KR20200038236A - 큐론을 포함하는 조성물 및 그의 용도 - Google Patents
큐론을 포함하는 조성물 및 그의 용도 Download PDFInfo
- Publication number
- KR20200038236A KR20200038236A KR1020207000972A KR20207000972A KR20200038236A KR 20200038236 A KR20200038236 A KR 20200038236A KR 1020207000972 A KR1020207000972 A KR 1020207000972A KR 20207000972 A KR20207000972 A KR 20207000972A KR 20200038236 A KR20200038236 A KR 20200038236A
- Authority
- KR
- South Korea
- Prior art keywords
- curon
- sequence
- nucleic acid
- synthetic
- acid sequence
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Ceased
Links
- 239000000203 mixture Substances 0.000 title claims description 90
- 239000008194 pharmaceutical composition Substances 0.000 claims abstract description 64
- 150000007523 nucleic acids Chemical group 0.000 claims description 606
- 108090000623 proteins and genes Proteins 0.000 claims description 390
- 230000002068 genetic effect Effects 0.000 claims description 386
- 239000002773 nucleotide Substances 0.000 claims description 363
- 125000003729 nucleotide group Chemical group 0.000 claims description 360
- 241001339993 Anelloviridae Species 0.000 claims description 316
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 292
- 102000004169 proteins and genes Human genes 0.000 claims description 289
- 102000039446 nucleic acids Human genes 0.000 claims description 255
- 108020004707 nucleic acids Proteins 0.000 claims description 255
- 210000004027 cell Anatomy 0.000 claims description 241
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 198
- 230000027455 binding Effects 0.000 claims description 144
- 239000012636 effector Substances 0.000 claims description 133
- 238000000034 method Methods 0.000 claims description 117
- 241000700605 Viruses Species 0.000 claims description 108
- 102100024407 Jouberin Human genes 0.000 claims description 107
- 101000833492 Homo sapiens Jouberin Proteins 0.000 claims description 106
- 101000651236 Homo sapiens NCK-interacting protein with SH3 domain Proteins 0.000 claims description 106
- 101710159752 Poly(3-hydroxyalkanoate) polymerase subunit PhaE Proteins 0.000 claims description 100
- 101710130262 Probable Vpr-like protein Proteins 0.000 claims description 100
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 87
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 74
- 108020004414 DNA Proteins 0.000 claims description 72
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 64
- 239000012634 fragment Substances 0.000 claims description 63
- 230000001717 pathogenic effect Effects 0.000 claims description 61
- 230000001105 regulatory effect Effects 0.000 claims description 61
- 230000014509 gene expression Effects 0.000 claims description 48
- 108091026898 Leader sequence (mRNA) Proteins 0.000 claims description 46
- 201000010099 disease Diseases 0.000 claims description 45
- 210000003527 eukaryotic cell Anatomy 0.000 claims description 39
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 38
- 150000001413 amino acids Chemical class 0.000 claims description 38
- 239000003814 drug Substances 0.000 claims description 31
- 229920001184 polypeptide Polymers 0.000 claims description 31
- 108020005004 Guide RNA Proteins 0.000 claims description 29
- 239000002245 particle Substances 0.000 claims description 28
- 230000008685 targeting Effects 0.000 claims description 27
- 108091033409 CRISPR Proteins 0.000 claims description 26
- 108020004459 Small interfering RNA Proteins 0.000 claims description 24
- 230000002163 immunogen Effects 0.000 claims description 22
- 239000003112 inhibitor Substances 0.000 claims description 21
- 206010028980 Neoplasm Diseases 0.000 claims description 19
- 208000035475 disorder Diseases 0.000 claims description 19
- 208000015181 infectious disease Diseases 0.000 claims description 19
- 108020004999 messenger RNA Proteins 0.000 claims description 19
- -1 for example Proteins 0.000 claims description 18
- 108090000790 Enzymes Proteins 0.000 claims description 16
- 102000004190 Enzymes Human genes 0.000 claims description 16
- 238000004519 manufacturing process Methods 0.000 claims description 16
- 239000000356 contaminant Substances 0.000 claims description 15
- 230000028993 immune response Effects 0.000 claims description 15
- 229940124597 therapeutic agent Drugs 0.000 claims description 15
- 230000001225 therapeutic effect Effects 0.000 claims description 15
- 150000003384 small molecules Chemical class 0.000 claims description 14
- 210000005260 human cell Anatomy 0.000 claims description 13
- 101710163270 Nuclease Proteins 0.000 claims description 12
- 239000003446 ligand Substances 0.000 claims description 12
- 239000002679 microRNA Substances 0.000 claims description 12
- 230000015572 biosynthetic process Effects 0.000 claims description 11
- 230000015556 catabolic process Effects 0.000 claims description 11
- 238000006731 degradation reaction Methods 0.000 claims description 11
- 238000003753 real-time PCR Methods 0.000 claims description 11
- 108091070501 miRNA Proteins 0.000 claims description 10
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 10
- 239000011541 reaction mixture Substances 0.000 claims description 10
- 238000003786 synthesis reaction Methods 0.000 claims description 10
- 239000000427 antigen Substances 0.000 claims description 9
- 108091007433 antigens Proteins 0.000 claims description 9
- 102000036639 antigens Human genes 0.000 claims description 9
- 210000004962 mammalian cell Anatomy 0.000 claims description 9
- 239000000556 agonist Substances 0.000 claims description 8
- 201000011510 cancer Diseases 0.000 claims description 8
- 102000005962 receptors Human genes 0.000 claims description 8
- 108020003175 receptors Proteins 0.000 claims description 8
- 239000000232 Lipid Bilayer Substances 0.000 claims description 7
- 238000003556 assay Methods 0.000 claims description 7
- 231100000433 cytotoxic Toxicity 0.000 claims description 7
- 230000001472 cytotoxic effect Effects 0.000 claims description 7
- 230000014616 translation Effects 0.000 claims description 7
- 239000005557 antagonist Substances 0.000 claims description 6
- 230000001973 epigenetic effect Effects 0.000 claims description 6
- 230000003362 replicative effect Effects 0.000 claims description 6
- 108020005544 Antisense RNA Proteins 0.000 claims description 5
- 208000023275 Autoimmune disease Diseases 0.000 claims description 5
- 208000035473 Communicable disease Diseases 0.000 claims description 5
- 208000018522 Gastrointestinal disease Diseases 0.000 claims description 5
- 206010058467 Lung neoplasm malignant Diseases 0.000 claims description 5
- 208000029265 Type 1 interferonopathy Diseases 0.000 claims description 5
- 239000003184 complementary RNA Substances 0.000 claims description 5
- 239000012642 immune effector Substances 0.000 claims description 5
- 208000026278 immune system disease Diseases 0.000 claims description 5
- 229940121354 immunomodulator Drugs 0.000 claims description 5
- 208000027866 inflammatory disease Diseases 0.000 claims description 5
- 201000005202 lung cancer Diseases 0.000 claims description 5
- 208000020816 lung neoplasm Diseases 0.000 claims description 5
- 108700042778 Antimicrobial Peptides Proteins 0.000 claims description 4
- 102000044503 Antimicrobial Peptides Human genes 0.000 claims description 4
- 108091023040 Transcription factor Proteins 0.000 claims description 4
- 102000040945 Transcription factor Human genes 0.000 claims description 4
- 239000012190 activator Substances 0.000 claims description 4
- 239000003937 drug carrier Substances 0.000 claims description 4
- 210000002443 helper t lymphocyte Anatomy 0.000 claims description 4
- 210000004072 lung Anatomy 0.000 claims description 4
- 108020005497 Nuclear hormone receptor Proteins 0.000 claims description 3
- 102000007399 Nuclear hormone receptor Human genes 0.000 claims description 3
- 230000006907 apoptotic process Effects 0.000 claims description 3
- 125000002619 bicyclic group Chemical group 0.000 claims description 3
- 238000006243 chemical reaction Methods 0.000 claims description 3
- 230000002860 competitive effect Effects 0.000 claims description 3
- 238000005538 encapsulation Methods 0.000 claims description 3
- 210000002919 epithelial cell Anatomy 0.000 claims description 3
- 210000002865 immune cell Anatomy 0.000 claims description 3
- 239000000411 inducer Substances 0.000 claims description 3
- 230000002147 killing effect Effects 0.000 claims description 3
- 210000005229 liver cell Anatomy 0.000 claims description 3
- 230000002101 lytic effect Effects 0.000 claims description 3
- 239000003607 modifier Substances 0.000 claims description 3
- 230000017074 necrotic cell death Effects 0.000 claims description 3
- 108020004017 nuclear receptors Proteins 0.000 claims description 3
- 238000001243 protein synthesis Methods 0.000 claims description 3
- 230000004044 response Effects 0.000 claims description 3
- 230000035945 sensitivity Effects 0.000 claims description 3
- 208000024891 symptom Diseases 0.000 claims description 3
- 108091092724 Noncoding DNA Proteins 0.000 claims description 2
- 230000002411 adverse Effects 0.000 claims description 2
- 230000030833 cell death Effects 0.000 claims description 2
- 230000036755 cellular response Effects 0.000 claims description 2
- 230000007541 cellular toxicity Effects 0.000 claims description 2
- 238000004128 high performance liquid chromatography Methods 0.000 claims description 2
- 239000000138 intercalating agent Substances 0.000 claims description 2
- 210000002540 macrophage Anatomy 0.000 claims description 2
- 210000000822 natural killer cell Anatomy 0.000 claims description 2
- 238000000424 optical density measurement Methods 0.000 claims description 2
- 108091006082 receptor inhibitors Proteins 0.000 claims description 2
- 230000002194 synthesizing effect Effects 0.000 claims description 2
- 231100000419 toxicity Toxicity 0.000 claims description 2
- 238000010354 CRISPR gene editing Methods 0.000 claims 1
- 108020005198 Long Noncoding RNA Proteins 0.000 claims 1
- OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical compound [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 claims 1
- 230000036039 immunity Effects 0.000 claims 1
- 229910052698 phosphorus Inorganic materials 0.000 claims 1
- 239000011574 phosphorus Substances 0.000 claims 1
- 238000009472 formulation Methods 0.000 abstract description 5
- 235000018102 proteins Nutrition 0.000 description 278
- 235000001014 amino acid Nutrition 0.000 description 67
- 229940024606 amino acid Drugs 0.000 description 66
- 241000960387 Torque teno virus Species 0.000 description 48
- 108700011259 MicroRNAs Proteins 0.000 description 39
- 239000000047 product Substances 0.000 description 33
- 108090000565 Capsid Proteins Proteins 0.000 description 32
- 102100023321 Ceruloplasmin Human genes 0.000 description 32
- 102000053602 DNA Human genes 0.000 description 32
- 210000001519 tissue Anatomy 0.000 description 30
- GOJUJUVQIVIZAV-UHFFFAOYSA-N 2-amino-4,6-dichloropyrimidine-5-carbaldehyde Chemical group NC1=NC(Cl)=C(C=O)C(Cl)=N1 GOJUJUVQIVIZAV-UHFFFAOYSA-N 0.000 description 28
- 241000332807 TTV-like mini virus Species 0.000 description 28
- 239000004055 small Interfering RNA Substances 0.000 description 28
- 108020004682 Single-Stranded DNA Proteins 0.000 description 26
- 102000040430 polynucleotide Human genes 0.000 description 26
- 108091033319 polynucleotide Proteins 0.000 description 26
- 239000002157 polynucleotide Substances 0.000 description 26
- 230000003612 virological effect Effects 0.000 description 24
- 239000002105 nanoparticle Substances 0.000 description 22
- 239000013598 vector Substances 0.000 description 22
- 239000003795 chemical substances by application Substances 0.000 description 21
- 150000002632 lipids Chemical class 0.000 description 21
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 description 20
- 239000011859 microparticle Substances 0.000 description 20
- 125000004122 cyclic group Chemical group 0.000 description 19
- 238000011282 treatment Methods 0.000 description 19
- 230000000295 complement effect Effects 0.000 description 18
- 108700026244 Open Reading Frames Proteins 0.000 description 16
- 150000001875 compounds Chemical class 0.000 description 16
- 229940088598 enzyme Drugs 0.000 description 15
- 108091046869 Telomeric non-coding RNA Proteins 0.000 description 14
- 238000012217 deletion Methods 0.000 description 14
- 230000037430 deletion Effects 0.000 description 14
- 239000012528 membrane Substances 0.000 description 14
- 230000032258 transport Effects 0.000 description 14
- 108010042407 Endonucleases Proteins 0.000 description 13
- 102000004533 Endonucleases Human genes 0.000 description 13
- 241001465754 Metazoa Species 0.000 description 13
- 230000000694 effects Effects 0.000 description 13
- 230000006870 function Effects 0.000 description 13
- 238000004806 packaging method and process Methods 0.000 description 13
- 108700026226 TATA Box Proteins 0.000 description 12
- 241001493065 dsRNA viruses Species 0.000 description 12
- 238000010586 diagram Methods 0.000 description 11
- 238000006467 substitution reaction Methods 0.000 description 11
- 235000012000 cholesterol Nutrition 0.000 description 10
- 238000010362 genome editing Methods 0.000 description 10
- 239000004475 Arginine Substances 0.000 description 9
- ODKSFYDXXFIFQN-BYPYZUCNSA-P L-argininium(2+) Chemical compound NC(=[NH2+])NCCC[C@H]([NH3+])C(O)=O ODKSFYDXXFIFQN-BYPYZUCNSA-P 0.000 description 9
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 description 9
- 229940079593 drug Drugs 0.000 description 9
- 239000003623 enhancer Substances 0.000 description 9
- 239000013612 plasmid Substances 0.000 description 9
- 230000010076 replication Effects 0.000 description 9
- 238000011144 upstream manufacturing Methods 0.000 description 9
- 241001339888 Alphatorquevirus Species 0.000 description 8
- 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 description 8
- 101710150114 Protein rep Proteins 0.000 description 8
- 108091034057 RNA (poly(A)) Proteins 0.000 description 8
- 101710152114 Replication protein Proteins 0.000 description 8
- 108700009124 Transcription Initiation Site Proteins 0.000 description 8
- 230000033228 biological regulation Effects 0.000 description 8
- 238000002360 preparation method Methods 0.000 description 8
- 241000894007 species Species 0.000 description 8
- 239000003981 vehicle Substances 0.000 description 8
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 7
- 230000004568 DNA-binding Effects 0.000 description 7
- 108091027967 Small hairpin RNA Proteins 0.000 description 7
- 230000007423 decrease Effects 0.000 description 7
- 239000003599 detergent Substances 0.000 description 7
- 230000004927 fusion Effects 0.000 description 7
- 230000002458 infectious effect Effects 0.000 description 7
- 229940124531 pharmaceutical excipient Drugs 0.000 description 7
- 238000013518 transcription Methods 0.000 description 7
- 230000035897 transcription Effects 0.000 description 7
- 241000701022 Cytomegalovirus Species 0.000 description 6
- ZDXPYRJPNDTMRX-VKHMYHEASA-N L-glutamine Chemical compound OC(=O)[C@@H](N)CCC(N)=O ZDXPYRJPNDTMRX-VKHMYHEASA-N 0.000 description 6
- OUYCCCASQSFEME-QMMMGPOBSA-N L-tyrosine Chemical compound OC(=O)[C@@H](N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-QMMMGPOBSA-N 0.000 description 6
- 241000124008 Mammalia Species 0.000 description 6
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 description 6
- 108700008625 Reporter Genes Proteins 0.000 description 6
- 230000009368 gene silencing by RNA Effects 0.000 description 6
- ZDXPYRJPNDTMRX-UHFFFAOYSA-N glutamine Natural products OC(=O)C(N)CCC(N)=O ZDXPYRJPNDTMRX-UHFFFAOYSA-N 0.000 description 6
- 238000011194 good manufacturing practice Methods 0.000 description 6
- 208000006454 hepatitis Diseases 0.000 description 6
- 231100000283 hepatitis Toxicity 0.000 description 6
- 230000005847 immunogenicity Effects 0.000 description 6
- 238000003780 insertion Methods 0.000 description 6
- 230000037431 insertion Effects 0.000 description 6
- 230000003834 intracellular effect Effects 0.000 description 6
- 125000005647 linker group Chemical group 0.000 description 6
- 239000002502 liposome Substances 0.000 description 6
- 230000008569 process Effects 0.000 description 6
- 239000000126 substance Substances 0.000 description 6
- 241000701161 unidentified adenovirus Species 0.000 description 6
- 241001529453 unidentified herpesvirus Species 0.000 description 6
- 241001430294 unidentified retrovirus Species 0.000 description 6
- 108700028146 Genetic Enhancer Elements Proteins 0.000 description 5
- AYFVYJQAPQTCCC-GBXIJSLDSA-N L-threonine Chemical compound C[C@@H](O)[C@H](N)C(O)=O AYFVYJQAPQTCCC-GBXIJSLDSA-N 0.000 description 5
- 108010071390 Serum Albumin Proteins 0.000 description 5
- 102000007562 Serum Albumin Human genes 0.000 description 5
- AYFVYJQAPQTCCC-UHFFFAOYSA-N Threonine Natural products CC(O)C(N)C(O)=O AYFVYJQAPQTCCC-UHFFFAOYSA-N 0.000 description 5
- 239000004473 Threonine Substances 0.000 description 5
- 108091028113 Trans-activating crRNA Proteins 0.000 description 5
- 102000004142 Trypsin Human genes 0.000 description 5
- 108090000631 Trypsin Proteins 0.000 description 5
- 238000007792 addition Methods 0.000 description 5
- 238000003776 cleavage reaction Methods 0.000 description 5
- 230000002255 enzymatic effect Effects 0.000 description 5
- 230000001939 inductive effect Effects 0.000 description 5
- 229910010272 inorganic material Inorganic materials 0.000 description 5
- 210000004779 membrane envelope Anatomy 0.000 description 5
- 210000000056 organ Anatomy 0.000 description 5
- 150000003904 phospholipids Chemical class 0.000 description 5
- 238000011002 quantification Methods 0.000 description 5
- 230000007017 scission Effects 0.000 description 5
- 239000012588 trypsin Substances 0.000 description 5
- 108020003589 5' Untranslated Regions Proteins 0.000 description 4
- 241000710929 Alphavirus Species 0.000 description 4
- 241001533384 Circovirus Species 0.000 description 4
- 108091026890 Coding region Proteins 0.000 description 4
- 241000702421 Dependoparvovirus Species 0.000 description 4
- 241000702463 Geminiviridae Species 0.000 description 4
- 108010050904 Interferons Proteins 0.000 description 4
- 102000014150 Interferons Human genes 0.000 description 4
- ROHFNLRQFUQHCH-YFKPBYRVSA-N L-leucine Chemical compound CC(C)C[C@H](N)C(O)=O ROHFNLRQFUQHCH-YFKPBYRVSA-N 0.000 description 4
- ROHFNLRQFUQHCH-UHFFFAOYSA-N Leucine Natural products CC(C)CC(N)C(O)=O ROHFNLRQFUQHCH-UHFFFAOYSA-N 0.000 description 4
- 241000702321 Microvirus Species 0.000 description 4
- 241000204031 Mycoplasma Species 0.000 description 4
- 230000004913 activation Effects 0.000 description 4
- 230000003321 amplification Effects 0.000 description 4
- 210000004899 c-terminal region Anatomy 0.000 description 4
- 230000021164 cell adhesion Effects 0.000 description 4
- 230000003915 cell function Effects 0.000 description 4
- 230000002950 deficient Effects 0.000 description 4
- 230000001419 dependent effect Effects 0.000 description 4
- 238000001514 detection method Methods 0.000 description 4
- 238000012377 drug delivery Methods 0.000 description 4
- 239000002158 endotoxin Substances 0.000 description 4
- 230000028023 exocytosis Effects 0.000 description 4
- WHUUTDBJXJRKMK-VKHMYHEASA-L glutamate group Chemical group N[C@@H](CCC(=O)[O-])C(=O)[O-] WHUUTDBJXJRKMK-VKHMYHEASA-L 0.000 description 4
- 239000012535 impurity Substances 0.000 description 4
- 238000000338 in vitro Methods 0.000 description 4
- 230000001965 increasing effect Effects 0.000 description 4
- 150000002484 inorganic compounds Chemical class 0.000 description 4
- 229940079322 interferon Drugs 0.000 description 4
- 230000004807 localization Effects 0.000 description 4
- 108091056454 miR-625 stem-loop Proteins 0.000 description 4
- 238000012544 monitoring process Methods 0.000 description 4
- 108091027963 non-coding RNA Proteins 0.000 description 4
- 102000042567 non-coding RNA Human genes 0.000 description 4
