV) |
| US4501728A (en) | 1983-01-06 | 1985-02-26 | Technology Unlimited, Inc. | Masking of liposomes from RES recognition |
| US4946787A (en) | 1985-01-07 | 1990-08-07 | Syntex (U.S.A.) Inc. | N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US5049386A (en) | 1985-01-07 | 1991-09-17 | Syntex (U.S.A.) Inc. | N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| US4774085A (en) | 1985-07-09 | 1988-09-27 | 501 Board of Regents, Univ. of Texas | Pharmaceutical administration systems containing a mixture of immunomodulators |
| ATE141646T1 (de) | 1986-04-09 | 1996-09-15 | Genzyme Corp | Genetisch transformierte tiere, die ein gewünschtes protein in milch absondern |
| US4837028A (en) | 1986-12-24 | 1989-06-06 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
| US4873316A (en) | 1987-06-23 | 1989-10-10 | Biogen, Inc. | Isolation of exogenous recombinant proteins from the milk of transgenic mammals |
| US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
| AU7979491A (en) | 1990-05-03 | 1991-11-27 | Vical, Inc. | Intracellular delivery of biologically active substances by means of self-assembling lipid complexes |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| US7150982B2 (en) * | 1991-09-09 | 2006-12-19 | Third Wave Technologies, Inc. | RNA detection assays |
| US5587308A (en) | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US7868149B2 (en) | 1999-07-20 | 2011-01-11 | Monsanto Technology Llc | Plant genome sequence and uses thereof |
| US6603061B1 (en) | 1999-07-29 | 2003-08-05 | Monsanto Company | Agrobacterium-mediated plant transformation method |
| JP3454818B1 (ja) * | 2001-03-16 | 2003-10-06 | 直哉 小林 | 肝臓細胞の増殖方法、該方法により得られる肝臓細胞、およびその用途 |
| US20090100536A1 (en) | 2001-12-04 | 2009-04-16 | Monsanto Company | Transgenic plants with enhanced agronomic traits |
| WO2007014275A2 (en) * | 2005-07-26 | 2007-02-01 | Sangamo Biosciences, Inc. | Targeted integration and expression of exogenous nucleic acid sequences |
| US9677123B2 (en) * | 2006-03-15 | 2017-06-13 | Siemens Healthcare Diagnostics Inc. | Degenerate nucleobase analogs |
| WO2010011961A2 (en) | 2008-07-25 | 2010-01-28 | University Of Georgia Research Foundation, Inc. | Prokaryotic rnai-like system and methods of use |
| US20100076057A1 (en) * | 2008-09-23 | 2010-03-25 | Northwestern University | TARGET DNA INTERFERENCE WITH crRNA |
| US9404098B2 (en) | 2008-11-06 | 2016-08-02 | University Of Georgia Research Foundation, Inc. | Method for cleaving a target RNA using a Cas6 polypeptide |
| AU2009322964B2 (en) * | 2008-12-04 | 2014-10-09 | Sangamo Therapeutics, Inc. | Genome editing in rats using zinc-finger nucleases |
| US8889394B2 (en) | 2009-09-07 | 2014-11-18 | Empire Technology Development Llc | Multiple domain proteins |
| DE112010004582T5 (de) * | 2009-11-27 | 2012-11-29 | Basf Plant Science Company Gmbh | Optimierte Endonukleasen und Anwendungen davon |
| US10087431B2 (en) | 2010-03-10 | 2018-10-02 | The Regents Of The University Of California | Methods of generating nucleic acid fragments |
| EA024121B9 (ru) | 2010-05-10 | 2017-01-30 | Дзе Реджентс Ов Дзе Юниверсити Ов Калифорния | Композиции эндорибонуклеаз и способы их использования |
| EP3156062A1 (en) * | 2010-05-17 | 2017-04-19 | Sangamo BioSciences, Inc. | Novel dna-binding proteins and uses thereof |
| US9405700B2 (en) | 2010-11-04 | 2016-08-02 | Sonics, Inc. | Methods and apparatus for virtualization in an integrated circuit |
| WO2012149470A1 (en) * | 2011-04-27 | 2012-11-01 | Amyris, Inc. | Methods for genomic modification |
