JP6993966B2 - ポリマーベシクルを使用する遺伝子編集ツールを送達するための組成物および方法 - Google Patents
ポリマーベシクルを使用する遺伝子編集ツールを送達するための組成物および方法 Download PDFInfo
- Publication number
- JP6993966B2 JP6993966B2 JP2018520385A JP2018520385A JP6993966B2 JP 6993966 B2 JP6993966 B2 JP 6993966B2 JP 2018520385 A JP2018520385 A JP 2018520385A JP 2018520385 A JP2018520385 A JP 2018520385A JP 6993966 B2 JP6993966 B2 JP 6993966B2
- Authority
- JP
- Japan
- Prior art keywords
- dna
- gene editing
- rna
- composition
- host cell
- 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.)
- Active
Links
- 239000000203 mixture Substances 0.000 title claims description 117
- 238000010362 genome editing Methods 0.000 title claims description 93
- 238000000034 method Methods 0.000 title claims description 31
- 229920000642 polymer Polymers 0.000 title description 45
- 102000053602 DNA Human genes 0.000 claims description 155
- 108020004414 DNA Proteins 0.000 claims description 155
- 229920000575 polymersome Polymers 0.000 claims description 130
- 150000007523 nucleic acids Chemical class 0.000 claims description 87
- 108090000623 proteins and genes Proteins 0.000 claims description 86
- 102000039446 nucleic acids Human genes 0.000 claims description 81
- 108020004707 nucleic acids Proteins 0.000 claims description 81
- 235000018102 proteins Nutrition 0.000 claims description 62
- 102000004169 proteins and genes Human genes 0.000 claims description 62
- 101710163270 Nuclease Proteins 0.000 claims description 55
- 229920002477 rna polymer Polymers 0.000 claims description 54
- 108091033409 CRISPR Proteins 0.000 claims description 44
- 102000004190 Enzymes Human genes 0.000 claims description 40
- 108090000790 Enzymes Proteins 0.000 claims description 40
- 108020005004 Guide RNA Proteins 0.000 claims description 29
- 235000004252 protein component Nutrition 0.000 claims description 24
- 102000008579 Transposases Human genes 0.000 claims description 21
- 108010020764 Transposases Proteins 0.000 claims description 21
- 229920003171 Poly (ethylene oxide) Polymers 0.000 claims description 20
- 238000003776 cleavage reaction Methods 0.000 claims description 20
- 230000007017 scission Effects 0.000 claims description 20
- 229920001059 synthetic polymer Polymers 0.000 claims description 19
- 239000013612 plasmid Substances 0.000 claims description 16
- 230000033616 DNA repair Effects 0.000 claims description 14
- 230000002209 hydrophobic effect Effects 0.000 claims description 14
- 229920001400 block copolymer Polymers 0.000 claims description 13
- 230000000295 complement effect Effects 0.000 claims description 11
- KAKZBPTYRLMSJV-UHFFFAOYSA-N Butadiene Chemical compound C=CC=C KAKZBPTYRLMSJV-UHFFFAOYSA-N 0.000 claims description 9
- 238000012239 gene modification Methods 0.000 claims description 6
- 108091029865 Exogenous DNA Proteins 0.000 claims description 4
- 108091081548 Palindromic sequence Proteins 0.000 claims description 4
- 230000005017 genetic modification Effects 0.000 claims description 4
- 235000013617 genetically modified food Nutrition 0.000 claims description 4
- 238000005538 encapsulation Methods 0.000 description 85
- 210000004027 cell Anatomy 0.000 description 76
- 238000009472 formulation Methods 0.000 description 43
- 239000005090 green fluorescent protein Substances 0.000 description 39
- 230000000052 comparative effect Effects 0.000 description 27
- 108091003079 Bovine Serum Albumin Proteins 0.000 description 22
- 230000017854 proteolysis Effects 0.000 description 21
- 239000013604 expression vector Substances 0.000 description 20
- 229920001223 polyethylene glycol Polymers 0.000 description 20
- 239000002202 Polyethylene glycol Substances 0.000 description 18
- 125000003729 nucleotide group Chemical group 0.000 description 18
- 230000008439 repair process Effects 0.000 description 18
- 229940098773 bovine serum albumin Drugs 0.000 description 17
- 239000002773 nucleotide Substances 0.000 description 17
- 239000013598 vector Substances 0.000 description 17
- 102100030856 Myoglobin Human genes 0.000 description 15
- 108010062374 Myoglobin Proteins 0.000 description 15
- 108091028043 Nucleic acid sequence Proteins 0.000 description 15
- 239000000470 constituent Substances 0.000 description 15
- 230000036571 hydration Effects 0.000 description 14
- 238000006703 hydration reaction Methods 0.000 description 14
- BASFCYQUMIYNBI-UHFFFAOYSA-N platinum Chemical compound [Pt] BASFCYQUMIYNBI-UHFFFAOYSA-N 0.000 description 14
- 229920001606 poly(lactic acid-co-glycolic acid) Polymers 0.000 description 13
- 108010043121 Green Fluorescent Proteins Proteins 0.000 description 12
- 102000004144 Green Fluorescent Proteins Human genes 0.000 description 12
- 238000011068 loading method Methods 0.000 description 12
- 229920001610 polycaprolactone Polymers 0.000 description 11
- 239000000725 suspension Substances 0.000 description 11
- 241000193996 Streptococcus pyogenes Species 0.000 description 10
- 229920000359 diblock copolymer Polymers 0.000 description 10
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 10
- 238000003780 insertion Methods 0.000 description 10
- 230000037431 insertion Effects 0.000 description 10
- 208000002267 Anti-neutrophil cytoplasmic antibody-associated vasculitis Diseases 0.000 description 9
- 241000702421 Dependoparvovirus Species 0.000 description 9
- 102000010292 Peptide Elongation Factor 1 Human genes 0.000 description 9
- 108010077524 Peptide Elongation Factor 1 Proteins 0.000 description 9
- -1 aliphatic poly (anhydride Chemical class 0.000 description 9
- 230000027455 binding Effects 0.000 description 9
- 238000002354 inductively-coupled plasma atomic emission spectroscopy Methods 0.000 description 9
- 239000002105 nanoparticle Substances 0.000 description 9