- 238000003199 nucleic acid amplification method Methods 0.000 description 4
- 150000002894 organic compounds Chemical class 0.000 description 4
- 244000052769 pathogen Species 0.000 description 4
- 230000001575 pathological effect Effects 0.000 description 4
- 108010079892 phosphoglycerol kinase Proteins 0.000 description 4
- 229920000724 poly(L-arginine) polymer Polymers 0.000 description 4
- 108010011110 polyarginine Proteins 0.000 description 4
- 108010040003 polyglutamine Proteins 0.000 description 4
- 229920000155 polyglutamine Polymers 0.000 description 4
- 230000035755 proliferation Effects 0.000 description 4
- 239000000243 solution Substances 0.000 description 4
- 241001147420 ssDNA viruses Species 0.000 description 4
- 230000002103 transcriptional effect Effects 0.000 description 4
- 238000013519 translation Methods 0.000 description 4
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 4
- MTCFGRXMJLQNBG-REOHCLBHSA-N (2S)-2-Amino-3-hydroxypropansäure Chemical compound OC[C@H](N)C(O)=O MTCFGRXMJLQNBG-REOHCLBHSA-N 0.000 description 3
- 102000009027 Albumins Human genes 0.000 description 3
- 108010088751 Albumins Proteins 0.000 description 3
- 108020004638 Circular DNA Proteins 0.000 description 3
- 241000702216 Cystovirus Species 0.000 description 3
- 108010008532 Deoxyribonuclease I Proteins 0.000 description 3
- 102000007260 Deoxyribonuclease I Human genes 0.000 description 3
- 241000196324 Embryophyta Species 0.000 description 3
- 241000723722 Furovirus Species 0.000 description 3
- 241000868843 Globulovirus Species 0.000 description 3
- 241000282412 Homo Species 0.000 description 3
- 241000702377 Inovirus Species 0.000 description 3
- 241000701372 Iridovirus Species 0.000 description 3
- AGPKZVBTJJNPAG-WHFBIAKZSA-N L-isoleucine Chemical compound CC[C@H](C)[C@H](N)C(O)=O AGPKZVBTJJNPAG-WHFBIAKZSA-N 0.000 description 3
- 241000713666 Lentivirus Species 0.000 description 3
- 241000699666 Mus <mouse, genus> Species 0.000 description 3
- 241001494793 Nanovirus Species 0.000 description 3
- 108091005461 Nucleic proteins Proteins 0.000 description 3
- 241001631646 Papillomaviridae Species 0.000 description 3
- 241000125945 Protoparvovirus Species 0.000 description 3
- 241000702670 Rotavirus Species 0.000 description 3
- 241000714474 Rous sarcoma virus Species 0.000 description 3
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 description 3
- 108010022394 Threonine synthase Proteins 0.000 description 3
- 241000723848 Tobamovirus Species 0.000 description 3
- 241000723717 Tobravirus Species 0.000 description 3
- 241000700618 Vaccinia virus Species 0.000 description 3
- 108020005202 Viral DNA Proteins 0.000 description 3
- 108010017070 Zinc Finger Nucleases Proteins 0.000 description 3
- 230000002159 abnormal effect Effects 0.000 description 3
- 101150063416 add gene Proteins 0.000 description 3
- 238000004630 atomic force microscopy Methods 0.000 description 3
- 230000008238 biochemical pathway Effects 0.000 description 3
- 210000000234 capsid Anatomy 0.000 description 3
- 150000001720 carbohydrates Chemical class 0.000 description 3
- 235000014633 carbohydrates Nutrition 0.000 description 3
- 125000003636 chemical group Chemical group 0.000 description 3
- 230000001461 cytolytic effect Effects 0.000 description 3
- 102000004419 dihydrofolate reductase Human genes 0.000 description 3
- 230000012202 endocytosis Effects 0.000 description 3
- 150000002148 esters Chemical class 0.000 description 3
- 230000017188 evasion or tolerance of host immune response Effects 0.000 description 3
- 102000035122 glycosylated proteins Human genes 0.000 description 3
- 108091005608 glycosylated proteins Proteins 0.000 description 3
- 239000005556 hormone Substances 0.000 description 3
- 229940088597 hormone Drugs 0.000 description 3
- 239000012216 imaging agent Substances 0.000 description 3
- 230000010354 integration Effects 0.000 description 3
- 150000002500 ions Chemical class 0.000 description 3
- AGPKZVBTJJNPAG-UHFFFAOYSA-N isoleucine Natural products CCC(C)C(N)C(O)=O AGPKZVBTJJNPAG-UHFFFAOYSA-N 0.000 description 3
- 229960000310 isoleucine Drugs 0.000 description 3
- 210000004185 liver Anatomy 0.000 description 3
- 239000000463 material Substances 0.000 description 3
- 230000004048 modification Effects 0.000 description 3
- 238000012986 modification Methods 0.000 description 3
- 230000035772 mutation Effects 0.000 description 3
- 210000002569 neuron Anatomy 0.000 description 3
- 208000002154 non-small cell lung carcinoma Diseases 0.000 description 3
- 210000004940 nucleus Anatomy 0.000 description 3
- 239000000816 peptidomimetic Substances 0.000 description 3
- WTJKGGKOPKCXLL-RRHRGVEJSA-N phosphatidylcholine Chemical compound CCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCC=CCCCCCCCC WTJKGGKOPKCXLL-RRHRGVEJSA-N 0.000 description 3
- 238000012552 review Methods 0.000 description 3
- 150000003839 salts Chemical class 0.000 description 3
- 230000011664 signaling Effects 0.000 description 3
- 239000011550 stock solution Substances 0.000 description 3
- 239000000758 substrate Substances 0.000 description 3
- 239000006228 supernatant Substances 0.000 description 3
- 238000002560 therapeutic procedure Methods 0.000 description 3
- 238000001890 transfection Methods 0.000 description 3
- 208000029729 tumor suppressor gene on chromosome 11 Diseases 0.000 description 3
- OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Natural products OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 description 3
- 241000701447 unidentified baculovirus Species 0.000 description 3
- 230000007332 vesicle formation Effects 0.000 description 3
- 230000035899 viability Effects 0.000 description 3
- NRJAVPSFFCBXDT-HUESYALOSA-N 1,2-distearoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCCCCCC NRJAVPSFFCBXDT-HUESYALOSA-N 0.000 description 2
- LMDZBCPBFSXMTL-UHFFFAOYSA-N 1-ethyl-3-(3-dimethylaminopropyl)carbodiimide Chemical compound CCN=C=NCCCN(C)C LMDZBCPBFSXMTL-UHFFFAOYSA-N 0.000 description 2
- LDGWQMRUWMSZIU-LQDDAWAPSA-M 2,3-bis[(z)-octadec-9-enoxy]propyl-trimethylazanium;chloride Chemical compound [Cl-].CCCCCCCC\C=C/CCCCCCCCOCC(C[N+](C)(C)C)OCCCCCCCC\C=C/CCCCCCCC LDGWQMRUWMSZIU-LQDDAWAPSA-M 0.000 description 2
- KSXTUUUQYQYKCR-LQDDAWAPSA-M 2,3-bis[[(z)-octadec-9-enoyl]oxy]propyl-trimethylazanium;chloride Chemical compound [Cl-].CCCCCCCC\C=C/CCCCCCCC(=O)OCC(C[N+](C)(C)C)OC(=O)CCCCCCC\C=C/CCCCCCCC KSXTUUUQYQYKCR-LQDDAWAPSA-M 0.000 description 2
- 102100023635 Alpha-fetoprotein Human genes 0.000 description 2
- DCXYFEDJOCDNAF-UHFFFAOYSA-N Asparagine Natural products OC(=O)C(N)CC(N)=O DCXYFEDJOCDNAF-UHFFFAOYSA-N 0.000 description 2
- 241000894006 Bacteria Species 0.000 description 2
- 206010006187 Breast cancer Diseases 0.000 description 2
- 208000026310 Breast neoplasm Diseases 0.000 description 2
- 241001533399 Circoviridae Species 0.000 description 2
- 108091035707 Consensus sequence Proteins 0.000 description 2
- 239000004971 Cross linker Substances 0.000 description 2
- 102220605874 Cytosolic arginine sensor for mTORC1 subunit 2_D10A_mutation Human genes 0.000 description 2
- 230000007018 DNA scission Effects 0.000 description 2
- 241000450599 DNA viruses Species 0.000 description 2
- 102000016928 DNA-directed DNA polymerase Human genes 0.000 description 2
- 108010014303 DNA-directed DNA polymerase Proteins 0.000 description 2
- 108010078532 Gal-VP16 Proteins 0.000 description 2
- 241001339886 Gammatorquevirus Species 0.000 description 2
- WHUUTDBJXJRKMK-UHFFFAOYSA-N Glutamic acid Natural products OC(=O)C(N)CCC(O)=O WHUUTDBJXJRKMK-UHFFFAOYSA-N 0.000 description 2
- 102000005720 Glutathione transferase Human genes 0.000 description 2
- 108010070675 Glutathione transferase Proteins 0.000 description 2
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 description 2
- AEMRFAOFKBGASW-UHFFFAOYSA-N Glycolic acid Chemical compound OCC(O)=O AEMRFAOFKBGASW-UHFFFAOYSA-N 0.000 description 2
- 241000725303 Human immunodeficiency virus Species 0.000 description 2
- 206010062016 Immunosuppression Diseases 0.000 description 2
- XEEYBQQBJWHFJM-UHFFFAOYSA-N Iron Chemical compound [Fe] XEEYBQQBJWHFJM-UHFFFAOYSA-N 0.000 description 2
- DCXYFEDJOCDNAF-REOHCLBHSA-N L-asparagine Chemical compound OC(=O)[C@@H](N)CC(N)=O DCXYFEDJOCDNAF-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
- WHUUTDBJXJRKMK-VKHMYHEASA-N L-glutamic acid Chemical compound OC(=O)[C@@H](N)CCC(O)=O WHUUTDBJXJRKMK-VKHMYHEASA-N 0.000 description 2
- HNDVDQJCIGZPNO-YFKPBYRVSA-N L-histidine Chemical compound OC(=O)[C@@H](N)CC1=CN=CN1 HNDVDQJCIGZPNO-YFKPBYRVSA-N 0.000 description 2
- FFEARJCKVFRZRR-BYPYZUCNSA-N L-methionine Chemical compound CSCC[C@H](N)C(O)=O FFEARJCKVFRZRR-BYPYZUCNSA-N 0.000 description 2
- COLNVLDHVKWLRT-QMMMGPOBSA-N L-phenylalanine Chemical compound OC(=O)[C@@H](N)CC1=CC=CC=C1 COLNVLDHVKWLRT-QMMMGPOBSA-N 0.000 description 2
- QIVBCDIJIAJPQS-VIFPVBQESA-N L-tryptophane Chemical compound C1=CC=C2C(C[C@H](N)C(O)=O)=CNC2=C1 QIVBCDIJIAJPQS-VIFPVBQESA-N 0.000 description 2
- 108060001084 Luciferase Proteins 0.000 description 2
- 239000005089 Luciferase Substances 0.000 description 2
- 101710175625 Maltose/maltodextrin-binding periplasmic protein Proteins 0.000 description 2
- 241000713333 Mouse mammary tumor virus Species 0.000 description 2
- 102000010292 Peptide Elongation Factor 1 Human genes 0.000 description 2
- 108010077524 Peptide Elongation Factor 1 Proteins 0.000 description 2
- 108010043958 Peptoids Proteins 0.000 description 2
- 229920000954 Polyglycolide Polymers 0.000 description 2
- 108010029485 Protein Isoforms Proteins 0.000 description 2
- 102000009572 RNA Polymerase II Human genes 0.000 description 2
- 108010009460 RNA Polymerase II Proteins 0.000 description 2
- 102000014450 RNA Polymerase III Human genes 0.000 description 2
- 108010078067 RNA Polymerase III Proteins 0.000 description 2
- 101710151532 Replicating protein Proteins 0.000 description 2
- 241000959822 SEN virus Species 0.000 description 2
- 108010003723 Single-Domain Antibodies Proteins 0.000 description 2