| WO2012164565A1 (en) | 2011-06-01 | 2012-12-06 | Yeda Research And Development Co. Ltd. | Compositions and methods for downregulating prokaryotic genes |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| WO2013141680A1 (en) | 2012-03-20 | 2013-09-26 | Vilnius University | RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX |
| US9637739B2 (en) | 2012-03-20 | 2017-05-02 | Vilnius University | RNA-directed DNA cleavage by the Cas9-crRNA complex |
| LT3401400T (lt) * | 2012-05-25 | 2019-06-10 | The Regents Of The University Of California | Būdai ir kompozicijos, skirtos rnr molekulės nukreipiamai tikslinės dnr modifikacijai ir rnr molekulės nukreipiamam transkripcijos moduliavimui |
| PL4397760T3 (pl) * | 2012-10-23 | 2026-03-30 | Toolgen Incorporated | Kompozycja do rozszczepiania docelowego dna zawierająca prowadzące rna specyficzne dla docelowego dna i kwas nukleinowy kodujący białko cas lub białko cas oraz jej zastosowanie |
| EP3135765A1 (en) * | 2012-12-06 | 2017-03-01 | Sigma-Aldrich Co. LLC | Crispr-based genome modification and regulation |
| WO2014093479A1 (en) | 2012-12-11 | 2014-06-19 | Montana State University | Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| JP2016504026A (ja) | 2012-12-12 | 2016-02-12 | ザ・ブロード・インスティテュート・インコーポレイテッド | 配列操作のための系、方法および最適化ガイド組成物のエンジニアリング |
| SG10201912991WA (en) | 2012-12-17 | 2020-03-30 | Harvard College | Rna-guided human genome engineering |
-
2013
- 2013-12-12 JP JP2015547573A patent/JP2016504026A/ja active Pending
- 2013-12-12 ES ES13818570.7T patent/ES2536353T3/es active Active
- 2013-12-12 WO PCT/US2013/074819 patent/WO2014093712A1/en not_active Ceased
- 2013-12-12 AU AU2013359123A patent/AU2013359123B2/en active Active
- 2013-12-12 PT PT151545399T patent/PT2896697E/pt unknown
- 2013-12-12 PL PL13818570T patent/PL2771468T3/pl unknown
- 2013-12-12 DK DK13818570.7T patent/DK2771468T3/en active
- 2013-12-12 CN CN202110406914.8A patent/CN113528577B/zh active Active
- 2013-12-12 ES ES14170383.5T patent/ES2542015T3/es active Active
- 2013-12-12 IL IL239344A patent/IL239344B2/en unknown
- 2013-12-12 EP EP13818570.7A patent/EP2771468B1/en not_active Revoked
- 2013-12-12 CN CN202411628310.8A patent/CN119752887A/zh active Pending
- 2013-12-12 PT PT138185707T patent/PT2771468E/pt unknown
- 2013-12-12 MX MX2015007549A patent/MX380562B/es unknown
- 2013-12-12 CA CA2894701A patent/CA2894701A1/en not_active Abandoned
- 2013-12-12 RU RU2015128098A patent/RU2701850C2/ru active
- 2013-12-12 EP EP25158824.0A patent/EP4545636A3/en active Pending
- 2013-12-12 ES ES15154566.2T patent/ES2598115T3/es active Active
- 2013-12-12 ES ES15154539.9T patent/ES2553782T3/es active Active
- 2013-12-12 PL PL15154566T patent/PL2921557T3/pl unknown
- 2013-12-12 PT PT141703835T patent/PT2784162E/pt unknown
- 2013-12-12 KR KR1020157018614A patent/KR20150105633A/ko not_active Ceased
- 2013-12-12 IL IL307735A patent/IL307735A/en unknown
- 2013-12-12 US US14/104,990 patent/US20140242664A1/en not_active Abandoned
- 2013-12-12 PT PT151545662T patent/PT2921557T/pt unknown
- 2013-12-12 EP EP23193042.1A patent/EP4279588A3/en active Pending
- 2013-12-12 CN CN201380070567.XA patent/CN105121648B/zh active Active
-
2014
- 2014-05-29 US US14/290,575 patent/US8906616B2/en active Active
-
2015
- 2015-05-04 US US14/703,511 patent/US20150232882A1/en active Pending
- 2015-05-05 US US14/704,551 patent/US20150247150A1/en active Pending
- 2015-11-09 HK HK15111023.1A patent/HK1210221A1/en unknown
-
2016
- 2016-02-15 JP JP2016025710A patent/JP6203879B2/ja active Active
- 2016-03-30 JP JP2016067687A patent/JP6420273B2/ja active Active
- 2016-06-14 JP JP2016117740A patent/JP2016165307A/ja active Pending
- 2016-08-05 US US15/230,025 patent/US20160340662A1/en active Pending
