ES2467156T3 - Una biblioteca de genes estructurales modificados o de partículas modificadas de cápside útiles para la identificación de clones virales con tropismo celular deseado - Google Patents
Una biblioteca de genes estructurales modificados o de partículas modificadas de cápside útiles para la identificación de clones virales con tropismo celular deseadoInfo
- Publication number
- ES2467156T3 ES2467156T3 ES02787962.6T ES02787962T ES2467156T3 ES 2467156 T3 ES2467156 T3 ES 2467156T3 ES 02787962 T ES02787962 T ES 02787962T ES 2467156 T3 ES2467156 T3 ES 2467156T3
- Authority
- ES
- Spain
- Prior art keywords
- nucleic acid
- insert
- library
- aav
- cells
- 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.)
- Expired - Lifetime
Links
- 108090000623 proteins and genes Proteins 0.000 title claims abstract description 115
- 230000003612 virological effect Effects 0.000 title claims description 56
- 210000000234 capsid Anatomy 0.000 title claims description 49
- 239000002245 particle Substances 0.000 title claims description 25
- 230000005101 cell tropism Effects 0.000 title description 2
- 150000007523 nucleic acids Chemical class 0.000 claims abstract description 85
- 150000001413 amino acids Chemical class 0.000 claims abstract description 81
- 238000003780 insertion Methods 0.000 claims abstract description 73
- 230000037431 insertion Effects 0.000 claims abstract description 73
- 108020004707 nucleic acids Proteins 0.000 claims abstract description 58
- 102000039446 nucleic acids Human genes 0.000 claims abstract description 58
- 238000000034 method Methods 0.000 claims abstract description 54
- 241000700605 Viruses Species 0.000 claims abstract description 47
- 238000004806 packaging method and process Methods 0.000 claims abstract description 34
- 108090000565 Capsid Proteins Proteins 0.000 claims abstract description 27
- 102100023321 Ceruloplasmin Human genes 0.000 claims abstract description 27
- 241000125945 Protoparvovirus Species 0.000 claims abstract description 27
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims abstract description 17
- 238000004519 manufacturing process Methods 0.000 claims abstract description 17
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims abstract description 14
- 241000702421 Dependoparvovirus Species 0.000 claims abstract description 13
- 230000015572 biosynthetic process Effects 0.000 claims abstract description 10
- 101710172711 Structural protein Proteins 0.000 claims abstract description 8
- 101710118046 RNA-directed RNA polymerase Proteins 0.000 claims abstract 9
- 125000003275 alpha amino acid group Chemical group 0.000 claims abstract 7
- 210000002845 virion Anatomy 0.000 claims description 83
- 101150044789 Cap gene Proteins 0.000 claims description 41
- 108020004705 Codon Proteins 0.000 claims description 34
- 239000013612 plasmid Substances 0.000 claims description 29
- 102000004169 proteins and genes Human genes 0.000 claims description 22
- 230000006870 function Effects 0.000 claims description 20
- 230000035772 mutation Effects 0.000 claims description 17
- 239000002773 nucleotide Substances 0.000 claims description 16
- 125000003729 nucleotide group Chemical group 0.000 claims description 16
- 102000006495 integrins Human genes 0.000 claims description 13
- 108010044426 integrins Proteins 0.000 claims description 13
- 102000008055 Heparan Sulfate Proteoglycans Human genes 0.000 claims description 12
- 229920002971 Heparan sulfate Polymers 0.000 claims description 12
- 108090000054 Syndecan-2 Proteins 0.000 claims description 12
- 230000010076 replication Effects 0.000 claims description 11
- 108020004414 DNA Proteins 0.000 claims description 10
- 238000012986 modification Methods 0.000 claims description 10
- 230000004048 modification Effects 0.000 claims description 10
- 238000011144 upstream manufacturing Methods 0.000 claims description 10
- 239000013607 AAV vector Substances 0.000 claims description 9
- 101710197658 Capsid protein VP1 Proteins 0.000 claims description 9
- 238000006467 substitution reaction Methods 0.000 claims description 9
- 238000005215 recombination Methods 0.000 claims description 8
- 230000006798 recombination Effects 0.000 claims description 8