- 229920000747 poly(lactic acid) Polymers 0.000 description 9
- 239000000243 solution Substances 0.000 description 9
- 239000010409 thin film Substances 0.000 description 9
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 description 8
- 238000010790 dilution Methods 0.000 description 8
- 239000012895 dilution Substances 0.000 description 8
- 201000010099 disease Diseases 0.000 description 8
- 230000014509 gene expression Effects 0.000 description 8
- 229940065514 poly(lactide) Drugs 0.000 description 8
- 229920000166 polytrimethylene carbonate Polymers 0.000 description 8
- 208000024891 symptom Diseases 0.000 description 8
- 230000005782 double-strand break Effects 0.000 description 7
- 238000001727 in vivo Methods 0.000 description 7
- 239000002502 liposome Substances 0.000 description 7
- 239000000178 monomer Substances 0.000 description 7
- 230000037361 pathway Effects 0.000 description 7
- 229910052697 platinum Inorganic materials 0.000 description 7
- 108090000765 processed proteins & peptides Proteins 0.000 description 7
- 150000001875 compounds Chemical class 0.000 description 6
- 229920001577 copolymer Polymers 0.000 description 6
- 230000006870 function Effects 0.000 description 6
- 239000000463 material Substances 0.000 description 6
- 239000012528 membrane Substances 0.000 description 6
- 239000002777 nucleoside Substances 0.000 description 6
- 239000013603 viral vector Substances 0.000 description 6
- 230000007018 DNA scission Effects 0.000 description 5
- YMWUJEATGCHHMB-UHFFFAOYSA-N Dichloromethane Chemical compound ClCCl YMWUJEATGCHHMB-UHFFFAOYSA-N 0.000 description 5
- 108091034117 Oligonucleotide Proteins 0.000 description 5
- 102000018120 Recombinases Human genes 0.000 description 5
- 108010091086 Recombinases Proteins 0.000 description 5
- 241000700605 Viruses Species 0.000 description 5
- 230000015572 biosynthetic process Effects 0.000 description 5
- 150000001720 carbohydrates Chemical class 0.000 description 5
- 239000003795 chemical substances by application Substances 0.000 description 5
- 150000002632 lipids Chemical class 0.000 description 5
- 238000012986 modification Methods 0.000 description 5
- 230000006780 non-homologous end joining Effects 0.000 description 5
- 150000003833 nucleoside derivatives Chemical class 0.000 description 5
- 239000008194 pharmaceutical composition Substances 0.000 description 5
- 239000000047 product Substances 0.000 description 5
- RKDVKSZUMVYZHH-UHFFFAOYSA-N 1,4-dioxane-2,5-dione Chemical compound O=C1COC(=O)CO1 RKDVKSZUMVYZHH-UHFFFAOYSA-N 0.000 description 4
- 102000012330 Integrases Human genes 0.000 description 4
- 108010061833 Integrases Proteins 0.000 description 4
- 229920001710 Polyorthoester Polymers 0.000 description 4
- IQFYYKKMVGJFEH-XLPZGREQSA-N Thymidine Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-XLPZGREQSA-N 0.000 description 4
- 238000010521 absorption reaction Methods 0.000 description 4
- 239000012190 activator Substances 0.000 description 4
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 description 4
- 230000006378 damage Effects 0.000 description 4
- 229920006237 degradable polymer Polymers 0.000 description 4
- 150000002148 esters Chemical class 0.000 description 4
- 238000001415 gene therapy Methods 0.000 description 4
- 230000003834 intracellular effect Effects 0.000 description 4
- 238000004519 manufacturing process Methods 0.000 description 4
- 230000007246 mechanism Effects 0.000 description 4
- 108020004999 messenger RNA Proteins 0.000 description 4
- 230000004048 modification Effects 0.000 description 4
- 230000035772 mutation Effects 0.000 description 4
- 239000002745 poly(ortho ester) Substances 0.000 description 4
- 229920002627 poly(phosphazenes) Polymers 0.000 description 4
- 238000002798 spectrophotometry method Methods 0.000 description 4
- 239000000126 substance Substances 0.000 description 4
- 238000012546 transfer Methods 0.000 description 4
- 230000003612 virological effect Effects 0.000 description 4
- ZDTFMPXQUSBYRL-UUOKFMHZSA-N 2-Aminoadenosine Chemical compound C12=NC(N)=NC(N)=C2N=CN1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O ZDTFMPXQUSBYRL-UUOKFMHZSA-N 0.000 description 3
- 239000004606 Fillers/Extenders Substances 0.000 description 3
- 240000007019 Oxalis corniculata Species 0.000 description 3
- 238000004847 absorption spectroscopy Methods 0.000 description 3
- 230000004075 alteration Effects 0.000 description 3
- 238000003556 assay Methods 0.000 description 3
- 230000008901 benefit Effects 0.000 description 3
- 238000011161 development Methods 0.000 description 3
- 230000012361 double-strand break repair Effects 0.000 description 3
- 230000000694 effects Effects 0.000 description 3
- 230000030279 gene silencing Effects 0.000 description 3
- 229920001519 homopolymer Polymers 0.000 description 3
- 230000009545 invasion Effects 0.000 description 3
- 238000002156 mixing Methods 0.000 description 3
- 239000002245 particle Substances 0.000 description 3
- 229920002961 polybutylene succinate Polymers 0.000 description 3
- 239000004631 polybutylene succinate Substances 0.000 description 3
- 102000040430 polynucleotide Human genes 0.000 description 3
- 108091033319 polynucleotide Proteins 0.000 description 3
- 239000002157 polynucleotide Substances 0.000 description 3
- 230000008569 process Effects 0.000 description 3
- 229920006395 saturated elastomer Polymers 0.000 description 3
- 241000894007 species Species 0.000 description 3
- 235000000346 sugar Nutrition 0.000 description 3
- 230000001225 therapeutic effect Effects 0.000 description 3
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 description 3
- 102000016938 Catalase Human genes 0.000 description 2
- 108010053835 Catalase Proteins 0.000 description 2
- 108091028732 Concatemer Proteins 0.000 description 2