- 2016-10-12 AU AU2016244241A patent/AU2016244241C1/en active Active
-
2017
- 2017-08-30 JP JP2017165304A patent/JP6495395B2/ja active Active
-
2018
- 2018-03-20 JP JP2018053045A patent/JP6726225B2/ja active Active
-
2019
- 2019-03-05 JP JP2019039723A patent/JP6960950B2/ja active Active
- 2019-03-05 JP JP2019039724A patent/JP6960951B2/ja active Active
- 2019-09-13 AU AU2019229420A patent/AU2019229420B2/en active Active
-
2021
- 2021-10-12 JP JP2021167368A patent/JP7198328B2/ja active Active
-
2022
- 2022-06-01 AU AU2022203762A patent/AU2022203762B2/en active Active
- 2022-12-16 JP JP2022200815A patent/JP7542595B2/ja active Active
-
2024
- 2024-08-20 JP JP2024138375A patent/JP7847619B2/ja active Active
-
2025
- 2025-01-16 US US19/025,692 patent/US20250250553A1/en active Pending
- 2025-05-22 AU AU2025203780A patent/AU2025203780A1/en not_active Withdrawn
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2009136452A (ru) * | 2007-03-02 | 2011-04-10 | Даниско А/С (Dk) | Культуры с повышенной устойчивостью к фагам |
Non-Patent Citations (1)
| Title |
|---|
| Dana Carrall et al. A CRISPR Approach to Gene Targeting, Mol. Ther., 2012 sep. 4; 20(9): 1658-1660. * |
Cited By (57)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12006520B2 (en) | 2011-07-22 | 2024-06-11 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US10954548B2 (en) | 2013-08-09 | 2021-03-23 | President And Fellows Of Harvard College | Nuclease profiling system |
| US11920181B2 (en) | 2013-08-09 | 2024-03-05 | President And Fellows Of Harvard College | Nuclease profiling system |
| US11046948B2 (en) | 2013-08-22 | 2021-06-29 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US10858639B2 (en) | 2013-09-06 | 2020-12-08 | President And Fellows Of Harvard College | CAS9 variants and uses thereof |
| US10912833B2 (en) | 2013-09-06 | 2021-02-09 | President And Fellows Of Harvard College | Delivery of negatively charged proteins using cationic lipids |
| US10682410B2 (en) | 2013-09-06 | 2020-06-16 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US10597679B2 (en) | 2013-09-06 | 2020-03-24 | President And Fellows Of Harvard College | Switchable Cas9 nucleases and uses thereof |
| US12584118B2 (en) | 2013-09-06 | 2026-03-24 | President And Fellows Of Harvard College | Cas9 variants and uses thereof |
| US11299755B2 (en) | 2013-09-06 | 2022-04-12 | President And Fellows Of Harvard College | Switchable CAS9 nucleases and uses thereof |
| US12473573B2 (en) | 2013-09-06 | 2025-11-18 | President And Fellows Of Harvard College | Switchable Cas9 nucleases and uses thereof |
| US12215365B2 (en) | 2013-12-12 | 2025-02-04 | President And Fellows Of Harvard College | Cas variants for gene editing |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| US11124782B2 (en) | 2013-12-12 | 2021-09-21 | President And Fellows Of Harvard College | Cas variants for gene editing |
| US10704062B2 (en) | 2014-07-30 | 2020-07-07 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| US11578343B2 (en) | 2014-07-30 | 2023-02-14 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| US12398406B2 (en) | 2014-07-30 | 2025-08-26 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| US12043852B2 (en) | 2015-10-23 | 2024-07-23 | President And Fellows Of Harvard College | Evolved Cas9 proteins for gene editing |
| US11214780B2 (en) | 2015-10-23 | 2022-01-04 | President And Fellows Of Harvard College | Nucleobase editors and uses thereof |
| US11702651B2 (en) | 2016-08-03 | 2023-07-18 | President And Fellows Of Harvard College | Adenosine nucleobase editors and uses thereof |