- 101710081079 Minor spike protein H Proteins 0.000 claims description 7
- 108091016585 CD44 antigen Proteins 0.000 claims description 6
- 101001040800 Homo sapiens Integral membrane protein GPR180 Proteins 0.000 claims description 6
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims description 6
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 5
- 230000001629 suppression Effects 0.000 claims description 5
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 4
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 4
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 4
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 4
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 4
- 108091008794 FGF receptors Proteins 0.000 claims description 4
- 210000004899 c-terminal region Anatomy 0.000 claims description 4
- 238000006243 chemical reaction Methods 0.000 claims description 4
- 238000012217 deletion Methods 0.000 claims description 4
- 230000037430 deletion Effects 0.000 claims description 4
- 239000013603 viral vector Substances 0.000 claims description 4
- 102000044168 Fibroblast Growth Factor Receptor Human genes 0.000 claims description 3
- 238000012856 packing Methods 0.000 claims description 3
- 101710136297 Protein VP2 Proteins 0.000 claims description 2
- 101710106388 Structural protein VP1 Proteins 0.000 claims description 2
- 230000002378 acidificating effect Effects 0.000 claims description 2
- 101710121537 mRNA (guanine-N(7))-methyltransferase Proteins 0.000 claims description 2
- 108091008146 restriction endonucleases Proteins 0.000 claims description 2
- 101710132601 Capsid protein Proteins 0.000 claims 7
- 101710108545 Viral protein 1 Proteins 0.000 claims 7
- 210000004027 cell Anatomy 0.000 description 241
- 238000010361 transduction Methods 0.000 description 65
- 230000026683 transduction Effects 0.000 description 65
- 208000015181 infectious disease Diseases 0.000 description 56
- 239000013598 vector Substances 0.000 description 48
- 210000002966 serum Anatomy 0.000 description 37
- 108090000765 processed proteins & peptides Proteins 0.000 description 36
- 102000004196 processed proteins & peptides Human genes 0.000 description 26
- 238000006386 neutralization reaction Methods 0.000 description 23
- 208000002267 Anti-neutrophil cytoplasmic antibody-associated vasculitis Diseases 0.000 description 22
- 208000010839 B-cell chronic lymphocytic leukemia Diseases 0.000 description 21
- 102000005962 receptors Human genes 0.000 description 21
- 108020003175 receptors Proteins 0.000 description 21
- 238000002474 experimental method Methods 0.000 description 16
- 230000003472 neutralizing effect Effects 0.000 description 15
- 108010043121 Green Fluorescent Proteins Proteins 0.000 description 14
- 102000004144 Green Fluorescent Proteins Human genes 0.000 description 14
- 239000005090 green fluorescent protein Substances 0.000 description 14
- IYMAXBFPHPZYIK-BQBZGAKWSA-N Arg-Gly-Asp Chemical group NC(N)=NCCC[C@H](N)C(=O)NCC(=O)N[C@@H](CC(O)=O)C(O)=O IYMAXBFPHPZYIK-BQBZGAKWSA-N 0.000 description 13
- 238000010790 dilution Methods 0.000 description 11
- 239000012895 dilution Substances 0.000 description 11
- 239000003446 ligand Substances 0.000 description 11
- 230000002829 reductive effect Effects 0.000 description 11
- 230000014509 gene expression Effects 0.000 description 10
- 238000011534 incubation Methods 0.000 description 10
- 238000002360 preparation method Methods 0.000 description 10
- HTTJABKRGRZYRN-UHFFFAOYSA-N Heparin Chemical compound OC1C(NC(=O)C)C(O)OC(COS(O)(=O)=O)C1OC1C(OS(O)(=O)=O)C(O)C(OC2C(C(OS(O)(=O)=O)C(OC3C(C(O)C(O)C(O3)C(O)=O)OS(O)(=O)=O)C(CO)O2)NS(O)(=O)=O)C(C(O)=O)O1 HTTJABKRGRZYRN-UHFFFAOYSA-N 0.000 description 9
- 238000005516 engineering process Methods 0.000 description 9
- 229920000669 heparin Polymers 0.000 description 9
- 229960002897 heparin Drugs 0.000 description 9
- 230000010415 tropism Effects 0.000 description 9
- 230000008901 benefit Effects 0.000 description 8
- 230000001413 cellular effect Effects 0.000 description 8
- 238000001415 gene therapy Methods 0.000 description 8
- 238000012546 transfer Methods 0.000 description 8
- 238000013459 approach Methods 0.000 description 7
- 238000003556 assay Methods 0.000 description 6