- 206010010356 Congenital anomaly Diseases 0.000 description 2
- 230000006820 DNA synthesis Effects 0.000 description 2
- LCGLNKUTAGEVQW-UHFFFAOYSA-N Dimethyl ether Chemical compound COC LCGLNKUTAGEVQW-UHFFFAOYSA-N 0.000 description 2
- AOJJSUZBOXZQNB-TZSSRYMLSA-N Doxorubicin Chemical compound O([C@H]1C[C@@](O)(CC=2C(O)=C3C(=O)C=4C=CC=C(C=4C(=O)C3=C(O)C=21)OC)C(=O)CO)[C@H]1C[C@H](N)[C@H](O)[C@H](C)O1 AOJJSUZBOXZQNB-TZSSRYMLSA-N 0.000 description 2
- 108010042407 Endonucleases Proteins 0.000 description 2
- 208000031448 Genomic Instability Diseases 0.000 description 2
- NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-UUOKFMHZSA-N 0.000 description 2
- XEEYBQQBJWHFJM-UHFFFAOYSA-N Iron Chemical compound [Fe] XEEYBQQBJWHFJM-UHFFFAOYSA-N 0.000 description 2
- 241001465754 Metazoa Species 0.000 description 2
- 108010059712 Pronase Proteins 0.000 description 2
- 108020004682 Single-Stranded DNA Proteins 0.000 description 2
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 description 2
- 230000001154 acute effect Effects 0.000 description 2
- 229920003232 aliphatic polyester Polymers 0.000 description 2
- 238000000137 annealing Methods 0.000 description 2
- 238000001479 atomic absorption spectroscopy Methods 0.000 description 2
- 229920002988 biodegradable polymer Polymers 0.000 description 2
- 239000004621 biodegradable polymer Substances 0.000 description 2
- 230000001413 cellular effect Effects 0.000 description 2
- 230000002759 chromosomal effect Effects 0.000 description 2
- 210000000349 chromosome Anatomy 0.000 description 2
- 125000004122 cyclic group Chemical group 0.000 description 2
- 208000035475 disorder Diseases 0.000 description 2
- 229940079593 drug Drugs 0.000 description 2
- 239000003814 drug Substances 0.000 description 2
- 238000012377 drug delivery Methods 0.000 description 2
- 238000002296 dynamic light scattering Methods 0.000 description 2
- 210000001163 endosome Anatomy 0.000 description 2
- 238000005516 engineering process Methods 0.000 description 2
- 230000002349 favourable effect Effects 0.000 description 2
- 239000012634 fragment Substances 0.000 description 2
- 230000002068 genetic effect Effects 0.000 description 2
- 238000000338 in vitro Methods 0.000 description 2
- 230000002401 inhibitory effect Effects 0.000 description 2
- 230000010354 integration Effects 0.000 description 2
- 239000011159 matrix material Substances 0.000 description 2
- 238000005259 measurement Methods 0.000 description 2
- 239000002088 nanocapsule Substances 0.000 description 2
- 150000004713 phosphodiesters Chemical class 0.000 description 2
- 235000010482 polyoxyethylene sorbitan monooleate Nutrition 0.000 description 2
- 229920000053 polysorbate 80 Polymers 0.000 description 2
- 239000013636 protein dimer Substances 0.000 description 2
- 230000010076 replication Effects 0.000 description 2
- 125000006850 spacer group Chemical group 0.000 description 2
- 238000004611 spectroscopical analysis Methods 0.000 description 2
- 150000008163 sugars Chemical class 0.000 description 2
- 238000003786 synthesis reaction Methods 0.000 description 2
- 230000008685 targeting Effects 0.000 description 2
- 210000001519 tissue Anatomy 0.000 description 2
- 231100000419 toxicity Toxicity 0.000 description 2
- 230000001988 toxicity Effects 0.000 description 2
- 241000701161 unidentified adenovirus Species 0.000 description 2
- 241001430294 unidentified retrovirus Species 0.000 description 2
- RIFDKYBNWNPCQK-IOSLPCCCSA-N (2r,3s,4r,5r)-2-(hydroxymethyl)-5-(6-imino-3-methylpurin-9-yl)oxolane-3,4-diol Chemical compound C1=2N(C)C=NC(=N)C=2N=CN1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O RIFDKYBNWNPCQK-IOSLPCCCSA-N 0.000 description 1
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 1
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 1
- RKSLVDIXBGWPIS-UAKXSSHOSA-N 1-[(2r,3r,4s,5r)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-5-iodopyrimidine-2,4-dione Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C(I)=C1 RKSLVDIXBGWPIS-UAKXSSHOSA-N 0.000 description 1
- QLOCVMVCRJOTTM-TURQNECASA-N 1-[(2r,3r,4s,5r)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-5-prop-1-ynylpyrimidine-2,4-dione Chemical compound O=C1NC(=O)C(C#CC)=CN1[C@H]1[C@H](O)[C@H](O)[C@@H](CO)O1 QLOCVMVCRJOTTM-TURQNECASA-N 0.000 description 1
- PISWNSOQFZRVJK-XLPZGREQSA-N 1-[(2r,4s,5r)-4-hydroxy-5-(hydroxymethyl)oxolan-2-yl]-5-methyl-2-sulfanylidenepyrimidin-4-one Chemical compound S=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 PISWNSOQFZRVJK-XLPZGREQSA-N 0.000 description 1
- UHDGCWIWMRVCDJ-UHFFFAOYSA-N 1-beta-D-Xylofuranosyl-NH-Cytosine Natural products O=C1N=C(N)C=CN1C1C(O)C(O)C(CO)O1 UHDGCWIWMRVCDJ-UHFFFAOYSA-N 0.000 description 1
- YKBGVTZYEHREMT-KVQBGUIXSA-N 2'-deoxyguanosine Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@H]1C[C@H](O)[C@@H](CO)O1 YKBGVTZYEHREMT-KVQBGUIXSA-N 0.000 description 1
- JRYMOPZHXMVHTA-DAGMQNCNSA-N 2-amino-7-[(2r,3r,4s,5r)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-1h-pyrrolo[2,3-d]pyrimidin-4-one Chemical compound C1=CC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O JRYMOPZHXMVHTA-DAGMQNCNSA-N 0.000 description 1
- NYHNVHGFPZAZGA-UHFFFAOYSA-N 2-hydroxyhexanoic acid Chemical compound CCCCC(O)C(O)=O NYHNVHGFPZAZGA-UHFFFAOYSA-N 0.000 description 1
- IYBOGQYZTIIPNI-UHFFFAOYSA-N 2-methylhexano-6-lactone Chemical compound CC1CCCCOC1=O IYBOGQYZTIIPNI-UHFFFAOYSA-N 0.000 description 1
- XXSIICQLPUAUDF-TURQNECASA-N 4-amino-1-[(2r,3r,4s,5r)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-5-prop-1-ynylpyrimidin-2-one Chemical compound O=C1N=C(N)C(C#CC)=CN1[C@H]1[C@H](O)[C@H](O)[C@@H](CO)O1 XXSIICQLPUAUDF-TURQNECASA-N 0.000 description 1
- 229940090248 4-hydroxybenzoic acid Drugs 0.000 description 1