| US10947530B2 (en) | 2016-08-03 | 2021-03-16 | President And Fellows Of Harvard College | Adenosine nucleobase editors and uses thereof |
| US11999947B2 (en) | 2016-08-03 | 2024-06-04 | President And Fellows Of Harvard College | Adenosine nucleobase editors and uses thereof |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| US12084663B2 (en) | 2016-08-24 | 2024-09-10 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| US11306324B2 (en) | 2016-10-14 | 2022-04-19 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| US11820969B2 (en) | 2016-12-23 | 2023-11-21 | President And Fellows Of Harvard College | Editing of CCR2 receptor gene to protect against HIV infection |
| US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| US12516308B2 (en) | 2017-03-09 | 2026-01-06 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| US11542496B2 (en) | 2017-03-10 | 2023-01-03 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| US12435331B2 (en) | 2017-03-10 | 2025-10-07 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| US11268082B2 (en) | 2017-03-23 | 2022-03-08 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable DNA binding proteins |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| US12359218B2 (en) | 2017-07-28 | 2025-07-15 | President And Fellows Of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE) |
| US11732274B2 (en) | 2017-07-28 | 2023-08-22 | President And Fellows Of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE) |
| US11932884B2 (en) | 2017-08-30 | 2024-03-19 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
| US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
| US11795443B2 (en) | 2017-10-16 | 2023-10-24 | The Broad Institute, Inc. | Uses of adenosine base editors |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| US12281303B2 (en) | 2019-03-19 | 2025-04-22 | The Broad Institute, Inc. | Methods and compositions for prime editing nucleotide sequences |
| US11447770B1 (en) | 2019-03-19 | 2022-09-20 | The Broad Institute, Inc. | Methods and compositions for prime editing nucleotide sequences |
| US11643652B2 (en) | 2019-03-19 | 2023-05-09 | The Broad Institute, Inc. | Methods and compositions for prime editing nucleotide sequences |
| US12509680B2 (en) | 2019-03-19 | 2025-12-30 | The Broad Institute, Inc. | Methods and compositions for prime editing nucleotide sequences |
| US12570972B2 (en) | 2019-03-19 | 2026-03-10 | The Broad Institute, Inc. | Methods and compositions for prime editing nucleotide sequences |
| US11795452B2 (en) | 2019-03-19 | 2023-10-24 | The Broad Institute, Inc. | Methods and compositions for prime editing nucleotide sequences |
| US12624354B2 (en) | 2019-03-19 | 2026-05-12 | The Broad Institute, Inc. | Methods and compositions for prime editing nucleotide sequences |
| US12624353B2 (en) | 2019-03-19 | 2026-05-12 | The Broad Institute, Inc. | Methods and compositions for prime editing nucleotide sequences |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| US12031126B2 (en) | 2020-05-08 | 2024-07-09 | The Broad Institute, Inc. | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| US11912985B2 (en) | 2020-05-08 | 2024-02-27 | The Broad Institute, Inc. | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7542595B2 (ja) | 配列操作のための系、方法および最適化ガイド組成物のエンジニアリング | |
| JP7463436B2 (ja) | 遺伝子産物の発現を変更するためのCRISPR-Cas系および方法 | |
| JP7542681B2 (ja) | 配列操作のためのCRISPR-Cas成分系、方法および組成物 | |
| DK2921557T3 (en) | Design of systems, methods and optimized sequence manipulation guide compositions | |
| RU2701662C2 (ru) | Компоненты системы crispr-cas, способы и композиции для манипуляции с последовательностями | |
| HK1216759B (zh) | 用於序列操纵的系统、方法和优化的指导组合物的工程化 |