- 230000008859 change Effects 0.000 description 6
- 238000010367 cloning Methods 0.000 description 6
- 230000000694 effects Effects 0.000 description 6
- 229920001184 polypeptide Polymers 0.000 description 6
- 238000012163 sequencing technique Methods 0.000 description 6
- 241000701161 unidentified adenovirus Species 0.000 description 6
- 238000004458 analytical method Methods 0.000 description 5
- 238000005119 centrifugation Methods 0.000 description 5
- 108010048367 enhanced green fluorescent protein Proteins 0.000 description 5
- 108010000421 fibronectin attachment peptide Proteins 0.000 description 5
- 239000012634 fragment Substances 0.000 description 5
- 230000005847 immunogenicity Effects 0.000 description 5
- 230000002458 infectious effect Effects 0.000 description 5
- 230000003993 interaction Effects 0.000 description 5
- 239000006166 lysate Substances 0.000 description 5
- 230000001404 mediated effect Effects 0.000 description 5
- 238000012216 screening Methods 0.000 description 5
- 239000000126 substance Substances 0.000 description 5
- 230000009466 transformation Effects 0.000 description 5
- HEDRZPFGACZZDS-UHFFFAOYSA-N Chloroform Chemical compound ClC(Cl)Cl HEDRZPFGACZZDS-UHFFFAOYSA-N 0.000 description 4
- 238000012413 Fluorescence activated cell sorting analysis Methods 0.000 description 4
- 208000031422 Lymphocytic Chronic B-Cell Leukemia Diseases 0.000 description 4
- 206010028980 Neoplasm Diseases 0.000 description 4
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 4
- 238000001042 affinity chromatography Methods 0.000 description 4
- 238000012512 characterization method Methods 0.000 description 4
- 239000003814 drug Substances 0.000 description 4
- 230000004927 fusion Effects 0.000 description 4
- 108010013726 glycyl-arginyl-glycyl-glutamyl-serine Proteins 0.000 description 4
- 239000002609 medium Substances 0.000 description 4
- 230000008569 process Effects 0.000 description 4
- 238000013207 serial dilution Methods 0.000 description 4
- 102100031780 Endonuclease Human genes 0.000 description 3
- 108010042407 Endonucleases Proteins 0.000 description 3
- 108091034117 Oligonucleotide Proteins 0.000 description 3
- 108020005202 Viral DNA Proteins 0.000 description 3
- 108010089975 arginyl-glycyl-aspartyl-serine Proteins 0.000 description 3
- 210000003719 b-lymphocyte Anatomy 0.000 description 3
- 239000011324 bead Substances 0.000 description 3
- 210000004369 blood Anatomy 0.000 description 3
- 239000008280 blood Substances 0.000 description 3
- 201000011510 cancer Diseases 0.000 description 3
- 229940079593 drug Drugs 0.000 description 3
- 230000002068 genetic effect Effects 0.000 description 3
- 239000001963 growth medium Substances 0.000 description 3
- 210000003958 hematopoietic stem cell Anatomy 0.000 description 3
- 230000005764 inhibitory process Effects 0.000 description 3
- NBQNWMBBSKPBAY-UHFFFAOYSA-N iodixanol Chemical compound IC=1C(C(=O)NCC(O)CO)=C(I)C(C(=O)NCC(O)CO)=C(I)C=1N(C(=O)C)CC(O)CN(C(C)=O)C1=C(I)C(C(=O)NCC(O)CO)=C(I)C(C(=O)NCC(O)CO)=C1I NBQNWMBBSKPBAY-UHFFFAOYSA-N 0.000 description 3
- 229960004359 iodixanol Drugs 0.000 description 3
- 239000013641 positive control Substances 0.000 description 3
- 238000000746 purification Methods 0.000 description 3
- 230000009467 reduction Effects 0.000 description 3
- 239000000523 sample Substances 0.000 description 3
- 238000010187 selection method Methods 0.000 description 3
- 239000006228 supernatant Substances 0.000 description 3
- 230000001225 therapeutic effect Effects 0.000 description 3
- 230000002463 transducing effect Effects 0.000 description 3
- NNRFRJQMBSBXGO-CIUDSAMLSA-N (3s)-3-[[2-[[(2s)-2-amino-5-(diaminomethylideneamino)pentanoyl]amino]acetyl]amino]-4-[[(1s)-1-carboxy-2-hydroxyethyl]amino]-4-oxobutanoic acid Chemical compound NC(N)=NCCC[C@H](N)C(=O)NCC(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](CO)C(O)=O NNRFRJQMBSBXGO-CIUDSAMLSA-N 0.000 description 2
- DJXDNYKQOZYOFK-GUBZILKMSA-N (4s)-4-[[2-[[(2s)-2-amino-5-(diaminomethylideneamino)pentanoyl]amino]acetyl]amino]-5-[[(1s)-1-carboxy-2-hydroxyethyl]amino]-5-oxopentanoic acid Chemical compound NC(N)=NCCC[C@H](N)C(=O)NCC(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CO)C(O)=O DJXDNYKQOZYOFK-GUBZILKMSA-N 0.000 description 2