- 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 description 1
- FHIDNBAQOFJWCA-UAKXSSHOSA-N 5-fluorouridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C(F)=C1 FHIDNBAQOFJWCA-UAKXSSHOSA-N 0.000 description 1
- KDOPAZIWBAHVJB-UHFFFAOYSA-N 5h-pyrrolo[3,2-d]pyrimidine Chemical compound C1=NC=C2NC=CC2=N1 KDOPAZIWBAHVJB-UHFFFAOYSA-N 0.000 description 1
- BXJHWYVXLGLDMZ-UHFFFAOYSA-N 6-O-methylguanine Chemical compound COC1=NC(N)=NC2=C1NC=N2 BXJHWYVXLGLDMZ-UHFFFAOYSA-N 0.000 description 1
- UEHOMUNTZPIBIL-UUOKFMHZSA-N 6-amino-9-[(2r,3r,4s,5r)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-7h-purin-8-one Chemical compound O=C1NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O UEHOMUNTZPIBIL-UUOKFMHZSA-N 0.000 description 1
- HCAJQHYUCKICQH-VPENINKCSA-N 8-Oxo-7,8-dihydro-2'-deoxyguanosine Chemical compound C1=2NC(N)=NC(=O)C=2NC(=O)N1[C@H]1C[C@H](O)[C@@H](CO)O1 HCAJQHYUCKICQH-VPENINKCSA-N 0.000 description 1
- HDZZVAMISRMYHH-UHFFFAOYSA-N 9beta-Ribofuranosyl-7-deazaadenin Natural products C1=CC=2C(N)=NC=NC=2N1C1OC(CO)C(O)C1O HDZZVAMISRMYHH-UHFFFAOYSA-N 0.000 description 1
- 241000894006 Bacteria Species 0.000 description 1
- DWRXFEITVBNRMK-UHFFFAOYSA-N Beta-D-1-Arabinofuranosylthymine Natural products O=C1NC(=O)C(C)=CN1C1C(O)C(O)C(CO)O1 DWRXFEITVBNRMK-UHFFFAOYSA-N 0.000 description 1
- 201000004569 Blindness Diseases 0.000 description 1
- FERIUCNNQQJTOY-UHFFFAOYSA-M Butyrate Chemical compound CCCC([O-])=O FERIUCNNQQJTOY-UHFFFAOYSA-M 0.000 description 1
- FERIUCNNQQJTOY-UHFFFAOYSA-N Butyric acid Natural products CCCC(O)=O FERIUCNNQQJTOY-UHFFFAOYSA-N 0.000 description 1
- 239000002126 C01EB10 - Adenosine Substances 0.000 description 1
- 108091079001 CRISPR RNA Proteins 0.000 description 1
- BVKZGUZCCUSVTD-UHFFFAOYSA-L Carbonate Chemical compound [O-]C([O-])=O BVKZGUZCCUSVTD-UHFFFAOYSA-L 0.000 description 1
- 108091060290 Chromatid Proteins 0.000 description 1
- 108020004705 Codon Proteins 0.000 description 1
- 108010051219 Cre recombinase Proteins 0.000 description 1
- MIKUYHXYGGJMLM-GIMIYPNGSA-N Crotonoside Natural products C1=NC2=C(N)NC(=O)N=C2N1[C@H]1O[C@@H](CO)[C@H](O)[C@@H]1O MIKUYHXYGGJMLM-GIMIYPNGSA-N 0.000 description 1
- UHDGCWIWMRVCDJ-PSQAKQOGSA-N Cytidine Natural products O=C1N=C(N)C=CN1[C@@H]1[C@@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-PSQAKQOGSA-N 0.000 description 1
- NYHBQMYGNKIUIF-UHFFFAOYSA-N D-guanosine Natural products C1=2NC(N)=NC(=O)C=2N=CN1C1OC(CO)C(O)C1O NYHBQMYGNKIUIF-UHFFFAOYSA-N 0.000 description 1
- 230000009946 DNA mutation Effects 0.000 description 1
- 230000008265 DNA repair mechanism Effects 0.000 description 1
- 230000004568 DNA-binding Effects 0.000 description 1
- 108010008532 Deoxyribonuclease I Proteins 0.000 description 1
- 102000007260 Deoxyribonuclease I Human genes 0.000 description 1
- 206010013457 Dissociation Diseases 0.000 description 1
- 102100031780 Endonuclease Human genes 0.000 description 1
- 102000004533 Endonucleases Human genes 0.000 description 1
- 241001663880 Gammaretrovirus Species 0.000 description 1
- 108700039691 Genetic Promoter Regions Proteins 0.000 description 1
- 102000001554 Hemoglobins Human genes 0.000 description 1
- 108010054147 Hemoglobins Proteins 0.000 description 1
- 108091027305 Heteroduplex Proteins 0.000 description 1
- 101001000998 Homo sapiens Protein phosphatase 1 regulatory subunit 12C Proteins 0.000 description 1
- 208000026350 Inborn Genetic disease Diseases 0.000 description 1
- 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 description 1
- 241000713666 Lentivirus Species 0.000 description 1
- 241000124008 Mammalia Species 0.000 description 1
- 208000024556 Mendelian disease Diseases 0.000 description 1
- 108060004795 Methyltransferase Proteins 0.000 description 1
- 206010028980 Neoplasm Diseases 0.000 description 1
- 108091005461 Nucleic proteins Proteins 0.000 description 1
- 108091005804 Peptidases Proteins 0.000 description 1
- 229920000475 Poly(ethylene oxide)-block-polycaprolactone Polymers 0.000 description 1
- 229920000954 Polyglycolide Polymers 0.000 description 1
- 239000004365 Protease Substances 0.000 description 1
- 102100035620 Protein phosphatase 1 regulatory subunit 12C Human genes 0.000 description 1
- 108020004511 Recombinant DNA Proteins 0.000 description 1
- 102100037486 Reverse transcriptase/ribonuclease H Human genes 0.000 description 1
- 108020004688 Small Nuclear RNA Proteins 0.000 description 1
- 102000039471 Small Nuclear RNA Human genes 0.000 description 1
- 108020004459 Small interfering RNA Proteins 0.000 description 1
- 241000187392 Streptomyces griseus Species 0.000 description 1
- 108020004566 Transfer RNA Proteins 0.000 description 1
- 241000251539 Vertebrata <Metazoa> Species 0.000 description 1
- 208000027418 Wounds and injury Diseases 0.000 description 1
- 108010017070 Zinc Finger Nucleases Proteins 0.000 description 1
- 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 1
- 239000002253 acid Substances 0.000 description 1
- 230000002378 acidificating effect Effects 0.000 description 1
- 239000013543 active substance Substances 0.000 description 1
- 231100000403 acute toxicity Toxicity 0.000 description 1
- 230000006978 adaptation Effects 0.000 description 1
- 210000005006 adaptive immune system Anatomy 0.000 description 1
- 229960005305 adenosine Drugs 0.000 description 1
- 230000002411 adverse Effects 0.000 description 1
- 230000002776 aggregation Effects 0.000 description 1
- 238000004220 aggregation Methods 0.000 description 1
- 206010001902 amaurosis Diseases 0.000 description 1
- 150000001413 amino acids Chemical class 0.000 description 1
- 238000004458 analytical method Methods 0.000 description 1
- 238000013459 approach Methods 0.000 description 1