- 101710172711 Structural protein Proteins 0.000 description 2
- 108091027544 Subgenomic mRNA Proteins 0.000 description 2
- 241001121097 TT virus genotype 6 Species 0.000 description 2
- 241000412398 TTV-like virus DXL1 Species 0.000 description 2
- 241000412397 TTV-like virus DXL2 Species 0.000 description 2
- 239000004098 Tetracycline Substances 0.000 description 2
- 102000002933 Thioredoxin Human genes 0.000 description 2
- 108091036066 Three prime untranslated region Proteins 0.000 description 2
- QIVBCDIJIAJPQS-UHFFFAOYSA-N Tryptophan Natural products C1=CC=C2C(CC(N)C(O)=O)=CNC2=C1 QIVBCDIJIAJPQS-UHFFFAOYSA-N 0.000 description 2
- 241000251539 Vertebrata <Metazoa> Species 0.000 description 2
- 206010058874 Viraemia Diseases 0.000 description 2
- 230000003213 activating effect Effects 0.000 description 2
- 230000002730 additional effect Effects 0.000 description 2
- 108010026331 alpha-Fetoproteins Proteins 0.000 description 2
- 210000004102 animal cell Anatomy 0.000 description 2
- 238000010171 animal model Methods 0.000 description 2
- 230000000840 anti-viral effect Effects 0.000 description 2
- 235000009582 asparagine Nutrition 0.000 description 2
- 229960001230 asparagine Drugs 0.000 description 2
- 235000003704 aspartic acid Nutrition 0.000 description 2
- 230000001580 bacterial effect Effects 0.000 description 2
- OQFSQFPPLPISGP-UHFFFAOYSA-N beta-carboxyaspartic acid Natural products OC(=O)C(N)C(C(O)=O)C(O)=O OQFSQFPPLPISGP-UHFFFAOYSA-N 0.000 description 2
- 210000000988 bone and bone Anatomy 0.000 description 2
- 210000001185 bone marrow Anatomy 0.000 description 2
- 238000007385 chemical modification Methods 0.000 description 2
- 102000021178 chitin binding proteins Human genes 0.000 description 2
- 108091011157 chitin binding proteins Proteins 0.000 description 2
- 229960001231 choline Drugs 0.000 description 2
- OEYIOHPDSNJKLS-UHFFFAOYSA-N choline Chemical compound C[N+](C)(C)CCO OEYIOHPDSNJKLS-UHFFFAOYSA-N 0.000 description 2
- 239000002131 composite material Substances 0.000 description 2
- 239000002872 contrast media Substances 0.000 description 2
- 229920001577 copolymer Polymers 0.000 description 2
- 230000000120 cytopathologic effect Effects 0.000 description 2
- 238000013461 design Methods 0.000 description 2
- UREBDLICKHMUKA-CXSFZGCWSA-N dexamethasone Chemical compound C1CC2=CC(=O)C=C[C@]2(C)[C@]2(F)[C@@H]1[C@@H]1C[C@@H](C)[C@@](C(=O)CO)(O)[C@@]1(C)C[C@@H]2O UREBDLICKHMUKA-CXSFZGCWSA-N 0.000 description 2
- 239000000032 diagnostic agent Substances 0.000 description 2
- 229940039227 diagnostic agent Drugs 0.000 description 2
- UMGXUWVIJIQANV-UHFFFAOYSA-M didecyl(dimethyl)azanium;bromide Chemical compound [Br-].CCCCCCCCCC[N+](C)(C)CCCCCCCCCC UMGXUWVIJIQANV-UHFFFAOYSA-M 0.000 description 2
- 238000002296 dynamic light scattering Methods 0.000 description 2
- 230000009144 enzymatic modification Effects 0.000 description 2
- 108020001507 fusion proteins Proteins 0.000 description 2
- 102000037865 fusion proteins Human genes 0.000 description 2
- 235000013922 glutamic acid Nutrition 0.000 description 2
- 239000004220 glutamic acid Substances 0.000 description 2
- 239000005090 green fluorescent protein Substances 0.000 description 2
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Natural products OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 description 2
- 230000001506 immunosuppresive effect Effects 0.000 description 2
- NOESYZHRGYRDHS-UHFFFAOYSA-N insulin Chemical compound N1C(=O)C(NC(=O)C(CCC(N)=O)NC(=O)C(CCC(O)=O)NC(=O)C(C(C)C)NC(=O)C(NC(=O)CN)C(C)CC)CSSCC(C(NC(CO)C(=O)NC(CC(C)C)C(=O)NC(CC=2C=CC(O)=CC=2)C(=O)NC(CCC(N)=O)C(=O)NC(CC(C)C)C(=O)NC(CCC(O)=O)C(=O)NC(CC(N)=O)C(=O)NC(CC=2C=CC(O)=CC=2)C(=O)NC(CSSCC(NC(=O)C(C(C)C)NC(=O)C(CC(C)C)NC(=O)C(CC=2C=CC(O)=CC=2)NC(=O)C(CC(C)C)NC(=O)C(C)NC(=O)C(CCC(O)=O)NC(=O)C(C(C)C)NC(=O)C(CC(C)C)NC(=O)C(CC=2NC=NC=2)NC(=O)C(CO)NC(=O)CNC2=O)C(=O)NCC(=O)NC(CCC(O)=O)C(=O)NC(CCCNC(N)=N)C(=O)NCC(=O)NC(CC=3C=CC=CC=3)C(=O)NC(CC=3C=CC=CC=3)C(=O)NC(CC=3C=CC(O)=CC=3)C(=O)NC(C(C)O)C(=O)N3C(CCC3)C(=O)NC(CCCCN)C(=O)NC(C)C(O)=O)C(=O)NC(CC(N)=O)C(O)=O)=O)NC(=O)C(C(C)CC)NC(=O)C(CO)NC(=O)C(C(C)O)NC(=O)C1CSSCC2NC(=O)C(CC(C)C)NC(=O)C(NC(=O)C(CCC(N)=O)NC(=O)C(CC(N)=O)NC(=O)C(NC(=O)C(N)CC=1C=CC=CC=1)C(C)C)CC1=CN=CN1 NOESYZHRGYRDHS-UHFFFAOYSA-N 0.000 description 2
- 239000000543 intermediate Substances 0.000 description 2
- 229930027917 kanamycin Natural products 0.000 description 2
- 229960000318 kanamycin Drugs 0.000 description 2
- SBUJHOSQTJFQJX-NOAMYHISSA-N kanamycin Chemical compound O[C@@H]1[C@@H](O)[C@H](O)[C@@H](CN)O[C@@H]1O[C@H]1[C@H](O)[C@@H](O[C@@H]2[C@@H]([C@@H](N)[C@H](O)[C@@H](CO)O2)O)[C@H](N)C[C@@H]1N SBUJHOSQTJFQJX-NOAMYHISSA-N 0.000 description 2
- 229930182823 kanamycin A Natural products 0.000 description 2
- JVTAAEKCZFNVCJ-UHFFFAOYSA-N lactic acid Chemical compound CC(O)C(O)=O JVTAAEKCZFNVCJ-UHFFFAOYSA-N 0.000 description 2
- 230000002934 lysing effect Effects 0.000 description 2
- 238000002595 magnetic resonance imaging Methods 0.000 description 2
- 230000014759 maintenance of location Effects 0.000 description 2
- 230000001404 mediated effect Effects 0.000 description 2
- 229930182817 methionine Natural products 0.000 description 2
- 230000000813 microbial effect Effects 0.000 description 2
- 230000003278 mimic effect Effects 0.000 description 2
- 210000003470 mitochondria Anatomy 0.000 description 2
- 210000003205 muscle Anatomy 0.000 description 2
- 239000002858 neurotransmitter agent Substances 0.000 description 2
- 230000008506 pathogenesis Effects 0.000 description 2
- 230000037361 pathway Effects 0.000 description 2
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Natural products OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 description 2
- 229920000747 poly(lactic acid) Polymers 0.000 description 2
- 229920001610 polycaprolactone Polymers 0.000 description 2
- 239000004632 polycaprolactone Substances 0.000 description 2
- 230000017854 proteolysis Effects 0.000 description 2
- 238000003908 quality control method Methods 0.000 description 2
- 102000005912 ran GTP Binding Protein Human genes 0.000 description 2
- 230000002829 reductive effect Effects 0.000 description 2
- 230000000717 retained effect Effects 0.000 description 2
- 239000000523 sample Substances 0.000 description 2
- 230000002269 spontaneous effect Effects 0.000 description 2
- 239000003381 stabilizer Substances 0.000 description 2
- 229960002180 tetracycline Drugs 0.000 description 2
- 229930101283 tetracycline Natural products 0.000 description 2
- 235000019364 tetracycline Nutrition 0.000 description 2
- 150000003522 tetracyclines Chemical class 0.000 description 2
- 108060008226 thioredoxin Proteins 0.000 description 2
- 229940094937 thioredoxin Drugs 0.000 description 2
- 239000003053 toxin Substances 0.000 description 2
- 231100000765 toxin Toxicity 0.000 description 2
- 108700012359 toxins Proteins 0.000 description 2
- 230000005945 translocation Effects 0.000 description 2
- 238000004627 transmission electron microscopy Methods 0.000 description 2
- NQPDZGIKBAWPEJ-UHFFFAOYSA-N valeric acid Chemical compound CCCCC(O)=O NQPDZGIKBAWPEJ-UHFFFAOYSA-N 0.000 description 2
- 230000029812 viral genome replication Effects 0.000 description 2
- 210000000605 viral structure Anatomy 0.000 description 2
- MSTNYGQPCMXVAQ-RYUDHWBXSA-N (6S)-5,6,7,8-tetrahydrofolic acid Chemical compound C([C@H]1CNC=2N=C(NC(=O)C=2N1)N)NC1=CC=C(C(=O)N[C@@H](CCC(O)=O)C(O)=O)C=C1 MSTNYGQPCMXVAQ-RYUDHWBXSA-N 0.000 description 1
- SNKAWJBJQDLSFF-NVKMUCNASA-N 1,2-dioleoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCC\C=C/CCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCC\C=C/CCCCCCCC SNKAWJBJQDLSFF-NVKMUCNASA-N 0.000 description 1
- LRFJOIPOPUJUMI-KWXKLSQISA-N 2-[2,2-bis[(9z,12z)-octadeca-9,12-dienyl]-1,3-dioxolan-4-yl]-n,n-dimethylethanamine Chemical compound CCCCC\C=C/C\C=C/CCCCCCCCC1(CCCCCCCC\C=C/C\C=C/CCCCC)OCC(CCN(C)C)O1 LRFJOIPOPUJUMI-KWXKLSQISA-N 0.000 description 1
- SHCCKWGIFIPGNJ-NSUCVBPYSA-N 2-aminoethyl [(2r)-2,3-bis[(z)-octadec-9-enoxy]propyl] hydrogen phosphate Chemical compound CCCCCCCC\C=C/CCCCCCCCOC[C@H](COP(O)(=O)OCCN)OCCCCCCCC\C=C/CCCCCCCC SHCCKWGIFIPGNJ-NSUCVBPYSA-N 0.000 description 1
- HVCOBJNICQPDBP-UHFFFAOYSA-N 3-[3-[3,5-dihydroxy-6-methyl-4-(3,4,5-trihydroxy-6-methyloxan-2-yl)oxyoxan-2-yl]oxydecanoyloxy]decanoic acid;hydrate Chemical compound O.OC1C(OC(CC(=O)OC(CCCCCCC)CC(O)=O)CCCCCCC)OC(C)C(O)C1OC1C(O)C(O)C(O)C(C)O1 HVCOBJNICQPDBP-UHFFFAOYSA-N 0.000 description 1
- AJNUQUGWNQHQDJ-UHFFFAOYSA-N 4',5'-bis(1,3,2-dithiarsolan-2-yl)-3',6'-dihydroxyspiro[2-benzofuran-3,9'-xanthene]-1-one Chemical compound S1CCS[As]1C=1C(O)=CC=C(C23C4=CC=CC=C4C(=O)O3)C=1OC1=C2C=CC(O)=C1[As]1SCCS1 AJNUQUGWNQHQDJ-UHFFFAOYSA-N 0.000 description 1
- KQPKMEYBZUPZGK-UHFFFAOYSA-N 4-[(4-azido-2-nitroanilino)methyl]-5-(hydroxymethyl)-2-methylpyridin-3-ol Chemical compound CC1=NC=C(CO)C(CNC=2C(=CC(=CC=2)N=[N+]=[N-])[N+]([O-])=O)=C1O KQPKMEYBZUPZGK-UHFFFAOYSA-N 0.000 description 1
- 108020005029 5' Flanking Region Proteins 0.000 description 1
- FHVDTGUDJYJELY-UHFFFAOYSA-N 6-{[2-carboxy-4,5-dihydroxy-6-(phosphanyloxy)oxan-3-yl]oxy}-4,5-dihydroxy-3-phosphanyloxane-2-carboxylic acid Chemical compound O1C(C(O)=O)C(P)C(O)C(O)C1OC1C(C(O)=O)OC(OP)C(O)C1O FHVDTGUDJYJELY-UHFFFAOYSA-N 0.000 description 1
- 241000604451 Acidaminococcus Species 0.000 description 1
- 101000860090 Acidaminococcus sp. (strain BV3L6) CRISPR-associated endonuclease Cas12a Proteins 0.000 description 1
- 108010085238 Actins Proteins 0.000 description 1
- 102000007469 Actins Human genes 0.000 description 1
- 108020004774 Alkaline Phosphatase Proteins 0.000 description 1
- 102000002260 Alkaline Phosphatase Human genes 0.000 description 1
- 241000701368 Alphafusellovirus Species 0.000 description 1
- 108091023037 Aptamer Proteins 0.000 description 1
- 206010003445 Ascites Diseases 0.000 description 1
- 241000157874 Ascovirus Species 0.000 description 1
- 241000714230 Avian leukemia virus Species 0.000 description 1