- UMZVBZDHGKJFGQ-UHFFFAOYSA-N 1-[2-[[2-[[2-[[2-[(2-aminoacetyl)amino]-5-(diaminomethylideneamino)pentanoyl]amino]acetyl]amino]-3-carboxypropanoyl]amino]-3-hydroxybutanoyl]pyrrolidine-2-carboxylic acid Chemical compound NC(N)=NCCCC(NC(=O)CN)C(=O)NCC(=O)NC(CC(O)=O)C(=O)NC(C(O)C)C(=O)N1CCCC1C(O)=O UMZVBZDHGKJFGQ-UHFFFAOYSA-N 0.000 description 2
- 208000023275 Autoimmune disease Diseases 0.000 description 2
- 108091003079 Bovine Serum Albumin Proteins 0.000 description 2
- 102100024495 Cdc42 effector protein 4 Human genes 0.000 description 2
- 206010009944 Colon cancer Diseases 0.000 description 2
- 208000035473 Communicable disease Diseases 0.000 description 2
- 102000053602 DNA Human genes 0.000 description 2
- 239000006144 Dulbecco’s modified Eagle's medium Substances 0.000 description 2
- 101000762421 Homo sapiens Cdc42 effector protein 4 Proteins 0.000 description 2
- 101000957426 Homo sapiens Centrosomal protein of 135 kDa Proteins 0.000 description 2
- 102000000646 Interleukin-3 Human genes 0.000 description 2
- 108010002386 Interleukin-3 Proteins 0.000 description 2
- 108700026244 Open Reading Frames Proteins 0.000 description 2
- ISWSIDIOOBJBQZ-UHFFFAOYSA-N Phenol Chemical compound OC1=CC=CC=C1 ISWSIDIOOBJBQZ-UHFFFAOYSA-N 0.000 description 2
- 108010052388 RGES peptide Proteins 0.000 description 2
- 108700005077 Viral Genes Proteins 0.000 description 2
- 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 2
- 230000003213 activating effect Effects 0.000 description 2
- 238000010276 construction Methods 0.000 description 2
- 230000008878 coupling Effects 0.000 description 2
- 238000010168 coupling process Methods 0.000 description 2
- 238000005859 coupling reaction Methods 0.000 description 2
- 238000000605 extraction Methods 0.000 description 2
- 239000012894 fetal calf serum Substances 0.000 description 2
- 238000000684 flow cytometry Methods 0.000 description 2
- 238000000799 fluorescence microscopy Methods 0.000 description 2
- 230000009395 genetic defect Effects 0.000 description 2
- 230000028993 immune response Effects 0.000 description 2
- 230000006872 improvement Effects 0.000 description 2
- 229940076264 interleukin-3 Drugs 0.000 description 2
- 238000011835 investigation Methods 0.000 description 2
- 239000012139 lysis buffer Substances 0.000 description 2
- 239000013642 negative control Substances 0.000 description 2
- 230000002265 prevention Effects 0.000 description 2
- 238000012545 processing Methods 0.000 description 2
- 230000012846 protein folding Effects 0.000 description 2
- 101150066583 rep gene Proteins 0.000 description 2
- 230000000717 retained effect Effects 0.000 description 2
- 238000002864 sequence alignment Methods 0.000 description 2
- 239000011780 sodium chloride Substances 0.000 description 2
- 239000000243 solution Substances 0.000 description 2
- UCSJYZPVAKXKNQ-HZYVHMACSA-N streptomycin Chemical compound CN[C@H]1[C@H](O)[C@@H](O)[C@H](CO)O[C@H]1O[C@@H]1[C@](C=O)(O)[C@H](C)O[C@H]1O[C@@H]1[C@@H](NC(N)=N)[C@H](O)[C@@H](NC(N)=N)[C@H](O)[C@H]1O UCSJYZPVAKXKNQ-HZYVHMACSA-N 0.000 description 2
- 230000008685 targeting Effects 0.000 description 2
- 238000012360 testing method Methods 0.000 description 2
- 230000029812 viral genome replication Effects 0.000 description 2
- QKNYBSVHEMOAJP-UHFFFAOYSA-N 2-amino-2-(hydroxymethyl)propane-1,3-diol;hydron;chloride Chemical compound Cl.OCC(N)(CO)CO QKNYBSVHEMOAJP-UHFFFAOYSA-N 0.000 description 1
- 108700026758 Adenovirus hexon capsid Proteins 0.000 description 1
- 102100024222 B-lymphocyte antigen CD19 Human genes 0.000 description 1
- 241000894006 Bacteria Species 0.000 description 1
- 201000009030 Carcinoma Diseases 0.000 description 1
- 102100025907 Dyslexia-associated protein KIAA0319-like protein Human genes 0.000 description 1
- 238000002965 ELISA Methods 0.000 description 1
- 108010067770 Endopeptidase K Proteins 0.000 description 1
- 101710091045 Envelope protein Proteins 0.000 description 1
- 241000588724 Escherichia coli Species 0.000 description 1
- 229920001917 Ficoll Polymers 0.000 description 1
- 241000282412 Homo Species 0.000 description 1
- 101000980825 Homo sapiens B-lymphocyte antigen CD19 Proteins 0.000 description 1
- 101001076904 Homo sapiens Dyslexia-associated protein KIAA0319-like protein Proteins 0.000 description 1