- PYMYPHUHKUWMLA-WDCZJNDASA-N arabinose Chemical compound OC[C@@H](O)[C@@H](O)[C@H](O)C=O PYMYPHUHKUWMLA-WDCZJNDASA-N 0.000 description 1
- PYMYPHUHKUWMLA-UHFFFAOYSA-N arabinose Natural products OCC(O)C(O)C(O)C=O PYMYPHUHKUWMLA-UHFFFAOYSA-N 0.000 description 1
- 210000004507 artificial chromosome Anatomy 0.000 description 1
- QVGXLLKOCUKJST-UHFFFAOYSA-N atomic oxygen Chemical compound [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 description 1
- SRBFZHDQGSBBOR-UHFFFAOYSA-N beta-D-Pyranose-Lyxose Natural products OC1COC(O)C(O)C1O SRBFZHDQGSBBOR-UHFFFAOYSA-N 0.000 description 1
- IQFYYKKMVGJFEH-UHFFFAOYSA-N beta-L-thymidine Natural products O=C1NC(=O)C(C)=CN1C1OC(CO)C(O)C1 IQFYYKKMVGJFEH-UHFFFAOYSA-N 0.000 description 1
- DRTQHJPVMGBUCF-PSQAKQOGSA-N beta-L-uridine Natural products O[C@H]1[C@@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-PSQAKQOGSA-N 0.000 description 1
- 229920000229 biodegradable polyester Polymers 0.000 description 1
- 239000004622 biodegradable polyester Substances 0.000 description 1
- 230000008827 biological function Effects 0.000 description 1
- 239000012620 biological material Substances 0.000 description 1
- 230000008512 biological response Effects 0.000 description 1
- 230000017531 blood circulation Effects 0.000 description 1
- 239000007853 buffer solution Substances 0.000 description 1
- 201000011510 cancer Diseases 0.000 description 1
- 210000000234 capsid Anatomy 0.000 description 1
- 239000000969 carrier Substances 0.000 description 1
- 125000002091 cationic group Chemical group 0.000 description 1
- 210000005056 cell body Anatomy 0.000 description 1
- 230000032823 cell division Effects 0.000 description 1
- 210000003855 cell nucleus Anatomy 0.000 description 1
- 230000008859 change Effects 0.000 description 1
- 239000003153 chemical reaction reagent Substances 0.000 description 1
- 210000004756 chromatid Anatomy 0.000 description 1
- DQLATGHUWYMOKM-UHFFFAOYSA-L cisplatin Chemical compound N[Pt](N)(Cl)Cl DQLATGHUWYMOKM-UHFFFAOYSA-L 0.000 description 1
- 229960004316 cisplatin Drugs 0.000 description 1
- 238000010367 cloning Methods 0.000 description 1
- 238000010276 construction Methods 0.000 description 1
- UHDGCWIWMRVCDJ-ZAKLUEHWSA-N cytidine Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-ZAKLUEHWSA-N 0.000 description 1
- 210000000172 cytosol Anatomy 0.000 description 1
- 230000007402 cytotoxic response Effects 0.000 description 1
- 230000002950 deficient Effects 0.000 description 1
- 238000012217 deletion Methods 0.000 description 1
- 230000037430 deletion Effects 0.000 description 1
- 238000004925 denaturation Methods 0.000 description 1
- 230000036425 denaturation Effects 0.000 description 1
- 230000001419 dependent effect Effects 0.000 description 1
- 239000003599 detergent Substances 0.000 description 1
- 238000000502 dialysis Methods 0.000 description 1
- 238000010494 dissociation reaction Methods 0.000 description 1
- 230000005593 dissociations Effects 0.000 description 1
- 208000018459 dissociative disease Diseases 0.000 description 1
- 238000009826 distribution Methods 0.000 description 1
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 description 1
- 229960004679 doxorubicin Drugs 0.000 description 1
- 239000012636 effector Substances 0.000 description 1
- 230000008030 elimination Effects 0.000 description 1
- 238000003379 elimination reaction Methods 0.000 description 1
- 210000002889 endothelial cell Anatomy 0.000 description 1
- QPADNTZLUBYNEN-UHFFFAOYSA-N etallobarbital Chemical compound C=CCC1(CC)C(=O)NC(=O)NC1=O QPADNTZLUBYNEN-UHFFFAOYSA-N 0.000 description 1
- 229940117927 ethylene oxide Drugs 0.000 description 1
- 210000003527 eukaryotic cell Anatomy 0.000 description 1
- 238000002474 experimental method Methods 0.000 description 1
- 239000012467 final product Substances 0.000 description 1
- 238000013467 fragmentation Methods 0.000 description 1
- 238000006062 fragmentation reaction Methods 0.000 description 1
- 231100000221 frame shift mutation induction Toxicity 0.000 description 1
- 230000037433 frameshift Effects 0.000 description 1
- 108020001507 fusion proteins Proteins 0.000 description 1
- 102000037865 fusion proteins Human genes 0.000 description 1
- 238000001476 gene delivery Methods 0.000 description 1
- 230000009395 genetic defect Effects 0.000 description 1
- 238000010353 genetic engineering Methods 0.000 description 1
- 229940029575 guanosine Drugs 0.000 description 1
- 150000003278 haem Chemical group 0.000 description 1
- 238000010438 heat treatment Methods 0.000 description 1
- 230000002440 hepatic effect Effects 0.000 description 1
- 210000003494 hepatocyte Anatomy 0.000 description 1
- 230000005745 host immune response Effects 0.000 description 1
- 210000003917 human chromosome Anatomy 0.000 description 1
- 238000009396 hybridization Methods 0.000 description 1
- 230000007062 hydrolysis Effects 0.000 description 1
- 238000006460 hydrolysis reaction Methods 0.000 description 1
- 150000002433 hydrophilic molecules Chemical class 0.000 description 1
- 125000004356 hydroxy functional group Chemical group O* 0.000 description 1
- 210000002865 immune cell Anatomy 0.000 description 1
- 230000028993 immune response Effects 0.000 description 1
- 230000036039 immunity Effects 0.000 description 1
- 230000005847 immunogenicity Effects 0.000 description 1
- 230000006872 improvement Effects 0.000 description 1
- 238000011065 in-situ storage Methods 0.000 description 1
- 230000000415 inactivating effect Effects 0.000 description 1
- 238000010348 incorporation Methods 0.000 description 1
- 238000009616 inductively coupled plasma Methods 0.000 description 1
- 230000000977 initiatory effect Effects 0.000 description 1
- 208000014674 injury Diseases 0.000 description 1
- 150000002484 inorganic compounds Chemical class 0.000 description 1