- 102100026189 Beta-galactosidase Human genes 0.000 description 1
- 241000405758 Betapartitivirus Species 0.000 description 1
- 241001339887 Betatorquevirus Species 0.000 description 1
- 108091079001 CRISPR RNA Proteins 0.000 description 1
- 238000010446 CRISPR interference Methods 0.000 description 1
- 101710167800 Capsid assembly scaffolding protein Proteins 0.000 description 1
- 201000009030 Carcinoma Diseases 0.000 description 1
- 102000014914 Carrier Proteins Human genes 0.000 description 1
- 108010078791 Carrier Proteins Proteins 0.000 description 1
- 108090000994 Catalytic RNA Proteins 0.000 description 1
- 102000053642 Catalytic RNA Human genes 0.000 description 1
- 108010035563 Chloramphenicol O-acetyltransferase Proteins 0.000 description 1
- VYZAMTAEIAYCRO-UHFFFAOYSA-N Chromium Chemical compound [Cr] VYZAMTAEIAYCRO-UHFFFAOYSA-N 0.000 description 1
- 108091033380 Coding strand Proteins 0.000 description 1
- 102000008186 Collagen Human genes 0.000 description 1
- 108010035532 Collagen Proteins 0.000 description 1
- RYGMFSIKBFXOCR-UHFFFAOYSA-N Copper Chemical compound [Cu] RYGMFSIKBFXOCR-UHFFFAOYSA-N 0.000 description 1
- 241000195493 Cryptophyta Species 0.000 description 1
- 102000004127 Cytokines Human genes 0.000 description 1
- 108090000695 Cytokines Proteins 0.000 description 1
- 230000033616 DNA repair Effects 0.000 description 1
- 230000004543 DNA replication Effects 0.000 description 1
- 108090000626 DNA-directed RNA polymerases Proteins 0.000 description 1
- 102000004163 DNA-directed RNA polymerases Human genes 0.000 description 1
- 229920002307 Dextran Polymers 0.000 description 1
- GZDFHIJNHHMENY-UHFFFAOYSA-N Dimethyl dicarbonate Chemical compound COC(=O)OC(=O)OC GZDFHIJNHHMENY-UHFFFAOYSA-N 0.000 description 1
- 208000027244 Dysbiosis Diseases 0.000 description 1
- 108010069091 Dystrophin Proteins 0.000 description 1
- 102000001039 Dystrophin Human genes 0.000 description 1
- 108010013369 Enteropeptidase Proteins 0.000 description 1
- 102100029727 Enteropeptidase Human genes 0.000 description 1
- 241000588724 Escherichia coli Species 0.000 description 1
- 108010074860 Factor Xa Proteins 0.000 description 1
- YXWOAJXNVLXPMU-ZXXMMSQZSA-N Fructose 2,6-diphosphate Chemical compound OP(=O)(O)O[C@]1(CO)O[C@H](COP(O)(O)=O)[C@@H](O)[C@@H]1O YXWOAJXNVLXPMU-ZXXMMSQZSA-N 0.000 description 1
- 229910052688 Gadolinium Inorganic materials 0.000 description 1
- 102100028652 Gamma-enolase Human genes 0.000 description 1
- 108700039691 Genetic Promoter Regions Proteins 0.000 description 1
- SXRSQZLOMIGNAQ-UHFFFAOYSA-N Glutaraldehyde Chemical compound O=CCCCC=O SXRSQZLOMIGNAQ-UHFFFAOYSA-N 0.000 description 1
- 239000004471 Glycine Substances 0.000 description 1
- 229930186217 Glycolipid Natural products 0.000 description 1
- 108010043121 Green Fluorescent Proteins Proteins 0.000 description 1
- 101710189078 Helicase Proteins 0.000 description 1
- 241000700721 Hepatitis B virus Species 0.000 description 1
- 101001111984 Homo sapiens N-acylneuraminate-9-phosphatase Proteins 0.000 description 1
- 101000979333 Homo sapiens Neurofilament light polypeptide Proteins 0.000 description 1
- 241000701044 Human gammaherpesvirus 4 Species 0.000 description 1
- 238000004566 IR spectroscopy Methods 0.000 description 1
- 102000006496 Immunoglobulin Heavy Chains Human genes 0.000 description 1
- 108010019476 Immunoglobulin Heavy Chains Proteins 0.000 description 1
- 241000702394 Inoviridae Species 0.000 description 1
- 108090001061 Insulin Proteins 0.000 description 1
- 102000004877 Insulin Human genes 0.000 description 1
- 102000016921 Integrin-Binding Sialoprotein Human genes 0.000 description 1
- 108010028750 Integrin-Binding Sialoprotein Proteins 0.000 description 1
- 102000002227 Interferon Type I Human genes 0.000 description 1
- 108010014726 Interferon Type I Proteins 0.000 description 1
- 206010056254 Intrauterine infection Diseases 0.000 description 1
- XUJNEKJLAYXESH-REOHCLBHSA-N L-Cysteine Chemical compound SC[C@H](N)C(O)=O XUJNEKJLAYXESH-REOHCLBHSA-N 0.000 description 1
- ONIBWKKTOPOVIA-BYPYZUCNSA-N L-Proline Chemical compound OC(=O)[C@@H]1CCCN1 ONIBWKKTOPOVIA-BYPYZUCNSA-N 0.000 description 1
- QNAYBMKLOCPYGJ-REOHCLBHSA-N L-alanine Chemical compound C[C@H](N)C(O)=O QNAYBMKLOCPYGJ-REOHCLBHSA-N 0.000 description 1
- KDXKERNSBIXSRK-YFKPBYRVSA-N L-lysine Chemical compound NCCCC[C@H](N)C(O)=O KDXKERNSBIXSRK-YFKPBYRVSA-N 0.000 description 1
- FBOZXECLQNJBKD-ZDUSSCGKSA-N L-methotrexate Chemical compound C=1N=C2N=C(N)N=C(N)C2=NC=1CN(C)C1=CC=C(C(=O)N[C@@H](CCC(O)=O)C(O)=O)C=C1 FBOZXECLQNJBKD-ZDUSSCGKSA-N 0.000 description 1
- KZSNJWFQEVHDMF-BYPYZUCNSA-N L-valine Chemical compound CC(C)[C@H](N)C(O)=O KZSNJWFQEVHDMF-BYPYZUCNSA-N 0.000 description 1
- 241001112693 Lachnospiraceae Species 0.000 description 1
- 102000003960 Ligases Human genes 0.000 description 1
- 108090000364 Ligases Proteins 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
- 108010059343 MM Form Creatine Kinase Proteins 0.000 description 1
- FYYHWMGAXLPEAU-UHFFFAOYSA-N Magnesium Chemical compound [Mg] FYYHWMGAXLPEAU-UHFFFAOYSA-N 0.000 description 1
- PEEHTFAAVSWFBL-UHFFFAOYSA-N Maleimide Chemical compound O=C1NC(=O)C=C1 PEEHTFAAVSWFBL-UHFFFAOYSA-N 0.000 description 1
- 102000003939 Membrane transport proteins Human genes 0.000 description 1
- 108090000301 Membrane transport proteins Proteins 0.000 description 1
- 241000702318 Microviridae Species 0.000 description 1
- 108010006519 Molecular Chaperones Proteins 0.000 description 1
- 108010085220 Multiprotein Complexes Proteins 0.000 description 1
- 102000007474 Multiprotein Complexes Human genes 0.000 description 1
- 101100219625 Mus musculus Casd1 gene Proteins 0.000 description 1
- 102100026057 Myosin regulatory light chain 2, atrial isoform Human genes 0.000 description 1
- 101710098224 Myosin regulatory light chain 2, atrial isoform Proteins 0.000 description 1
- 102100023906 N-acylneuraminate-9-phosphatase Human genes 0.000 description 1
- 108091061960 Naked DNA Proteins 0.000 description 1
- 241001336717 Nanoviridae Species 0.000 description 1
- 241000588653 Neisseria Species 0.000 description 1
- 102000019040 Nuclear Antigens Human genes 0.000 description 1
- 108010051791 Nuclear Antigens Proteins 0.000 description 1
- 108020003217 Nuclear RNA Proteins 0.000 description 1
- 102000043141 Nuclear RNA Human genes 0.000 description 1
- 101710113540 ORF2 protein Proteins 0.000 description 1
- 102000003840 Opioid Receptors Human genes 0.000 description 1
- 108090000137 Opioid Receptors Proteins 0.000 description 1
- 206010048685 Oral infection Diseases 0.000 description 1
- 241000713112 Orthobunyavirus Species 0.000 description 1
- 241000712464 Orthomyxoviridae Species 0.000 description 1
- 102000004067 Osteocalcin Human genes 0.000 description 1
- 108090000573 Osteocalcin Proteins 0.000 description 1
- 229910019142 PO4 Inorganic materials 0.000 description 1
- 241000701945 Parvoviridae Species 0.000 description 1
- 102000012435 Phosphofructokinase-1 Human genes 0.000 description 1
- 108010022684 Phosphofructokinase-1 Proteins 0.000 description 1
- 102000012288 Phosphopyruvate Hydratase Human genes 0.000 description 1
- 108010022181 Phosphopyruvate Hydratase Proteins 0.000 description 1
- 229920002732 Polyanhydride Polymers 0.000 description 1
- 241000932075 Priacanthus hamrur Species 0.000 description 1
- ONIBWKKTOPOVIA-UHFFFAOYSA-N Proline Natural products OC(=O)C1CCCN1 ONIBWKKTOPOVIA-UHFFFAOYSA-N 0.000 description 1
- 229940079156 Proteasome inhibitor Drugs 0.000 description 1
- 102000001708 Protein Isoforms Human genes 0.000 description 1
- 101710090523 Putative movement protein Proteins 0.000 description 1
- 230000004570 RNA-binding Effects 0.000 description 1
- 101710118046 RNA-directed RNA polymerase Proteins 0.000 description 1
- 238000001069 Raman spectroscopy Methods 0.000 description 1
- 108010008281 Recombinant Fusion Proteins Proteins 0.000 description 1
- 102000007056 Recombinant Fusion Proteins Human genes 0.000 description 1
- 241000702263 Reovirus sp. Species 0.000 description 1
- 241001068263 Replication competent viruses Species 0.000 description 1
- 108010057163 Ribonuclease III Proteins 0.000 description 1
- 102000003661 Ribonuclease III Human genes 0.000 description 1
- 240000004808 Saccharomyces cerevisiae Species 0.000 description 1
- 241000242583 Scyphozoa Species 0.000 description 1
- 108010032838 Sialoglycoproteins Proteins 0.000 description 1
- 102000007365 Sialoglycoproteins Human genes 0.000 description 1
- 241000194017 Streptococcus Species 0.000 description 1
- 241000193996 Streptococcus pyogenes Species 0.000 description 1
- 241000194020 Streptococcus thermophilus 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
- 108010076818 TEV protease Proteins 0.000 description 1
- 108090000190 Thrombin Proteins 0.000 description 1
- 102100035100 Transcription factor p65 Human genes 0.000 description 1
- 108090000848 Ubiquitin Proteins 0.000 description 1
- 102000044159 Ubiquitin Human genes 0.000 description 1
- 101150004676 VGF gene Proteins 0.000 description 1
- KZSNJWFQEVHDMF-UHFFFAOYSA-N Valine Natural products CC(C)C(N)C(O)=O KZSNJWFQEVHDMF-UHFFFAOYSA-N 0.000 description 1
- 108010003533 Viral Envelope Proteins Proteins 0.000 description 1
- 108010067390 Viral Proteins Proteins 0.000 description 1
- 108020000999 Viral RNA Proteins 0.000 description 1
- 244000000001 Virome Species 0.000 description 1
- 101000756604 Xenopus laevis Actin, cytoplasmic 1 Proteins 0.000 description 1
- 229920002494 Zein Polymers 0.000 description 1
- 230000001154 acute effect Effects 0.000 description 1
- 230000003044 adaptive effect Effects 0.000 description 1
- 239000000654 additive Substances 0.000 description 1
- 238000012382 advanced drug delivery Methods 0.000 description 1
- 238000013019 agitation Methods 0.000 description 1
- 235000004279 alanine Nutrition 0.000 description 1
- 150000001299 aldehydes Chemical class 0.000 description 1
- 229940072056 alginate Drugs 0.000 description 1
- 229920000615 alginic acid Polymers 0.000 description 1
- 235000010443 alginic acid Nutrition 0.000 description 1
- 229920003232 aliphatic polyester Polymers 0.000 description 1
- 125000000217 alkyl group Chemical group 0.000 description 1
- 125000002947 alkylene group Chemical group 0.000 description 1
- 230000004075 alteration Effects 0.000 description 1
- 150000001412 amines Chemical class 0.000 description 1
- 125000000539 amino acid group Chemical group 0.000 description 1
- 238000004458 analytical method Methods 0.000 description 1
- 125000000129 anionic group Chemical group 0.000 description 1
- 230000000692 anti-sense effect Effects 0.000 description 1
- 239000007864 aqueous solution Substances 0.000 description 1
- 230000037429 base substitution Effects 0.000 description 1
- 230000008901 benefit Effects 0.000 description 1
- 108010005774 beta-Galactosidase Proteins 0.000 description 1
- 239000003833 bile salt Substances 0.000 description 1
- 229940093761 bile salts Drugs 0.000 description 1
- 230000000975 bioactive effect Effects 0.000 description 1
- 230000004071 biological effect Effects 0.000 description 1
- 230000008827 biological function Effects 0.000 description 1
- 230000031018 biological processes and functions Effects 0.000 description 1
- 239000012472 biological sample Substances 0.000 description 1
- 230000005540 biological transmission Effects 0.000 description 1
- 229960002685 biotin Drugs 0.000 description 1
- YBJHBAHKTGYVGT-ZKWXMUAHSA-N biotin Natural products N1C(=O)N[C@@H]2[C@H](CCCCC(=O)O)SC[C@@H]21 YBJHBAHKTGYVGT-ZKWXMUAHSA-N 0.000 description 1
- 235000020958 biotin Nutrition 0.000 description 1
- 239000011616 biotin Substances 0.000 description 1
- 230000006287 biotinylation Effects 0.000 description 1
- 238000007413 biotinylation Methods 0.000 description 1
- 230000000903 blocking effect Effects 0.000 description 1
- 210000004369 blood Anatomy 0.000 description 1
- 239000008280 blood Substances 0.000 description 1
- PFKFTWBEEFSNDU-UHFFFAOYSA-N carbonyldiimidazole Chemical compound C1=CN=CN1C(=O)N1C=CN=C1 PFKFTWBEEFSNDU-UHFFFAOYSA-N 0.000 description 1
- 150000001732 carboxylic acid derivatives Chemical class 0.000 description 1
- 210000004413 cardiac myocyte Anatomy 0.000 description 1
- 239000002327 cardiovascular agent Substances 0.000 description 1
- 229940125692 cardiovascular agent Drugs 0.000 description 1
- 101150055766 cat gene Proteins 0.000 description 1
- 230000001925 catabolic effect Effects 0.000 description 1
- 125000002091 cationic group Chemical group 0.000 description 1
- 210000000170 cell membrane Anatomy 0.000 description 1
- 230000003833 cell viability Effects 0.000 description 1
- 230000001413 cellular effect Effects 0.000 description 1
- 230000005754 cellular signaling Effects 0.000 description 1
- 239000001913 cellulose Substances 0.000 description 1
- 229920002678 cellulose Polymers 0.000 description 1
- 210000003169 central nervous system Anatomy 0.000 description 1
- 239000013522 chelant Substances 0.000 description 1
- 239000013043 chemical agent Substances 0.000 description 1
- 229910052804 chromium Inorganic materials 0.000 description 1
- 239000011651 chromium Substances 0.000 description 1
- 238000010367 cloning Methods 0.000 description 1
- 229920001436 collagen Polymers 0.000 description 1
- 239000002299 complementary DNA Substances 0.000 description 1
- 238000002591 computed tomography Methods 0.000 description 1
- 238000012790 confirmation Methods 0.000 description 1
- 230000001276 controlling effect Effects 0.000 description 1
- 229910052802 copper Inorganic materials 0.000 description 1
- 239000010949 copper Substances 0.000 description 1
- 230000008878 coupling Effects 0.000 description 1
- 238000010168 coupling process Methods 0.000 description 1
- 238000005859 coupling reaction Methods 0.000 description 1
- 238000004132 cross linking Methods 0.000 description 1
- 239000003431 cross linking reagent Substances 0.000 description 1
- 238000012258 culturing Methods 0.000 description 1
- 235000018417 cysteine Nutrition 0.000 description 1
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 description 1
- 230000009089 cytolysis Effects 0.000 description 1
- 210000000805 cytoplasm Anatomy 0.000 description 1
- 230000001086 cytosolic effect Effects 0.000 description 1
- 230000003247 decreasing effect Effects 0.000 description 1
- 230000007123 defense Effects 0.000 description 1
- 230000003111 delayed effect Effects 0.000 description 1
- KXGVEGMKQFWNSR-LLQZFEROSA-N deoxycholic acid Chemical compound C([C@H]1CC2)[C@H](O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H]([C@@H](CCC(O)=O)C)[C@@]2(C)[C@@H](O)C1 KXGVEGMKQFWNSR-LLQZFEROSA-N 0.000 description 1
- 229960003964 deoxycholic acid Drugs 0.000 description 1
- 238000011161 development Methods 0.000 description 1
- 229960003957 dexamethasone Drugs 0.000 description 1
- 235000014113 dietary fatty acids Nutrition 0.000 description 1
- 238000009792 diffusion process Methods 0.000 description 1
- 238000010790 dilution Methods 0.000 description 1
- 239000012895 dilution Substances 0.000 description 1
- 208000037765 diseases and disorders Diseases 0.000 description 1
- 230000005782 double-strand break Effects 0.000 description 1
- 230000003828 downregulation Effects 0.000 description 1
- 238000002651 drug therapy Methods 0.000 description 1
- 230000007140 dysbiosis Effects 0.000 description 1
- 230000008482 dysregulation Effects 0.000 description 1
- 230000002900 effect on cell Effects 0.000 description 1
- 239000003792 electrolyte Substances 0.000 description 1
- 238000004520 electroporation Methods 0.000 description 1
- 239000000839 emulsion Substances 0.000 description 1
- 238000005516 engineering process Methods 0.000 description 1
- 230000026502 entry into host cell Effects 0.000 description 1
- 230000007071 enzymatic hydrolysis Effects 0.000 description 1
- 238000006047 enzymatic hydrolysis reaction Methods 0.000 description 1
- 208000037828 epithelial carcinoma Diseases 0.000 description 1
- 230000003628 erosive effect Effects 0.000 description 1
- 210000001808 exosome Anatomy 0.000 description 1
- 238000002474 experimental method Methods 0.000 description 1
- 238000001125 extrusion Methods 0.000 description 1
- 229930195729 fatty acid Natural products 0.000 description 1
- 239000000194 fatty acid Substances 0.000 description 1
- 150000004665 fatty acids Chemical class 0.000 description 1
- 238000000799 fluorescence microscopy Methods 0.000 description 1
- 238000001506 fluorescence spectroscopy Methods 0.000 description 1
- 238000002594 fluoroscopy Methods 0.000 description 1
- 238000005755 formation reaction Methods 0.000 description 1
- 125000000524 functional group Chemical group 0.000 description 1
- UIWYJDYFSGRHKR-UHFFFAOYSA-N gadolinium atom Chemical compound [Gd] UIWYJDYFSGRHKR-UHFFFAOYSA-N 0.000 description 1
- ZXQYGBMAQZUVMI-GCMPRSNUSA-N gamma-cyhalothrin Chemical compound CC1(C)[C@@H](\C=C(/Cl)C(F)(F)F)[C@H]1C(=O)O[C@H](C#N)C1=CC=CC(OC=2C=CC=CC=2)=C1 ZXQYGBMAQZUVMI-GCMPRSNUSA-N 0.000 description 1
- 238000012224 gene deletion Methods 0.000 description 1
- 230000030279 gene silencing Effects 0.000 description 1
- 238000001415 gene therapy Methods 0.000 description 1
- 108091006104 gene-regulatory proteins Proteins 0.000 description 1
- 102000034356 gene-regulatory proteins Human genes 0.000 description 1
- 230000007614 genetic variation Effects 0.000 description 1
- 150000004676 glycans Chemical class 0.000 description 1
- 210000002216 heart Anatomy 0.000 description 1
- 210000001357 hemopoietic progenitor cell Anatomy 0.000 description 1
- 230000006801 homologous recombination Effects 0.000 description 1
- 238000002744 homologous recombination Methods 0.000 description 1
- 239000012943 hotmelt Substances 0.000 description 1
- 230000007062 hydrolysis Effects 0.000 description 1
- 238000006460 hydrolysis reaction Methods 0.000 description 1
- 230000002209 hydrophobic effect Effects 0.000 description 1
- 125000002887 hydroxy group Chemical group [H]O* 0.000 description 1
- 230000033444 hydroxylation Effects 0.000 description 1
- 238000005805 hydroxylation reaction Methods 0.000 description 1
- 238000003119 immunoblot Methods 0.000 description 1
- 230000001976 improved effect Effects 0.000 description 1
- 230000006872 improvement Effects 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 230000000415 inactivating effect Effects 0.000 description 1
- 238000010348 incorporation Methods 0.000 description 1
- 238000011534 incubation Methods 0.000 description 1
- 230000006698 induction Effects 0.000 description 1
- 230000002757 inflammatory effect Effects 0.000 description 1
- 206010022000 influenza Diseases 0.000 description 1
- 230000004941 influx Effects 0.000 description 1
- 230000002401 inhibitory effect Effects 0.000 description 1
- 239000011147 inorganic material Substances 0.000 description 1
- 229940125396 insulin Drugs 0.000 description 1
- 230000017730 intein-mediated protein splicing Effects 0.000 description 1
- 230000010189 intracellular transport Effects 0.000 description 1
- 229910052742 iron Inorganic materials 0.000 description 1
- 238000002955 isolation Methods 0.000 description 1
- 210000003292 kidney cell Anatomy 0.000 description 1
- 230000003907 kidney function Effects 0.000 description 1
- 239000004310 lactic acid Substances 0.000 description 1
- 235000014655 lactic acid Nutrition 0.000 description 1
- 230000000670 limiting effect Effects 0.000 description 1
- AGBQKNBQESQNJD-UHFFFAOYSA-N lipoic acid Chemical compound OC(=O)CCCCC1CCSS1 AGBQKNBQESQNJD-UHFFFAOYSA-N 0.000 description 1
- 235000019136 lipoic acid Nutrition 0.000 description 1
- 230000007774 longterm Effects 0.000 description 1
- 238000004020 luminiscence type Methods 0.000 description 1
- 210000004698 lymphocyte Anatomy 0.000 description 1
- 239000011777 magnesium Substances 0.000 description 1
- 229910052749 magnesium Inorganic materials 0.000 description 1
- 125000005439 maleimidyl group Chemical group C1(C=CC(N1*)=O)=O 0.000 description 1
- 210000001161 mammalian embryo Anatomy 0.000 description 1
- 238000007726 management method Methods 0.000 description 1
- WPBNNNQJVZRUHP-UHFFFAOYSA-L manganese(2+);methyl n-[[2-(methoxycarbonylcarbamothioylamino)phenyl]carbamothioyl]carbamate;n-[2-(sulfidocarbothioylamino)ethyl]carbamodithioate Chemical compound [Mn+2].[S-]C(=S)NCCNC([S-])=S.COC(=O)NC(=S)NC1=CC=CC=C1NC(=S)NC(=O)OC WPBNNNQJVZRUHP-UHFFFAOYSA-L 0.000 description 1
- 239000003550 marker Substances 0.000 description 1
- 238000005259 measurement Methods 0.000 description 1
- 102000006240 membrane receptors Human genes 0.000 description 1
- 108020004084 membrane receptors Proteins 0.000 description 1
- 230000004060 metabolic process Effects 0.000 description 1
- 229960000485 methotrexate Drugs 0.000 description 1
- 244000005700 microbiome Species 0.000 description 1
- 238000001167 microscope projection photolithography Methods 0.000 description 1
- 210000000214 mouth Anatomy 0.000 description 1
- UZHSEJADLWPNLE-GRGSLBFTSA-N naloxone Chemical compound O=C([C@@H]1O2)CC[C@@]3(O)[C@H]4CC5=CC=C(O)C2=C5[C@@]13CCN4CC=C UZHSEJADLWPNLE-GRGSLBFTSA-N 0.000 description 1
- 229960004127 naloxone Drugs 0.000 description 1
- DQCKKXVULJGBQN-XFWGSAIBSA-N naltrexone Chemical compound N1([C@@H]2CC3=CC=C(C=4O[C@@H]5[C@](C3=4)([C@]2(CCC5=O)O)CC1)O)CC1CC1 DQCKKXVULJGBQN-XFWGSAIBSA-N 0.000 description 1
- 229960003086 naltrexone Drugs 0.000 description 1
- 229940042880 natural phospholipid Drugs 0.000 description 1
- 229920005615 natural polymer Polymers 0.000 description 1
- 239000006225 natural substrate Substances 0.000 description 1
- 210000003758 neuroeffector junction Anatomy 0.000 description 1
- 230000007935 neutral effect Effects 0.000 description 1
- 231100000252 nontoxic Toxicity 0.000 description 1
- 230000003000 nontoxic effect Effects 0.000 description 1
- 210000000633 nuclear envelope Anatomy 0.000 description 1
- 238000007899 nucleic acid hybridization Methods 0.000 description 1
- 210000003463 organelle Anatomy 0.000 description 1
- 150000002902 organometallic compounds Chemical class 0.000 description 1
- 230000003647 oxidation Effects 0.000 description 1
- 238000007254 oxidation reaction Methods 0.000 description 1
- 238000002638 palliative care Methods 0.000 description 1
- 210000000496 pancreas Anatomy 0.000 description 1
- 230000036961 partial effect Effects 0.000 description 1
- 239000008188 pellet Substances 0.000 description 1
- 210000003819 peripheral blood mononuclear cell Anatomy 0.000 description 1
- 230000035699 permeability Effects 0.000 description 1
- 230000000144 pharmacologic effect Effects 0.000 description 1
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 description 1
- 239000010452 phosphate Substances 0.000 description 1
- YHHSONZFOIEMCP-UHFFFAOYSA-O phosphocholine Chemical compound C[N+](C)(C)CCOP(O)(O)=O YHHSONZFOIEMCP-UHFFFAOYSA-O 0.000 description 1
- 108091008695 photoreceptors Proteins 0.000 description 1
- 230000000704 physical effect Effects 0.000 description 1
- 230000004962 physiological condition Effects 0.000 description 1
- 229920000111 poly(butyric acid) Polymers 0.000 description 1
- 229920001606 poly(lactic acid-co-glycolic acid) Polymers 0.000 description 1
- 229920001306 poly(lactide-co-caprolactone) Polymers 0.000 description 1
- 230000008488 polyadenylation Effects 0.000 description 1
- 229920000642 polymer Polymers 0.000 description 1
- 229920001282 polysaccharide Polymers 0.000 description 1
- 239000005017 polysaccharide Substances 0.000 description 1
- 229920002635 polyurethane Polymers 0.000 description 1
- 239000004814 polyurethane Substances 0.000 description 1
- 238000002600 positron emission tomography Methods 0.000 description 1
- 238000012545 processing Methods 0.000 description 1
- 108060006613 prolamin Proteins 0.000 description 1
- 230000000644 propagated effect Effects 0.000 description 1
- 238000011321 prophylaxis Methods 0.000 description 1
- 239000003207 proteasome inhibitor Substances 0.000 description 1
- 230000004853 protein function Effects 0.000 description 1
- ZAHRKKWIAAJSAO-UHFFFAOYSA-N rapamycin Natural products COCC(O)C(=C/C(C)C(=O)CC(OC(=O)C1CCCCN1C(=O)C(=O)C2(O)OC(CC(OC)C(=CC=CC=CC(C)CC(C)C(=O)C)C)CCC2C)C(C)CC3CCC(O)C(C3)OC)C ZAHRKKWIAAJSAO-UHFFFAOYSA-N 0.000 description 1
- 238000010188 recombinant method Methods 0.000 description 1
- 238000011160 research Methods 0.000 description 1
- 210000001525 retina Anatomy 0.000 description 1
- 230000001177 retroviral effect Effects 0.000 description 1
- 108091092562 ribozyme Proteins 0.000 description 1
- 238000012216 screening Methods 0.000 description 1
- 238000012163 sequencing technique Methods 0.000 description 1
- 230000000405 serological effect Effects 0.000 description 1
- 238000002603 single-photon emission computed tomography Methods 0.000 description 1
- 230000005783 single-strand break Effects 0.000 description 1
- 229960002930 sirolimus Drugs 0.000 description 1
- QFJCIRLUMZQUOT-HPLJOQBZSA-N sirolimus Chemical compound C1C[C@@H](O)[C@H](OC)C[C@@H]1C[C@@H](C)[C@H]1OC(=O)[C@@H]2CCCCN2C(=O)C(=O)[C@](O)(O2)[C@H](C)CC[C@H]2C[C@H](OC)/C(C)=C/C=C/C=C/[C@@H](C)C[C@@H](C)C(=O)[C@H](OC)[C@H](O)/C(C)=C/[C@@H](C)C(=O)C1 QFJCIRLUMZQUOT-HPLJOQBZSA-N 0.000 description 1
- 210000002027 skeletal muscle Anatomy 0.000 description 1
- 210000003491 skin Anatomy 0.000 description 1
- 238000005063 solubilization Methods 0.000 description 1
- 230000007928 solubilization Effects 0.000 description 1
- 239000002904 solvent Substances 0.000 description 1
- 238000000935 solvent evaporation Methods 0.000 description 1
- 230000009870 specific binding Effects 0.000 description 1
- 238000001694 spray drying Methods 0.000 description 1
- 230000006641 stabilisation Effects 0.000 description 1
- 238000011105 stabilization Methods 0.000 description 1
- 238000010561 standard procedure Methods 0.000 description 1
- 230000000946 synaptic effect Effects 0.000 description 1
- 229920001059 synthetic polymer Polymers 0.000 description 1
- 238000007910 systemic administration Methods 0.000 description 1
- 238000012360 testing method Methods 0.000 description 1
- 239000005460 tetrahydrofolate Substances 0.000 description 1
- 229960002663 thioctic acid Drugs 0.000 description 1
- 125000003396 thiol group Chemical group [H]S* 0.000 description 1
- 229960004072 thrombin Drugs 0.000 description 1
- 108091006106 transcriptional activators Proteins 0.000 description 1
- 230000009752 translational inhibition Effects 0.000 description 1
- 229940005605 valeric acid Drugs 0.000 description 1
- 239000004474 valine Substances 0.000 description 1
- 108700026220 vif Genes Proteins 0.000 description 1
- 230000004095 viral genome expression Effects 0.000 description 1
- 230000006656 viral protein synthesis Effects 0.000 description 1
- 238000011179 visual inspection Methods 0.000 description 1
- 238000012800 visualization Methods 0.000 description 1
- 239000005019 zein Substances 0.000 description 1
- 229940093612 zein Drugs 0.000 description 1
Images
Classifications
-
- 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
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
- C12N15/113—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
- C12N15/1136—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 against growth factors, growth regulators, cytokines, lymphokines or hormones
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2750/00021—Viruses as such, e.g. new isolates, mutants or their genomic sequences
-
- 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
- C12N2750/00022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- C12N2750/00041—Use of virus, viral particle or viral elements as a vector
- C12N2750/00043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Communicable Diseases (AREA)
- Endocrinology (AREA)
- Oncology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| KR1020257004957A KR20250028508A (ko) | 2017-06-13 | 2018-06-13 | 큐론을 포함하는 조성물 및 그의 용도 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762518898P | 2017-06-13 | 2017-06-13 | |
| US62/518,898 | 2017-06-13 | ||
| US201762597387P | 2017-12-11 | 2017-12-11 | |
| US62/597,387 | 2017-12-11 | ||
| US201862676730P | 2018-05-25 | 2018-05-25 | |
| US62/676,730 | 2018-05-25 | ||
| PCT/US2018/037379 WO2018232017A1 (en) | 2017-06-13 | 2018-06-13 | Compositions comprising curons and uses thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020257004957A Division KR20250028508A (ko) | 2017-06-13 | 2018-06-13 | 큐론을 포함하는 조성물 및 그의 용도 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20200038236A true KR20200038236A (ko) | 2020-04-10 |
Family
ID=62846247
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020207000972A Ceased KR20200038236A (ko) | 2017-06-13 | 2018-06-13 | 큐론을 포함하는 조성물 및 그의 용도 |
| KR1020257004957A Pending KR20250028508A (ko) | 2017-06-13 | 2018-06-13 | 큐론을 포함하는 조성물 및 그의 용도 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020257004957A Pending KR20250028508A (ko) | 2017-06-13 | 2018-06-13 | 큐론을 포함하는 조성물 및 그의 용도 |
Country Status (11)
| Country | Link |
|---|---|
| US (4) | US20200123203A1 (enExample) |
| EP (1) | EP3638797A1 (enExample) |
| JP (3) | JP2020524993A (enExample) |
| KR (2) | KR20200038236A (enExample) |
| CN (1) | CN111108208A (enExample) |
| AU (2) | AU2018285860A1 (enExample) |
| BR (1) | BR112019026226A2 (enExample) |
| CA (1) | CA3066750A1 (enExample) |
| IL (1) | IL271275A (enExample) |
| MX (2) | MX2019015018A (enExample) |
| WO (1) | WO2018232017A1 (enExample) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL280880B2 (en) | 2018-08-27 | 2025-04-01 | Regeneron Pharma | Using Raman Spectroscopy in Downstream Purification |
| CA3121528A1 (en) * | 2018-12-12 | 2020-06-18 | Flagship Pioneering Innovations V, Inc. | Anellosomes and methods of use |
| MX2021006943A (es) * | 2018-12-12 | 2021-11-17 | Flagship Pioneering Innovations V Inc | Anelosomas para suministrar modalidades terapéuticas secretadas. |
| US11166996B2 (en) | 2018-12-12 | 2021-11-09 | Flagship Pioneering Innovations V, Inc. | Anellovirus compositions and methods of use |
| WO2020123753A2 (en) * | 2018-12-12 | 2020-06-18 | Flagship Pioneering Innovations V, Inc. | Anellosomes for delivering intracellular therapeutic modalities |
| JP2022513459A (ja) * | 2018-12-12 | 2022-02-08 | フラッグシップ パイオニアリング イノベーションズ ブイ， インコーポレイテッド | タンパク質代替治療モダリティを送達するためのアネロソーム |
| CN114258398A (zh) | 2019-06-13 | 2022-03-29 | 总医院公司 | 工程化的人内源性病毒样颗粒及使用其递送至细胞的方法 |
| EP3999642A1 (en) | 2019-07-19 | 2022-05-25 | Flagship Pioneering Innovations VI, LLC | Recombinase compositions and methods of use |
| CA3186872A1 (en) * | 2020-06-12 | 2021-12-16 | Flagship Pioneering Innovations V, Inc. | Baculovirus expression systems |
| CA3186894A1 (en) * | 2020-06-12 | 2021-12-16 | Flagship Pioneering Innovations V, Inc. | Tandem anellovirus constructs |
| TW202221126A (zh) * | 2020-06-17 | 2022-06-01 | 美商旗艦先鋒創新公司 | 用於鑑別及特徵化指環病毒之方法及其用途 |
| BR112023001272A2 (pt) | 2020-07-24 | 2023-04-04 | Massachusetts Gen Hospital | Partículas semelhantes a vírus aprimoradas e métodos de uso das mesmas para entrega às células |
| US12018254B2 (en) * | 2020-07-27 | 2024-06-25 | Wisconsin Alumni Research Foundation | Methods of making unbiased phage libraries |
| JP2024501287A (ja) * | 2020-12-23 | 2024-01-11 | フラッグシップ パイオニアリング イノベーションズ ブイ， インコーポレイテッド | ＲＮＡを閉じ込めたアネロウイルス（Ａｎｅｌｌｏｖｉｒｕｓ）キャプシドのインビトロアセンブリ |
| AU2022216311A1 (en) * | 2021-02-08 | 2023-08-17 | Flagship Pioneering Innovations V, Inc. | Hybrid aav-anellovectors |
| EP4419677A4 (en) * | 2021-10-18 | 2025-09-24 | Flagship Pioneering Innovations Vii Llc | DNA COMPOSITIONS AND RELATED METHODS |
| TW202430215A (zh) | 2022-12-14 | 2024-08-01 | 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 | 用於將治療劑遞送至骨之組成物和方法 |
| WO2025194138A1 (en) | 2024-03-14 | 2025-09-18 | Tessera Therapeutics, Inc. | St1cas9 compositions and methods for modulating a genome |
Family Cites Families (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR751473A (fr) * | 1932-06-01 | 1933-09-04 | Poste d'incendie | |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US6693086B1 (en) | 1998-06-25 | 2004-02-17 | National Jewish Medical And Research Center | Systemic immune activation method using nucleic acid-lipid complexes |
| US6395472B1 (en) * | 1999-02-05 | 2002-05-28 | Abbott Laboratories | Methods of utilizing the TT virus |
| US7901708B2 (en) | 2002-06-28 | 2011-03-08 | Protiva Biotherapeutics, Inc. | Liposomal apparatus and manufacturing methods |
| SG190613A1 (en) | 2003-07-16 | 2013-06-28 | Protiva Biotherapeutics Inc | Lipid encapsulated interfering rna |
| EP1664316B1 (en) | 2003-09-15 | 2012-08-29 | Protiva Biotherapeutics Inc. | Polyethyleneglycol-modified lipid compounds and uses thereof |
| JP5243789B2 (ja) | 2004-03-15 | 2013-07-24 | シティ・オブ・ホープ | 二本鎖ｒｎａによる遺伝子発現の特異的阻害のための方法及び組成物 |
| JP4764426B2 (ja) | 2004-06-07 | 2011-09-07 | プロチバ バイオセラピューティクス インコーポレイティッド | カチオン性脂質および使用方法 |
| AU2005252273B2 (en) | 2004-06-07 | 2011-04-28 | Arbutus Biopharma Corporation | Lipid encapsulated interfering RNA |
| EP1768692B8 (en) | 2004-07-01 | 2015-06-17 | Yale University | Targeted and high density drug loaded polymeric materials |
| US7838658B2 (en) | 2005-10-20 | 2010-11-23 | Ian Maclachlan | siRNA silencing of filovirus gene expression |
| US8101741B2 (en) | 2005-11-02 | 2012-01-24 | Protiva Biotherapeutics, Inc. | Modified siRNA molecules and uses thereof |
| US7915399B2 (en) | 2006-06-09 | 2011-03-29 | Protiva Biotherapeutics, Inc. | Modified siRNA molecules and uses thereof |
| US8058069B2 (en) | 2008-04-15 | 2011-11-15 | Protiva Biotherapeutics, Inc. | Lipid formulations for nucleic acid delivery |
| CN105907756A (zh) | 2008-12-18 | 2016-08-31 | 戴瑟纳制药公司 | 延长的dicer酶底物和特异性抑制基因表达的方法 |
| WO2010093788A2 (en) | 2009-02-11 | 2010-08-19 | Dicerna Pharmaceuticals, Inc. | Multiplex dicer substrate rna interference molecules having joining sequences |
| US8603966B2 (en) | 2009-02-27 | 2013-12-10 | The Administrators Of The Tulane Educational Fund | Amino acid-based compounds, their methods of use, and methods of screening |
| EP2449114B9 (en) | 2009-07-01 | 2017-04-19 | Protiva Biotherapeutics Inc. | Novel lipid formulations for delivery of therapeutic agents to solid tumors |
| EP2449106B1 (en) | 2009-07-01 | 2015-04-08 | Protiva Biotherapeutics Inc. | Compositions and methods for silencing apolipoprotein b |
| US9676828B2 (en) * | 2010-06-23 | 2017-06-13 | Deutsches Krebsforschungszentrum | Rearranged TT virus molecules for use in diagnosis, prevention and treatment of cancer and autoimmunity |
| US9409987B2 (en) * | 2011-04-15 | 2016-08-09 | Compugen Ltd | Polypeptides and polynucleotides, and uses thereof for treatment of immune related disorders and cancer |
| FI3597749T3 (fi) | 2012-05-25 | 2023-10-09 | Univ California | Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten |
| KR101656236B1 (ko) | 2012-10-23 | 2016-09-12 | 주식회사 툴젠 | 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도 |
| PT2784162E (pt) | 2012-12-12 | 2015-08-27 | Broad Inst Inc | Engenharia de sistemas, métodos e composições guia otimizadas para a manipulação de sequências |
| EP4234696A3 (en) | 2012-12-12 | 2023-09-06 | The Broad Institute Inc. | Crispr-cas component systems, methods and compositions for sequence manipulation |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| 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 |
| 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 |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| EP3079726B1 (en) * | 2013-12-12 | 2018-12-05 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
| US9790490B2 (en) | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
-
2018
- 2018-06-13 US US16/622,146 patent/US20200123203A1/en not_active Abandoned
- 2018-06-13 CN CN201880051296.6A patent/CN111108208A/zh active Pending
- 2018-06-13 KR KR1020207000972A patent/KR20200038236A/ko not_active Ceased
- 2018-06-13 AU AU2018285860A patent/AU2018285860A1/en not_active Abandoned
- 2018-06-13 CA CA3066750A patent/CA3066750A1/en active Pending
- 2018-06-13 BR BR112019026226-1A patent/BR112019026226A2/pt unknown
- 2018-06-13 WO PCT/US2018/037379 patent/WO2018232017A1/en not_active Ceased
- 2018-06-13 KR KR1020257004957A patent/KR20250028508A/ko active Pending
- 2018-06-13 MX MX2019015018A patent/MX2019015018A/es unknown
- 2018-06-13 JP JP2019568601A patent/JP2020524993A/ja not_active Withdrawn
- 2018-06-13 EP EP18738411.0A patent/EP3638797A1/en active Pending
-
2019
- 2019-03-27 US US16/366,571 patent/US20190211361A1/en not_active Abandoned
- 2019-12-09 IL IL271275A patent/IL271275A/en unknown
- 2019-12-11 MX MX2023011657A patent/MX2023011657A/es unknown
-
2020
- 2020-01-16 US US16/744,363 patent/US20200385757A1/en not_active Abandoned
-
2022
- 2022-07-15 US US17/812,896 patent/US20230279423A1/en not_active Abandoned
- 2022-11-24 JP JP2022187239A patent/JP2023010961A/ja not_active Withdrawn
-
2024
- 2024-04-23 JP JP2024069618A patent/JP2024087003A/ja active Pending
-
2025
- 2025-01-24 AU AU2025200497A patent/AU2025200497A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| AU2025200497A1 (en) | 2025-04-10 |
| US20200123203A1 (en) | 2020-04-23 |
| AU2018285860A1 (en) | 2020-01-02 |
| MX2023011657A (es) | 2023-10-11 |
| BR112019026226A2 (pt) | 2020-06-30 |
| RU2020100074A3 (enExample) | 2022-02-08 |
| WO2018232017A1 (en) | 2018-12-20 |
| MX2019015018A (es) | 2020-09-10 |
| IL271275A (en) | 2020-01-30 |
| US20230279423A1 (en) | 2023-09-07 |
| EP3638797A1 (en) | 2020-04-22 |
| JP2023010961A (ja) | 2023-01-20 |
| RU2020100074A (ru) | 2021-08-03 |
| CN111108208A (zh) | 2020-05-05 |
| JP2020524993A (ja) | 2020-08-27 |
| JP2024087003A (ja) | 2024-06-28 |
| CA3066750A1 (en) | 2018-12-20 |
| US20200385757A1 (en) | 2020-12-10 |
| KR20250028508A (ko) | 2025-02-28 |
| US20190211361A1 (en) | 2019-07-11 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| KR20200038236A (ko) | 큐론을 포함하는 조성물 및 그의 용도 | |
| AU2018383712B2 (en) | Cpf1-related methods and compositions for gene editing | |
| CN110418647B (zh) | Rna指导的核酸修饰酶及其使用方法 | |
| AU2018240515B2 (en) | Nucleic acids encoding CRISPR-associated proteins and uses thereof | |
| KR20230111189A (ko) | 재프로그램 가능한 iscb 뉴클레아제 및 이의 용도 | |
| KR20230053735A (ko) | 게놈의 조정을 위한 개선된 방법 및 조성물 | |
| KR20210131309A (ko) | 분비형 치료 양식을 운반하기 위한 아넬로좀 | |
| KR20230057487A (ko) | 게놈 조정을 위한 방법 및 조성물 | |
| KR20210125990A (ko) | 단백질 대체 치료 양식을 운반하기 위한 아넬로좀 | |
| AU2020270508A1 (en) | C/EBP alpha short activating RNA compositions and methods of use | |
| KR20210131310A (ko) | 아넬로좀 및 사용 방법 | |
| KR20200116933A (ko) | 인간 심근세포에서 디스트로핀 돌연변이를 교정하기 위한 조성물 및 방법 | |
| AU2018200955A1 (en) | Optimal maize loci | |
| AU2018201717A1 (en) | Optimal maize loci | |
| AU2018203259A1 (en) | Recombinant HCMV and RhCMV vectors and uses thereof | |
| WO2018136396A2 (en) | Crisprs | |
| KR20200058509A (ko) | Attr 아밀로이드증의 ttr 유전자 편집 및 치료를 위한 조성물 및 방법 | |
| KR20210060429A (ko) | 적응 면역의 조절을 위한 조성물 및 방법 | |
| KR20220044811A (ko) | Crispr/cas13을 사용하는 표적화된 트랜스-이어맞추기 | |
| KR20210131308A (ko) | 세포내 치료 양식을 운반하기 위한 아넬로좀 | |
| AU2019403015B2 (en) | Nuclease-mediated repeat expansion | |
| KR20230127221A (ko) | Cag 반복 질환을 치료하기 위한 rna 표적화 조성물및 방법 | |
| KR20230036109A (ko) | 바큘로바이러스 발현 시스템 | |
| KR20240035382A (ko) | 아데노바이러스 유전자 요법 벡터 | |
| KR20230145051A (ko) | 아데노바이러스 유전자 치료 벡터 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PA0105 | International application |
Patent event date: 20200113 Patent event code: PA01051R01D Comment text: International Patent Application |
|
| PG1501 | Laying open of application | ||
| PA0201 | Request for examination |
Patent event code: PA02012R01D Patent event date: 20210611 Comment text: Request for Examination of Application |
|
| E902 | Notification of reason for refusal | ||
| PE0902 | Notice of grounds for rejection |
Comment text: Notification of reason for refusal Patent event date: 20231019 Patent event code: PE09021S01D |
|
| E601 | Decision to refuse application | ||
| PE0601 | Decision on rejection of patent |
Patent event date: 20241016 Comment text: Decision to Refuse Application Patent event code: PE06012S01D |