- 108090000144 Human Proteins Proteins 0.000 description 1
- 102000003839 Human Proteins Human genes 0.000 description 1
- 241000701024 Human betaherpesvirus 5 Species 0.000 description 1
- 102000001706 Immunoglobulin Fab Fragments Human genes 0.000 description 1
- 108010054477 Immunoglobulin Fab Fragments Proteins 0.000 description 1
- 102100034349 Integrase Human genes 0.000 description 1
- ZDXPYRJPNDTMRX-VKHMYHEASA-N L-glutamine Chemical compound OC(=O)[C@@H](N)CCC(N)=O ZDXPYRJPNDTMRX-VKHMYHEASA-N 0.000 description 1
- 229930182816 L-glutamine Natural products 0.000 description 1
- 241000124008 Mammalia Species 0.000 description 1
- 208000035490 Megakaryoblastic Acute Leukemia Diseases 0.000 description 1
- 241001465754 Metazoa Species 0.000 description 1
- 241000283973 Oryctolagus cuniculus Species 0.000 description 1
- 208000008071 Parvoviridae Infections Diseases 0.000 description 1
- 206010057343 Parvovirus infection Diseases 0.000 description 1
- 229930182555 Penicillin Natural products 0.000 description 1
- JGSARLDLIJGVTE-MBNYWOFBSA-N Penicillin G Chemical compound N([C@H]1[C@H]2SC([C@@H](N2C1=O)C(O)=O)(C)C)C(=O)CC1=CC=CC=C1 JGSARLDLIJGVTE-MBNYWOFBSA-N 0.000 description 1
- 101710188315 Protein X Proteins 0.000 description 1
- 239000012979 RPMI medium Substances 0.000 description 1
- 229920002684 Sepharose Polymers 0.000 description 1
- 238000012300 Sequence Analysis Methods 0.000 description 1
- 108020004682 Single-Stranded DNA Proteins 0.000 description 1
- 108020005038 Terminator Codon Proteins 0.000 description 1
- 239000007983 Tris buffer Substances 0.000 description 1
- 108010003533 Viral Envelope Proteins Proteins 0.000 description 1
- 108010067390 Viral Proteins Proteins 0.000 description 1
- MKUXAQIIEYXACX-UHFFFAOYSA-N aciclovir Chemical compound N1C(N)=NC(=O)C2=C1N(COCCO)C=N2 MKUXAQIIEYXACX-UHFFFAOYSA-N 0.000 description 1
- 229960004150 aciclovir Drugs 0.000 description 1
- 239000002253 acid Substances 0.000 description 1
- 208000020700 acute megakaryocytic leukemia Diseases 0.000 description 1
- 230000003321 amplification Effects 0.000 description 1
- 210000004102 animal cell Anatomy 0.000 description 1
- 108010072041 arginyl-glycyl-aspartic acid Proteins 0.000 description 1
- 230000004071 biological effect Effects 0.000 description 1
- 230000000903 blocking effect Effects 0.000 description 1
- 208000035269 cancer or benign tumor Diseases 0.000 description 1
- 238000004113 cell culture Methods 0.000 description 1
- 230000003915 cell function Effects 0.000 description 1
- 210000000170 cell membrane Anatomy 0.000 description 1
- 208000032852 chronic lymphocytic leukemia Diseases 0.000 description 1
- 230000002860 competitive effect Effects 0.000 description 1
- ATDGTVJJHBUTRL-UHFFFAOYSA-N cyanogen bromide Chemical compound BrC#N ATDGTVJJHBUTRL-UHFFFAOYSA-N 0.000 description 1
- 230000002435 cytoreductive effect Effects 0.000 description 1
- 230000002950 deficient Effects 0.000 description 1
- 230000001419 dependent effect Effects 0.000 description 1
- 238000011161 development Methods 0.000 description 1
- 238000003745 diagnosis Methods 0.000 description 1
- 238000009792 diffusion process Methods 0.000 description 1
- 230000029087 digestion Effects 0.000 description 1
- 238000004520 electroporation Methods 0.000 description 1
- 239000003623 enhancer Substances 0.000 description 1
- 210000003527 eukaryotic cell Anatomy 0.000 description 1
- 238000011156 evaluation Methods 0.000 description 1
- 238000001476 gene delivery Methods 0.000 description 1
- 238000012239 gene modification Methods 0.000 description 1
- 238000010353 genetic engineering Methods 0.000 description 1
- 230000005017 genetic modification Effects 0.000 description 1
- 235000013617 genetically modified food Nutrition 0.000 description 1
- 238000004128 high performance liquid chromatography Methods 0.000 description 1
- 238000009396 hybridization Methods 0.000 description 1
- 239000012642 immune effector Substances 0.000 description 1
- 230000008105 immune reaction Effects 0.000 description 1
- 210000000987 immune system Anatomy 0.000 description 1
- 238000010185 immunofluorescence analysis Methods 0.000 description 1
- 230000002163 immunogen Effects 0.000 description 1
- 229940121354 immunomodulator Drugs 0.000 description 1
- 230000001771 impaired effect Effects 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 230000002401 inhibitory effect Effects 0.000 description 1
- 238000002743 insertional mutagenesis Methods 0.000 description 1
- 230000003834 intracellular effect Effects 0.000 description 1
- 238000004255 ion exchange chromatography Methods 0.000 description 1
- 210000003734 kidney Anatomy 0.000 description 1
- 208000032839 leukemia Diseases 0.000 description 1
- 230000000670 limiting effect Effects 0.000 description 1
- 230000007774 longterm Effects 0.000 description 1
- 230000003211 malignant effect Effects 0.000 description 1
- 239000000463 material Substances 0.000 description 1
- 230000007246 mechanism Effects 0.000 description 1
- 210000003593 megakaryocyte Anatomy 0.000 description 1
- MIKKOBKEXMRYFQ-WZTVWXICSA-N meglumine amidotrizoate Chemical compound C[NH2+]C[C@H](O)[C@@H](O)[C@H](O)[C@H](O)CO.CC(=O)NC1=C(I)C(NC(C)=O)=C(I)C(C([O-])=O)=C1I MIKKOBKEXMRYFQ-WZTVWXICSA-N 0.000 description 1
- 210000004779 membrane envelope Anatomy 0.000 description 1
- 239000000203 mixture Substances 0.000 description 1
- 210000001616 monocyte Anatomy 0.000 description 1
- 210000005087 mononuclear cell Anatomy 0.000 description 1
- 238000002703 mutagenesis Methods 0.000 description 1
- 231100000350 mutagenesis Toxicity 0.000 description 1
- 244000309711 non-enveloped viruses Species 0.000 description 1
- 230000005937 nuclear translocation Effects 0.000 description 1
- 238000003199 nucleic acid amplification method Methods 0.000 description 1
- 238000005457 optimization Methods 0.000 description 1
- 230000007918 pathogenicity Effects 0.000 description 1
- 230000037361 pathway Effects 0.000 description 1
- 229940049954 penicillin Drugs 0.000 description 1
- 210000005259 peripheral blood Anatomy 0.000 description 1
- 239000011886 peripheral blood Substances 0.000 description 1
- 238000002823 phage display Methods 0.000 description 1
- 239000013600 plasmid vector Substances 0.000 description 1
- 238000003752 polymerase chain reaction Methods 0.000 description 1
- 230000000750 progressive effect Effects 0.000 description 1
- 238000011002 quantification Methods 0.000 description 1
- 238000011084 recovery Methods 0.000 description 1
- 239000011347 resin Substances 0.000 description 1
- 229920005989 resin Polymers 0.000 description 1
- 238000012552 review Methods 0.000 description 1
- 238000002741 site-directed mutagenesis Methods 0.000 description 1
- 230000000392 somatic effect Effects 0.000 description 1
- 238000009331 sowing Methods 0.000 description 1
- 239000007858 starting material Substances 0.000 description 1
- 229960005322 streptomycin Drugs 0.000 description 1
- 238000012916 structural analysis Methods 0.000 description 1
- 238000003786 synthesis reaction Methods 0.000 description 1
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 description 1
- 238000012800 visualization Methods 0.000 description 1
- 238000005406 washing Methods 0.000 description 1
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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
-
- 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
- A61P31/12—Antivirals
- A61P31/20—Antivirals for DNA viruses
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—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
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Biomedical Technology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Oncology (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Communicable Diseases (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Immunology (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US34413101P | 2001-12-21 | 2001-12-21 | |
| US344131P | 2001-12-21 | ||
| US36234902P | 2002-03-07 | 2002-03-07 | |
| US362349P | 2002-03-07 | ||
| US40711602P | 2002-08-30 | 2002-08-30 | |
| US407116P | 2002-08-30 | ||
| PCT/EP2002/014750 WO2003054197A2 (en) | 2001-12-21 | 2002-12-23 | A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES2467156T3 true ES2467156T3 (es) | 2014-06-12 |
Family
ID=27407597