- 229910010272 inorganic material Inorganic materials 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 230000005865 ionizing radiation Effects 0.000 description 1
- 229910052742 iron Inorganic materials 0.000 description 1
- 230000031700 light absorption Effects 0.000 description 1
- 230000007774 longterm Effects 0.000 description 1
- 238000004949 mass spectrometry Methods 0.000 description 1
- 238000001840 matrix-assisted laser desorption--ionisation time-of-flight mass spectrometry Methods 0.000 description 1
- 238000000691 measurement method Methods 0.000 description 1
- 230000001404 mediated effect Effects 0.000 description 1
- 238000002703 mutagenesis Methods 0.000 description 1
- 231100000350 mutagenesis Toxicity 0.000 description 1
- 210000002569 neuron Anatomy 0.000 description 1
- 230000003472 neutralizing effect Effects 0.000 description 1
- 108091027963 non-coding RNA Proteins 0.000 description 1
- 102000042567 non-coding RNA Human genes 0.000 description 1
- 230000009871 nonspecific binding Effects 0.000 description 1
- 125000003835 nucleoside group Chemical group 0.000 description 1
- 210000004940 nucleus Anatomy 0.000 description 1
- 150000002894 organic compounds Chemical class 0.000 description 1
- 229910052760 oxygen Inorganic materials 0.000 description 1
- 239000001301 oxygen Substances 0.000 description 1
- FJKROLUGYXJWQN-UHFFFAOYSA-N papa-hydroxy-benzoic acid Natural products OC(=O)C1=CC=C(O)C=C1 FJKROLUGYXJWQN-UHFFFAOYSA-N 0.000 description 1
- 230000007918 pathogenicity Effects 0.000 description 1
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 description 1
- 230000004962 physiological condition Effects 0.000 description 1
- HRGDZIGMBDGFTC-UHFFFAOYSA-N platinum(2+) Chemical compound [Pt+2] HRGDZIGMBDGFTC-UHFFFAOYSA-N 0.000 description 1
- 229920000218 poly(hydroxyvalerate) Polymers 0.000 description 1
- 229920002647 polyamide Polymers 0.000 description 1
- 239000000843 powder Substances 0.000 description 1
- 230000000750 progressive effect Effects 0.000 description 1
- 238000002731 protein assay Methods 0.000 description 1
- 230000006916 protein interaction Effects 0.000 description 1
- 239000012460 protein solution Substances 0.000 description 1
- 238000000746 purification Methods 0.000 description 1
- 239000003642 reactive oxygen metabolite Substances 0.000 description 1
- 238000003259 recombinant expression Methods 0.000 description 1
- 230000006798 recombination Effects 0.000 description 1
- 238000005215 recombination Methods 0.000 description 1
- 230000009467 reduction Effects 0.000 description 1
- 230000001105 regulatory effect Effects 0.000 description 1
- 230000008263 repair mechanism Effects 0.000 description 1
- 230000004044 response Effects 0.000 description 1
- 230000002207 retinal effect Effects 0.000 description 1
- 238000010839 reverse transcription Methods 0.000 description 1
- 238000001338 self-assembly Methods 0.000 description 1
- 238000013207 serial dilution Methods 0.000 description 1
- 210000002027 skeletal muscle Anatomy 0.000 description 1
- 239000002002 slurry Substances 0.000 description 1
- 150000003384 small molecules Chemical class 0.000 description 1
- JVBXVOWTABLYPX-UHFFFAOYSA-L sodium dithionite Chemical compound [Na+].[Na+].[O-]S(=O)S([O-])=O JVBXVOWTABLYPX-UHFFFAOYSA-L 0.000 description 1
- 238000000527 sonication Methods 0.000 description 1
- 238000001228 spectrum Methods 0.000 description 1
- 230000010473 stable expression Effects 0.000 description 1
- 238000003860 storage Methods 0.000 description 1
- 231100000456 subacute toxicity Toxicity 0.000 description 1
- 238000006467 substitution reaction Methods 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
- 238000009210 therapy by ultrasound Methods 0.000 description 1
- 229940104230 thymidine Drugs 0.000 description 1
- 230000002103 transcriptional effect Effects 0.000 description 1
- 230000005945 translocation Effects 0.000 description 1
- 229920000428 triblock copolymer Polymers 0.000 description 1
- 125000003258 trimethylene group Chemical group [H]C([H])([*:2])C([H])([H])C([H])([H])[*:1] 0.000 description 1
- HDZZVAMISRMYHH-KCGFPETGSA-N tubercidin Chemical compound C1=CC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O HDZZVAMISRMYHH-KCGFPETGSA-N 0.000 description 1
- 238000000870 ultraviolet spectroscopy Methods 0.000 description 1
- 238000011144 upstream manufacturing Methods 0.000 description 1
- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 description 1
- 229940045145 uridine Drugs 0.000 description 1
- 210000005166 vasculature Anatomy 0.000 description 1
Images
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/5005—Wall or coating material
- A61K9/5021—Organic macromolecular compounds
- A61K9/5031—Organic macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, poly(lactide-co-glycolide)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
- A61K9/1273—Polymersomes; Liposomes with polymerisable or polymerised bilayer-forming substances
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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]
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Dispersion Chemistry (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Enzymes And Modification Thereof (AREA)
- Polyesters Or Polycarbonates (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562187942P | 2015-07-02 | 2015-07-02 | |
| US62/187,942 | 2015-07-02 | ||
| US201662322346P | 2016-04-14 | 2016-04-14 | |
| US62/322,346 | 2016-04-14 | ||
| US15/199,021 US20170000743A1 (en) | 2015-07-02 | 2016-06-30 | Compositions and Methods for Delivery of Gene Editing Tools Using Polymeric Vesicles |
| US15/199,021 | 2016-06-30 | ||