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES02787962.6T Expired - Lifetime ES2467156T3 (es) | 2001-12-21 | 2002-12-23 | Una biblioteca de genes estructurales modificados o de partículas modificadas de cápside útiles para la identificación de clones virales con tropismo celular deseado |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20050106558A1 (enExample) |
| EP (2) | EP1456383B1 (enExample) |
| JP (1) | JP2005512569A (enExample) |
| AU (2) | AU2002352261B2 (enExample) |
| CA (1) | CA2468882C (enExample) |
| ES (1) | ES2467156T3 (enExample) |
| WO (1) | WO2003054197A2 (enExample) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE19827457C1 (de) * | 1998-06-19 | 2000-03-02 | Medigene Ag | Strukturprotein von AAV, seine Herstellung und Verwendung |
| US20070172460A1 (en) * | 2003-03-19 | 2007-07-26 | Jurgen Kleinschmidt | Random peptide library displayed on aav vectors |
| EP1486567A1 (en) * | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| WO2007084773A2 (en) * | 2006-01-20 | 2007-07-26 | University Of North Carolina At Chapel Hill | Enhanced production of infectious parvovirus vectors in insect cells |
| CN101511373B (zh) * | 2006-06-19 | 2015-12-09 | 阿斯克肋匹奥生物制药公司 | 用于基因治疗的修饰的因子ⅷ和因子ⅸ基因和载体 |
| EP2012122A1 (en) | 2007-07-06 | 2009-01-07 | Medigene AG | Mutated parvovirus structural proteins as vaccines |
| EP2158211B1 (en) * | 2007-05-31 | 2016-08-10 | Medigene AG | Mutated structural protein of a parvovirus |
| EP2098536A1 (en) * | 2008-03-05 | 2009-09-09 | 4-Antibody AG | Isolation and identification of antigen- or ligand-specific binding proteins |
| EP2396343B1 (en) | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| JP5765790B2 (ja) * | 2010-10-21 | 2015-08-19 | ドイッチェス・クレープスフォルシュングスツェントルムＤｅｕｔｓｃｈｅｓ Ｋｒｅｂｓｆｏｒｓｃｈｕｎｇｓｚｅｎｔｒｕｍ | ラットパルボウイルスｈ−１ｐｖのそのカプシドの遺伝子操作による癌細胞への再標的化 |
| DK3693025T3 (da) | 2011-04-22 | 2022-01-10 | Univ California | Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf |
| US9821043B2 (en) | 2011-09-15 | 2017-11-21 | Medigene Ag | Anti-HER2 vaccine based upon AAV derived multimeric structures |
| EP2692731A1 (en) * | 2012-07-31 | 2014-02-05 | Paul-Ehrlich-Institut Bundesamt für Sera und Impfstoffe | Recombinant Adeno-Associated virus (AAV) vector particles displaying high-affinity ligands for cell-type specific gene delivery |
| EP2940131B1 (en) * | 2012-12-25 | 2019-02-20 | Takara Bio Inc. | Aav variant |
| US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| ES2822551T3 (es) | 2014-03-17 | 2021-05-04 | Adverum Biotechnologies Inc | Composiciones y métodos para la expresión génica mejorada en células de los conos |
| EP3221456B1 (en) * | 2014-11-21 | 2021-09-22 | University of Florida Research Foundation, Inc. | Genome-modified recombinant adeno-associated virus vectors |
| FI3265178T3 (fi) | 2015-03-02 | 2026-08-03 | Adverum Biotechnologies Inc | Koostumuksia ja menetelmiä polynukleotidien viemiseksi intravitreaalisesti retinaalisiin tappisoluihin |
| JP6836999B2 (ja) | 2015-03-24 | 2021-03-03 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ | アデノ随伴ウイルス変異体及びその使用方法 |
| WO2017197355A2 (en) | 2016-05-13 | 2017-11-16 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and methods of use thereof |
| CN109640949B (zh) | 2016-07-29 | 2022-11-25 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体和其使用方法 |
| CA3034089A1 (en) * | 2016-08-18 | 2018-02-22 | The Regents Of The University Of California | Crispr-cas genome engineering via a modular aav delivery system |
| AU2017345470B2 (en) | 2016-10-19 | 2023-08-03 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
| KR20250023600A (ko) | 2017-06-30 | 2025-02-18 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법 |
| MX2020001997A (es) | 2017-08-28 | 2020-07-20 | Univ California | Variantes de capside de virus adenoasociado y metodos de uso de estas. |
| DK3684423T5 (da) | 2017-09-20 | 2024-08-26 | 4D Molecular Therapeutics Inc | Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf |
| WO2019084015A1 (en) * | 2017-10-23 | 2019-05-02 | Daniel Schmidt | PROGRAMMABLE SET OF VIRUS COMPOSITES FOR ADMINISTRATION OF GENES TARGETING A RECEPTOR |
| US11766489B2 (en) | 2017-11-27 | 2023-09-26 | 4D Molecular Therapeutics, Inc. | Adeno-associated virus variant capsids and use for inhibiting angiogenesis |
| CA3097375A1 (en) * | 2018-04-27 | 2019-10-31 | Universitat Heidelberg | Modified aav capsid polypeptides for treatment of muscular diseases |
| EP3931335A2 (en) | 2019-02-25 | 2022-01-05 | Novartis AG | Compositions and methods to treat bietti crystalline dystrophy |
| US20220154210A1 (en) | 2019-02-25 | 2022-05-19 | Novartis Ag | Compositions and methods to treat bietti crystalline dystrophy |
| EP3715358A1 (en) | 2019-03-28 | 2020-09-30 | Universität zu Köln | Mutated adeno-associated virus capsid proteins, aav particle comprising the same and liver directed aav vector gene therapy |
| CN114269926A (zh) | 2019-04-26 | 2022-04-01 | 美商圣加莫治疗股份有限公司 | Aav工程化 |
| JP2022551986A (ja) * | 2019-10-16 | 2022-12-14 | ザ・ブロード・インスティテュート・インコーポレイテッド | 修飾筋肉標的化組成物 |
| CN119317718A (zh) | 2022-01-25 | 2025-01-14 | 宾夕法尼亚州大学信托人 | 用于改善心脏转导和肝脏去靶向的aav衣壳 |
| WO2025102034A1 (en) | 2023-11-10 | 2025-05-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for barth syndrome |
| WO2026122921A1 (en) | 2024-12-05 | 2026-06-11 | The Trustees Of The University Of Pennsylvania | Gene therapy for barth syndrome |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6031071A (en) * | 1996-01-24 | 2000-02-29 | Biophage, Inc. | Methods of generating novel peptides |
| WO1997038723A1 (en) * | 1996-04-16 | 1997-10-23 | Immusol Incorporated | Targeted viral vectors |
| ATE291633T1 (de) * | 1999-06-01 | 2005-04-15 | Univ Washington | Rekombinante adenovirale vektoren, die chimäre fiberproteine exprimieren, für die zellspezifische infektion und genomische integration |
| WO2002053703A2 (en) * | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
-
2002
- 2002-12-23 US US10/498,163 patent/US20050106558A1/en not_active Abandoned
- 2002-12-23 AU AU2002352261A patent/AU2002352261B2/en not_active Expired
- 2002-12-23 JP JP2003554898A patent/JP2005512569A/ja active Pending
- 2002-12-23 WO PCT/EP2002/014750 patent/WO2003054197A2/en not_active Ceased
- 2002-12-23 ES ES02787962.6T patent/ES2467156T3/es not_active Expired - Lifetime
- 2002-12-23 EP EP02787962.6A patent/EP1456383B1/en not_active Expired - Lifetime
- 2002-12-23 EP EP10011286A patent/EP2363487A3/en not_active Withdrawn
- 2002-12-23 CA CA2468882A patent/CA2468882C/en not_active Expired - Lifetime
-
2008
- 2008-05-07 AU AU2008202032A patent/AU2008202032B2/en not_active Expired
Also Published As
| Publication number | Publication date |
|---|---|
| WO2003054197A3 (en) | 2004-03-18 |
| CA2468882A1 (en) | 2003-07-03 |
| AU2008202032A1 (en) | 2009-11-26 |
| CA2468882C (en) | 2015-09-29 |
| AU2002352261A1 (en) | 2003-07-09 |
| WO2003054197A2 (en) | 2003-07-03 |
| EP1456383A2 (en) | 2004-09-15 |
| EP1456383B1 (en) | 2014-03-12 |
| EP2363487A3 (en) | 2012-03-21 |
| US20050106558A1 (en) | 2005-05-19 |
| EP2363487A2 (en) | 2011-09-07 |
| AU2008202032B2 (en) | 2012-09-20 |
| JP2005512569A (ja) | 2005-05-12 |
| AU2002352261B2 (en) | 2008-02-07 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| AU2002352261B2 (en) | A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism | |
| ES2273498T3 (es) | Proteina estructural de vaa, produccion y uso de la misma. | |
| US7220577B2 (en) | Modified AAV | |
| ES2288037T3 (es) | Secuencia de acido nucleico del serotipo 1 de adenovirus asociado, vectores y celulas huesped que las contienen. | |
| US7314912B1 (en) | AAv scleroprotein, production and use thereof | |
| ES2711349T3 (es) | Cápside | |
| US7749492B2 (en) | AAV vectors and methods | |
| US7252997B1 (en) | Structural protein of adeno-associated virus with modified antigenicity, its production and its use | |
| US6962815B2 (en) | AAV2 vectors and methods | |
| ES2230569T3 (es) | Funciones accesorias para uso en la produccion de viriones aav recombinantes. | |
| US20070172460A1 (en) | Random peptide library displayed on aav vectors | |
| Bertran et al. | Recombinant adeno-associated virus-mediated high-efficiency, transient expression of the murine cationic amino acid transporter (ecotropic retroviral receptor) permits stable transduction of human HeLa cells by ecotropic retroviral vectors | |
| Xu et al. | Capsid modification of adeno-associated virus and tumor targeting gene therapy | |
| AU2013254897A1 (en) | Vectors with modified initiation codon for the translation of AAV-Rep78 useful for production of AAV in insect cells | |
| HK40020047A (en) | Capsid |