| PCT/US2016/040673 WO2017004509A1 (en) | 2015-07-02 | 2016-07-01 | Compositions and methods for delivery of gene editing tools using polymeric vesicles |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019105213A Division JP2019165745A (ja) | 2015-07-02 | 2019-06-05 | ポリマーベシクルを使用する遺伝子編集ツールを送達するための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018532408A JP2018532408A (ja) | 2018-11-08 |
| JP2018532408A5 JP2018532408A5 (https=) | 2019-07-11 |
| JP6993966B2 true JP6993966B2 (ja) | 2022-02-04 |
Family
ID=57609243
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018520385A Active JP6993966B2 (ja) | 2015-07-02 | 2016-07-01 | ポリマーベシクルを使用する遺伝子編集ツールを送達するための組成物および方法 |
| JP2019105213A Pending JP2019165745A (ja) | 2015-07-02 | 2019-06-05 | ポリマーベシクルを使用する遺伝子編集ツールを送達するための組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019105213A Pending JP2019165745A (ja) | 2015-07-02 | 2019-06-05 | ポリマーベシクルを使用する遺伝子編集ツールを送達するための組成物および方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20170000743A1 (https=) |
| EP (1) | EP3317414A4 (https=) |
| JP (2) | JP6993966B2 (https=) |
| CN (1) | CN108699563A (https=) |
| AU (1) | AU2016288237B2 (https=) |
| CA (1) | CA2991109A1 (https=) |
| HK (1) | HK1255209A1 (https=) |
| WO (1) | WO2017004509A1 (https=) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10456452B2 (en) | 2015-07-02 | 2019-10-29 | Poseida Therapeutics, Inc. | Compositions and methods for improved encapsulation of functional proteins in polymeric vesicles |
| US11213594B2 (en) | 2016-04-29 | 2022-01-04 | Poseida Therapeutics, Inc. | Poly(histidine)-based micelles for complexation and delivery of proteins and nucleic acids |
| CA3036926C (en) | 2016-09-30 | 2022-05-31 | Poseida Therapeutics, Inc. | Modified stem cell memory t cells, methods of making and methods of using same |
| US20190119636A1 (en) | 2017-10-23 | 2019-04-25 | Poseida Therapeutics, Inc. | Modified stem cell memory t cells, methods of making and methods of using same |
| ES3051391T3 (en) | 2016-10-06 | 2025-12-29 | Poseida Therapeutics Inc | Inducible caspases and methods for use |
| JP2020523383A (ja) | 2017-06-14 | 2020-08-06 | ダナ−ファーバー キャンサー インスティテュート,インコーポレイテッド | B細胞成熟抗原(bcma)指向性ナノ粒子 |
| WO2018232356A1 (en) | 2017-06-15 | 2018-12-20 | The Regents Of The University Of California | Targeted non-viral dna insertions |
| US10329543B2 (en) | 2017-10-23 | 2019-06-25 | Poseida Therapeutics, Inc. | Modified stem cell memory T cells, methods of making and methods of using same |
| JP7101419B2 (ja) | 2017-10-27 | 2022-07-15 | 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 | 内因性t細胞受容体の標的置換 |
| CA3124103A1 (en) | 2018-12-20 | 2020-06-25 | Poseida Therapeutics, Inc. | Nanotransposon compositions and methods of use |
| US20220372105A1 (en) | 2019-09-05 | 2022-11-24 | Poseida Therapeutics, Inc. | Allogeneic cell compositions and methods of use |
| AU2020407235A1 (en) | 2019-12-20 | 2022-07-07 | Poseida Therapeutics, Inc. | Anti-MUC1 compositions and methods of use |
| WO2021183795A1 (en) | 2020-03-11 | 2021-09-16 | Poseida Therapeutics, Inc. | Chimeric stimulatory receptors and methods of use in t cell activation and differentiation |
| GB202004254D0 (en) | 2020-03-24 | 2020-05-06 | Puridify Ltd | Characterization of gene therapy vectors |
| EP4298205A1 (en) | 2021-02-23 | 2024-01-03 | Poseida Therapeutics, Inc. | Genetically modified induced pluripotent stem cells and methods of use thereof |
| CN117355607A (zh) * | 2021-04-13 | 2024-01-05 | 阿森纳生物科学公司 | 非病毒同源性介导的末端连接 |
| EP4413141A1 (en) | 2021-10-04 | 2024-08-14 | Poseida Therapeutics, Inc. | Transposon compositions and methods of use thereof |
| JP2024537991A (ja) | 2021-10-14 | 2024-10-18 | アーセナル バイオサイエンシズ インコーポレイテッド | 共発現されるshRNAと論理ゲートシステムとを有する免疫細胞 |
| EP4482967A1 (en) | 2022-02-23 | 2025-01-01 | Poseida Therapeutics, Inc. | Genetically modified cells and methods of use thereof |
| WO2023193665A1 (zh) * | 2022-04-07 | 2023-10-12 | 威海纽兰生物科技有限公司 | 基于细胞外囊泡酯酶响应药物递送载体及其制备方法和用途 |
| EP4646189A1 (en) * | 2023-01-06 | 2025-11-12 | Vianautis Bio Limited | Polymersomes for delivery of nucleic acid cargoes |
| EP4669746A1 (en) | 2023-02-21 | 2025-12-31 | Poseida Therapeutics, Inc. | COMPOSITIONS AND GENOMIC EDITING PROCESSES |
| KR20250166122A (ko) | 2023-02-21 | 2025-11-27 | 포세이다 테라퓨틱스, 인크. | 게놈 편집을 위한 조성물 및 방법 |
| CN121335979A (zh) | 2023-05-17 | 2026-01-13 | 波西达治疗公司 | 靶向hbg1和hbg2的组合物及其使用方法 |
| WO2025096980A1 (en) | 2023-11-02 | 2025-05-08 | Poseida Therapeutics, Inc. | Compositions targeting klkb1 and methods of use thereof |
| WO2025221789A1 (en) | 2024-04-16 | 2025-10-23 | Poseida Therapeutics, Inc. | Compositions and methods for use in car/tcr cell therapies |
| WO2026080393A1 (en) | 2024-10-07 | 2026-04-16 | Poseida Therapeutics, Inc. | Compositions and methods for use in car cell therapies |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2014093635A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation |
| WO2014093622A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| WO2015191693A2 (en) | 2014-06-10 | 2015-12-17 | Massachusetts Institute Of Technology | Method for gene editing |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2001089579A2 (en) * | 2000-05-19 | 2001-11-29 | Regents Of The University Of Minnesota | Composition for delivery of compounds to cells |
| US20040265835A1 (en) * | 2003-06-30 | 2004-12-30 | Lemaster David M. | Method of sorting vesicle-entrapped, coupled nucleic acid-protein displays |
| CA2756670A1 (en) * | 2009-03-26 | 2010-09-30 | The Regents Of The University Of California | Mesenchymal stem cells producing inhibitory rna for disease modification |
| TWI576432B (zh) * | 2009-04-07 | 2017-04-01 | 陶氏農業科學公司 | 序列特異性核酸酶之奈米粒子媒介式遞送技術 |
| WO2011106376A2 (en) * | 2010-02-23 | 2011-09-01 | The General Hospital Corporation | Use of microvesicles in the treatment of medical conditions |
| CA2792035A1 (en) * | 2010-03-02 | 2011-09-09 | Vindico Nanobio Technology, Inc. | Compositions and methods for treating or preventing immuno-inflammatory disease |
| US20140363496A1 (en) | 2011-01-07 | 2014-12-11 | Vindico NanoBio Technology Inc. | Compositions and Methods for Inducing Nanoparticle-mediated Microvascular Embolization of Tumors |
| AU2012249390B2 (en) * | 2011-04-27 | 2017-03-30 | Amyris, Inc. | Methods for genomic modification |
| RU2662932C2 (ru) * | 2013-03-14 | 2018-07-31 | Карибо Биосайенсиз, Инк. | Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты |
| WO2014194190A1 (en) * | 2013-05-30 | 2014-12-04 | The Penn State Research Foundation | Gene targeting and genetic modification of plants via rna-guided genome editing |
-
2016
- 2016-06-30 US US15/199,021 patent/US20170000743A1/en not_active Abandoned
- 2016-07-01 EP EP16818879.5A patent/EP3317414A4/en not_active Withdrawn
- 2016-07-01 CA CA2991109A patent/CA2991109A1/en active Pending
- 2016-07-01 AU AU2016288237A patent/AU2016288237B2/en not_active Ceased
- 2016-07-01 WO PCT/US2016/040673 patent/WO2017004509A1/en not_active Ceased
- 2016-07-01 JP JP2018520385A patent/JP6993966B2/ja active Active
- 2016-07-01 HK HK18114337.3A patent/HK1255209A1/zh unknown
- 2016-07-01 CN CN201680050998.3A patent/CN108699563A/zh active Pending
-
2019
- 2019-06-05 JP JP2019105213A patent/JP2019165745A/ja active Pending
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2014093635A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation |
| WO2014093622A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| WO2015191693A2 (en) | 2014-06-10 | 2015-12-17 | Massachusetts Institute Of Technology | Method for gene editing |
Non-Patent Citations (4)
| Title |
|---|
| ACCOUNTS OF CHEMICAL RESEARCH (2011) Vol.44, No.10, pp.1039-1049 |
| Langmuir (2012) Vol.28, pp.12816-12830 |
| Pharm. Res. (2016) Vol.33, pp.573-589 (Published online: 27 October 2015) |
| PNAS (2013) Vol.110, No.25, pp.E2279-E2287 |
Also Published As
| Publication number | Publication date |
|---|---|
| EP3317414A4 (en) | 2019-03-13 |
| EP3317414A1 (en) | 2018-05-09 |
| CN108699563A (zh) | 2018-10-23 |
| CA2991109A1 (en) | 2017-01-05 |
| AU2016288237A1 (en) | 2018-01-25 |
| WO2017004509A1 (en) | 2017-01-05 |
| JP2019165745A (ja) | 2019-10-03 |
| US20170000743A1 (en) | 2017-01-05 |
| JP2018532408A (ja) | 2018-11-08 |
| HK1255209A1 (zh) | 2019-08-09 |
| AU2016288237B2 (en) | 2022-04-14 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP6993966B2 (ja) | ポリマーベシクルを使用する遺伝子編集ツールを送達するための組成物および方法 | |
| US11213594B2 (en) | Poly(histidine)-based micelles for complexation and delivery of proteins and nucleic acids | |
| Li et al. | CRISPR/Cas9 therapeutics: progress and prospects | |
| Damase et al. | The limitless future of RNA therapeutics | |
| Xu et al. | Delivery of CRISPR/Cas9 for therapeutic genome editing | |
| Ryu et al. | Effective PEI-mediated delivery of CRISPR-Cas9 complex for targeted gene therapy | |
| Givens et al. | Nanoparticle-based delivery of CRISPR/Cas9 genome-editing therapeutics | |
| Sahel et al. | CRISPR/Cas9 genome editing for tissue‐specific in vivo targeting: nanomaterials and translational perspective | |
| Ashok et al. | Lipid-and polymer-based nanoparticle systems for the delivery of CRISPR/Cas9 | |
| Allemailem et al. | Current updates of CRISPR/Cas9‐mediated genome editing and targeting within tumor cells: an innovative strategy of cancer management | |
| WO2019126589A1 (en) | Micelles for complexation and delivery of proteins and nucleic acids | |
| JP2018532408A5 (https=) | ||
| Onuma et al. | Lipid nanoparticle-based ribonucleoprotein delivery for in vivo genome editing | |
| TW202333747A (zh) | 調節pcsk9之方法及其用途 | |
| Khirallah et al. | Clinical progress in genome-editing technology and in vivo delivery techniques | |
| Rostami et al. | Exploring advanced CRISPR delivery technologies for therapeutic genome editing | |
| Lei et al. | Nanomaterials-assisted gene editing and synthetic biology for optimizing the treatment of pulmonary diseases | |
| Wang et al. | Advancing cancer gene therapy: the emerging role of nanoparticle delivery systems | |
| JP2024150579A (ja) | ゲノム配列変異を有する細胞死滅誘導用組成物及び該組成物を用いた細胞死滅誘導方法 | |
| Tuyen Ho et al. | Bioinspired and biomimetic gene delivery systems | |
| Eksi et al. | Nanodelivery in gene therapy: unlocking the potential for precision medicine | |
| Kanduri et al. | Current advances toward the encapsulation of Cas9 | |
| Mozafari et al. | Importance of divalent cations in nanolipoplex gene delivery | |
| Saw et al. | Nanoparticles‐Mediated CRISPR/Cas Gene Editing Delivery System | |
| Singh et al. | Advancements in RNA-based therapies from bench to bedside |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20190605 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20190605 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20200603 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20200828 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20201202 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20210520 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20210805 |
|
| TRDD | Decision of grant or rejection written | ||
| A01 | Written decision to grant a patent or to grant a registration (utility model) |
Free format text: JAPANESE INTERMEDIATE CODE: A01 Effective date: 20211208 |
|
| A61 | First payment of annual fees (during grant procedure) |
Free format text: JAPANESE INTERMEDIATE CODE: A61 Effective date: 20211210 |
|
| R150 | Certificate of patent or registration of utility model |
Ref document number: 6993966 Country of ref document: JP Free format text: JAPANESE INTERMEDIATE CODE: R150 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |