CA2379564C - Structural protein of adeno-associated virus with modified chromatographic properties, its production and use - Google Patents
Structural protein of adeno-associated virus with modified chromatographic properties, its production and use Download PDFInfo
- Publication number
- CA2379564C CA2379564C CA2379564A CA2379564A CA2379564C CA 2379564 C CA2379564 C CA 2379564C CA 2379564 A CA2379564 A CA 2379564A CA 2379564 A CA2379564 A CA 2379564A CA 2379564 C CA2379564 C CA 2379564C
- Authority
- CA
- Canada
- Prior art keywords
- structural protein
- aav
- virus
- mutation
- protein
- 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 - Fee Related
Links
- 241000702421 Dependoparvovirus Species 0.000 title claims abstract description 24
- 101710172711 Structural protein Proteins 0.000 title claims description 76
- 238000004519 manufacturing process Methods 0.000 title abstract description 7
- 230000035772 mutation Effects 0.000 claims abstract description 57
- 210000004027 cell Anatomy 0.000 claims description 59
- 241000700605 Viruses Species 0.000 claims description 58
- 239000002245 particle Substances 0.000 claims description 58
- 150000001413 amino acids Chemical class 0.000 claims description 50
- 210000000234 capsid Anatomy 0.000 claims description 44
- 238000003780 insertion Methods 0.000 claims description 39
- 230000037431 insertion Effects 0.000 claims description 39
- 108090000623 proteins and genes Proteins 0.000 claims description 37
- 238000000746 purification Methods 0.000 claims description 37
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 29
- 102000004169 proteins and genes Human genes 0.000 claims description 26
- 238000012217 deletion Methods 0.000 claims description 19
- 230000037430 deletion Effects 0.000 claims description 19
- 150000007523 nucleic acids Chemical class 0.000 claims description 17
- 108020004707 nucleic acids Proteins 0.000 claims description 16
- 102000039446 nucleic acids Human genes 0.000 claims description 16
- 102000005962 receptors Human genes 0.000 claims description 15
- 108020003175 receptors Proteins 0.000 claims description 15
- 230000004075 alteration Effects 0.000 claims description 13
- 102000006495 integrins Human genes 0.000 claims description 11
- 108010044426 integrins Proteins 0.000 claims description 11
- 239000003446 ligand Substances 0.000 claims description 11
- 230000004048 modification Effects 0.000 claims description 11
- 238000012986 modification Methods 0.000 claims description 11
- 230000015572 biosynthetic process Effects 0.000 claims description 10
- 108090000695 Cytokines Proteins 0.000 claims description 8
- 102000004127 Cytokines Human genes 0.000 claims description 8
- 239000000427 antigen Substances 0.000 claims description 6
- 108091007433 antigens Proteins 0.000 claims description 6
- 102000036639 antigens Human genes 0.000 claims description 6
- 230000009467 reduction Effects 0.000 claims description 6
- 102000006240 membrane receptors Human genes 0.000 claims description 5
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 4
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 4
- 108010001857 Cell Surface Receptors Proteins 0.000 claims description 4
- 108090000790 Enzymes Proteins 0.000 claims description 4
- 102000004190 Enzymes Human genes 0.000 claims description 4
- 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 claims description 4
- 101710120037 Toxin CcdB Proteins 0.000 claims description 4
- 239000003102 growth factor Substances 0.000 claims description 4
- 229960002897 heparin Drugs 0.000 claims description 4
- 229920000669 heparin Polymers 0.000 claims description 4
- 230000006872 improvement Effects 0.000 claims description 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 3
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 3
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 3
- 108090001090 Lectins Proteins 0.000 claims description 3
- 102000004856 Lectins Human genes 0.000 claims description 3
- 238000001042 affinity chromatography Methods 0.000 claims description 3
- 239000005556 hormone Substances 0.000 claims description 3
- 229940088597 hormone Drugs 0.000 claims description 3
- 239000002523 lectin Substances 0.000 claims description 3
- 239000000758 substrate Substances 0.000 claims description 3
- 108010070675 Glutathione transferase Proteins 0.000 claims description 2
- 102000005720 Glutathione transferase Human genes 0.000 claims description 2
- 108060003951 Immunoglobulin Proteins 0.000 claims description 2
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Natural products OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 claims description 2
- 102000018358 immunoglobulin Human genes 0.000 claims description 2
- 230000001506 immunosuppresive effect Effects 0.000 claims description 2
- 238000003786 synthesis reaction Methods 0.000 claims description 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 3
- 102000034240 fibrous proteins Human genes 0.000 abstract 2
- 108091005899 fibrous proteins Proteins 0.000 abstract 2
- 101000805768 Banna virus (strain Indonesia/JKT-6423/1980) mRNA (guanine-N(7))-methyltransferase Proteins 0.000 description 23
- 101000686790 Chaetoceros protobacilladnavirus 2 Replication-associated protein Proteins 0.000 description 23
- 101000864475 Chlamydia phage 1 Internal scaffolding protein VP3 Proteins 0.000 description 23
- 101000803553 Eumenes pomiformis Venom peptide 3 Proteins 0.000 description 23
- 101000583961 Halorubrum pleomorphic virus 1 Matrix protein Proteins 0.000 description 23
- 208000015181 infectious disease Diseases 0.000 description 14
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 12
- 238000003776 cleavage reaction Methods 0.000 description 12
- 238000004806 packaging method and process Methods 0.000 description 12
- 230000007017 scission Effects 0.000 description 12
- 230000003612 virological effect Effects 0.000 description 12
- 238000010828 elution Methods 0.000 description 11
- 238000000034 method Methods 0.000 description 11
- 239000002609 medium Substances 0.000 description 10
- 150000003839 salts Chemical class 0.000 description 10
- 239000013598 vector Substances 0.000 description 10
- 108090000565 Capsid Proteins Proteins 0.000 description 9
- 102100023321 Ceruloplasmin Human genes 0.000 description 9
- 238000004587 chromatography analysis Methods 0.000 description 9
- 239000013612 plasmid Substances 0.000 description 9
- 108020004414 DNA Proteins 0.000 description 8
- 238000001415 gene therapy Methods 0.000 description 8
- 101710132601 Capsid protein Proteins 0.000 description 7
- 101710197658 Capsid protein VP1 Proteins 0.000 description 7
- 101710118046 RNA-directed RNA polymerase Proteins 0.000 description 7
- 101710108545 Viral protein 1 Proteins 0.000 description 7
- 241000701931 Canine parvovirus Species 0.000 description 6
- 238000002965 ELISA Methods 0.000 description 6
- 239000011780 sodium chloride Substances 0.000 description 6
- 238000012360 testing method Methods 0.000 description 6
- 101710081079 Minor spike protein H Proteins 0.000 description 5
- 230000000694 effects Effects 0.000 description 5
- 238000000926 separation method Methods 0.000 description 5
- 210000002845 virion Anatomy 0.000 description 5
- 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 4
- 239000013607 AAV vector Substances 0.000 description 4
- OSCLNNWLKKIQJM-WDSKDSINSA-N Gln-Ser-Gly Chemical compound [H]N[C@@H](CCC(N)=O)C(=O)N[C@@H](CO)C(=O)NCC(O)=O OSCLNNWLKKIQJM-WDSKDSINSA-N 0.000 description 4
- 238000003556 assay Methods 0.000 description 4
- 230000008901 benefit Effects 0.000 description 4
- 238000001514 detection method Methods 0.000 description 4
- 239000012634 fragment Substances 0.000 description 4
- 230000003993 interaction Effects 0.000 description 4
- 238000002703 mutagenesis Methods 0.000 description 4
- 231100000350 mutagenesis Toxicity 0.000 description 4
- 238000002360 preparation method Methods 0.000 description 4
- 241000701161 unidentified adenovirus Species 0.000 description 4
- JKMHFZQWWAIEOD-UHFFFAOYSA-N 2-[4-(2-hydroxyethyl)piperazin-1-yl]ethanesulfonic acid Chemical compound OCC[NH+]1CCN(CCS([O-])(=O)=O)CC1 JKMHFZQWWAIEOD-UHFFFAOYSA-N 0.000 description 3
- OPIFSICVWOWJMJ-AEOCFKNESA-N 5-bromo-4-chloro-3-indolyl beta-D-galactoside Chemical compound O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)O[C@H]1OC1=CNC2=CC=C(Br)C(Cl)=C12 OPIFSICVWOWJMJ-AEOCFKNESA-N 0.000 description 3
- 101000829980 Homo sapiens Ral guanine nucleotide dissociation stimulator Proteins 0.000 description 3
- OKKJLVBELUTLKV-UHFFFAOYSA-N Methanol Chemical compound OC OKKJLVBELUTLKV-UHFFFAOYSA-N 0.000 description 3
- 102100023320 Ral guanine nucleotide dissociation stimulator Human genes 0.000 description 3
- AIYUHDOJVYHVIT-UHFFFAOYSA-M caesium chloride Chemical compound [Cl-].[Cs+] AIYUHDOJVYHVIT-UHFFFAOYSA-M 0.000 description 3
- 238000010790 dilution Methods 0.000 description 3
- 239000012895 dilution Substances 0.000 description 3
- 238000002474 experimental method Methods 0.000 description 3
- 239000012535 impurity Substances 0.000 description 3
- 238000001727 in vivo Methods 0.000 description 3
- 230000010354 integration Effects 0.000 description 3
- 239000000463 material Substances 0.000 description 3
- 239000013608 rAAV vector Substances 0.000 description 3
- 239000000523 sample Substances 0.000 description 3
- 239000000126 substance Substances 0.000 description 3
- 238000010361 transduction Methods 0.000 description 3
- 230000026683 transduction Effects 0.000 description 3
- 230000010415 tropism Effects 0.000 description 3
- YBJHBAHKTGYVGT-ZKWXMUAHSA-N (+)-Biotin Chemical compound N1C(=O)N[C@@H]2[C@H](CCCCC(=O)O)SC[C@@H]21 YBJHBAHKTGYVGT-ZKWXMUAHSA-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
- CSCPPACGZOOCGX-UHFFFAOYSA-N Acetone Chemical compound CC(C)=O CSCPPACGZOOCGX-UHFFFAOYSA-N 0.000 description 2
- VHQSGALUSWIYOD-QXEWZRGKSA-N Asn-Pro-Val Chemical compound [H]N[C@@H](CC(N)=O)C(=O)N1CCC[C@H]1C(=O)N[C@@H](C(C)C)C(O)=O VHQSGALUSWIYOD-QXEWZRGKSA-N 0.000 description 2
- WUQXMTITJLFXAU-JIOCBJNQSA-N Asn-Thr-Pro Chemical compound C[C@H]([C@@H](C(=O)N1CCC[C@@H]1C(=O)O)NC(=O)[C@H](CC(=O)N)N)O WUQXMTITJLFXAU-JIOCBJNQSA-N 0.000 description 2
- JZLFYAAGGYMRIK-BYULHYEWSA-N Asn-Val-Asp Chemical compound [H]N[C@@H](CC(N)=O)C(=O)N[C@@H](C(C)C)C(=O)N[C@@H](CC(O)=O)C(O)=O JZLFYAAGGYMRIK-BYULHYEWSA-N 0.000 description 2
- UTLCRGFJFSZWAW-OLHMAJIHSA-N Asp-Thr-Asn Chemical compound C[C@H]([C@@H](C(=O)N[C@@H](CC(=O)N)C(=O)O)NC(=O)[C@H](CC(=O)O)N)O UTLCRGFJFSZWAW-OLHMAJIHSA-N 0.000 description 2
- 108010029697 CD40 Ligand Proteins 0.000 description 2
- 101150013553 CD40 gene Proteins 0.000 description 2
- 102100032937 CD40 ligand Human genes 0.000 description 2
- 102000053602 DNA Human genes 0.000 description 2
- YJIUYQKQBBQYHZ-ACZMJKKPSA-N Gln-Ala-Ala Chemical compound [H]N[C@@H](CCC(N)=O)C(=O)N[C@@H](C)C(=O)N[C@@H](C)C(O)=O YJIUYQKQBBQYHZ-ACZMJKKPSA-N 0.000 description 2
- LGYZYFFDELZWRS-DCAQKATOSA-N Glu-Glu-Lys Chemical compound NCCCC[C@@H](C(O)=O)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@@H](N)CCC(O)=O LGYZYFFDELZWRS-DCAQKATOSA-N 0.000 description 2
- DWUKOTKSTDWGAE-BQBZGAKWSA-N Gly-Asn-Arg Chemical compound NCC(=O)N[C@@H](CC(N)=O)C(=O)N[C@H](C(O)=O)CCCN=C(N)N DWUKOTKSTDWGAE-BQBZGAKWSA-N 0.000 description 2
- CSMYMGFCEJWALV-WDSKDSINSA-N Gly-Ser-Gln Chemical compound NCC(=O)N[C@@H](CO)C(=O)N[C@H](C(O)=O)CCC(N)=O CSMYMGFCEJWALV-WDSKDSINSA-N 0.000 description 2
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 description 2
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 description 2
- 241000702617 Human parvovirus B19 Species 0.000 description 2
- PXKACEXYLPBMAD-JBDRJPRFSA-N Ile-Ser-Ser Chemical compound CC[C@H](C)[C@@H](C(=O)N[C@@H](CO)C(=O)N[C@@H](CO)C(=O)O)N PXKACEXYLPBMAD-JBDRJPRFSA-N 0.000 description 2
- PMGDADKJMCOXHX-UHFFFAOYSA-N L-Arginyl-L-glutamin-acetat Natural products NC(=N)NCCCC(N)C(=O)NC(CCC(N)=O)C(O)=O PMGDADKJMCOXHX-UHFFFAOYSA-N 0.000 description 2
- 108010085895 Laminin Proteins 0.000 description 2
- 102000007547 Laminin Human genes 0.000 description 2
- DBVWMYGBVFCRBE-CIUDSAMLSA-N Leu-Asn-Asn Chemical compound [H]N[C@@H](CC(C)C)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](CC(N)=O)C(O)=O DBVWMYGBVFCRBE-CIUDSAMLSA-N 0.000 description 2
- VPKIQULSKFVCSM-SRVKXCTJSA-N Leu-Gln-Arg Chemical compound [H]N[C@@H](CC(C)C)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@@H](CCCNC(N)=N)C(O)=O VPKIQULSKFVCSM-SRVKXCTJSA-N 0.000 description 2
- 241000699660 Mus musculus Species 0.000 description 2
- 108010079364 N-glycylalanine Proteins 0.000 description 2
- 206010028980 Neoplasm Diseases 0.000 description 2
- 108700026244 Open Reading Frames Proteins 0.000 description 2
- WKLMCMXFMQEKCX-SLFFLAALSA-N Phe-Phe-Pro Chemical compound C1C[C@@H](N(C1)C(=O)[C@H](CC2=CC=CC=C2)NC(=O)[C@H](CC3=CC=CC=C3)N)C(=O)O WKLMCMXFMQEKCX-SLFFLAALSA-N 0.000 description 2
- MSSXKZBDKZAHCX-UNQGMJICSA-N Phe-Thr-Val Chemical compound [H]N[C@@H](CC1=CC=CC=C1)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H](C(C)C)C(O)=O MSSXKZBDKZAHCX-UNQGMJICSA-N 0.000 description 2
- 241000125945 Protoparvovirus Species 0.000 description 2
- 108010052388 RGES peptide Proteins 0.000 description 2
- 108700008625 Reporter Genes Proteins 0.000 description 2
- OYEDZGNMSBZCIM-XGEHTFHBSA-N Ser-Arg-Thr Chemical compound [H]N[C@@H](CO)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H]([C@@H](C)O)C(O)=O OYEDZGNMSBZCIM-XGEHTFHBSA-N 0.000 description 2
- DMWNPLOERDAHSY-MEYUZBJRSA-N Tyr-Leu-Thr Chemical compound [H]N[C@@H](CC1=CC=C(O)C=C1)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H]([C@@H](C)O)C(O)=O DMWNPLOERDAHSY-MEYUZBJRSA-N 0.000 description 2
- BYAKMYBZADCNMN-JYJNAYRXSA-N Tyr-Lys-Gln Chemical compound [H]N[C@@H](CC1=CC=C(O)C=C1)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CCC(N)=O)C(O)=O BYAKMYBZADCNMN-JYJNAYRXSA-N 0.000 description 2
- HZDQUVQEVVYDDA-ACRUOGEOSA-N Tyr-Tyr-Leu Chemical compound C([C@@H](C(=O)N[C@@H](CC(C)C)C(O)=O)NC(=O)[C@@H](N)CC=1C=CC(O)=CC=1)C1=CC=C(O)C=C1 HZDQUVQEVVYDDA-ACRUOGEOSA-N 0.000 description 2
- 108020005202 Viral DNA Proteins 0.000 description 2
- 238000010521 absorption reaction Methods 0.000 description 2
- 238000004458 analytical method Methods 0.000 description 2
- 230000000890 antigenic effect Effects 0.000 description 2
- 108010008355 arginyl-glutamine Proteins 0.000 description 2
- 108010089975 arginyl-glycyl-aspartyl-serine Proteins 0.000 description 2
- 108010069205 aspartyl-phenylalanine Proteins 0.000 description 2
- 239000000969 carrier Substances 0.000 description 2
- 238000005341 cation exchange Methods 0.000 description 2
- 150000001768 cations Chemical class 0.000 description 2
- 230000001413 cellular effect Effects 0.000 description 2
- 238000012512 characterization method Methods 0.000 description 2
- 150000001875 compounds Chemical class 0.000 description 2
- 239000000470 constituent Substances 0.000 description 2
- 239000013078 crystal Substances 0.000 description 2
- 238000005520 cutting process Methods 0.000 description 2
- 230000006870 function Effects 0.000 description 2
- 108020001507 fusion proteins Proteins 0.000 description 2
- 102000037865 fusion proteins Human genes 0.000 description 2
- RWSXRVCMGQZWBV-WDSKDSINSA-N glutathione Chemical compound OC(=O)[C@@H](N)CCC(=O)N[C@@H](CS)C(=O)NCC(O)=O RWSXRVCMGQZWBV-WDSKDSINSA-N 0.000 description 2
- VPZXBVLAVMBEQI-UHFFFAOYSA-N glycyl-DL-alpha-alanine Natural products OC(=O)C(C)NC(=O)CN VPZXBVLAVMBEQI-UHFFFAOYSA-N 0.000 description 2
- 108010037850 glycylvaline Proteins 0.000 description 2
- 230000002209 hydrophobic effect Effects 0.000 description 2
- 230000028993 immune response Effects 0.000 description 2
- 238000010166 immunofluorescence Methods 0.000 description 2
- 230000001771 impaired effect Effects 0.000 description 2
- 238000000338 in vitro Methods 0.000 description 2
- 238000011065 in-situ storage Methods 0.000 description 2
- 230000006698 induction Effects 0.000 description 2
- 108010038320 lysylphenylalanine Proteins 0.000 description 2
- 210000004962 mammalian cell Anatomy 0.000 description 2
- 239000012528 membrane Substances 0.000 description 2
- 108010031719 prolyl-serine Proteins 0.000 description 2
- 230000010076 replication Effects 0.000 description 2
- 108091008146 restriction endonucleases Proteins 0.000 description 2
- 238000010186 staining Methods 0.000 description 2
- 239000011550 stock solution Substances 0.000 description 2
- 238000001890 transfection Methods 0.000 description 2
- 238000001262 western blot Methods 0.000 description 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 1
- YIYCUMYWGOOSNU-FMZZOXHWSA-N 2-[[(2s)-1-[(2s,3s)-2-[[(2s,3r)-2-[[2-[[(2s)-2-amino-4-methylpentanoyl]amino]acetyl]amino]-3-hydroxybutanoyl]amino]-3-methylpentanoyl]pyrrolidine-2-carbonyl]amino]acetic acid Chemical compound CC(C)C[C@H](N)C(=O)NCC(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H]([C@@H](C)CC)C(=O)N1CCC[C@H]1C(=O)NCC(O)=O YIYCUMYWGOOSNU-FMZZOXHWSA-N 0.000 description 1
- LSMDIAAALJJLRO-XQXXSGGOSA-N Ala-Thr-Glu Chemical compound [H]N[C@@H](C)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H](CCC(O)=O)C(O)=O LSMDIAAALJJLRO-XQXXSGGOSA-N 0.000 description 1
- IYMAXBFPHPZYIK-BQBZGAKWSA-N Arg-Gly-Asp Chemical compound 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 1
- 241000894006 Bacteria Species 0.000 description 1
- 108010017384 Blood Proteins Proteins 0.000 description 1
- 102000004506 Blood Proteins Human genes 0.000 description 1
- 101150044789 Cap gene Proteins 0.000 description 1
- 108091026890 Coding region Proteins 0.000 description 1
- 208000003322 Coinfection Diseases 0.000 description 1
- 230000005526 G1 to G0 transition Effects 0.000 description 1
- 108010046649 GDNP peptide Proteins 0.000 description 1
- 102000002464 Galactosidases Human genes 0.000 description 1
- 108010093031 Galactosidases Proteins 0.000 description 1
- HHWQMFIGMMOVFK-WDSKDSINSA-N Gln-Ala-Gly Chemical compound OC(=O)CNC(=O)[C@H](C)NC(=O)[C@@H](N)CCC(N)=O HHWQMFIGMMOVFK-WDSKDSINSA-N 0.000 description 1
- SGVGIVDZLSHSEN-RYUDHWBXSA-N Gln-Tyr-Gly Chemical compound [H]N[C@@H](CCC(N)=O)C(=O)N[C@@H](CC1=CC=C(O)C=C1)C(=O)NCC(O)=O SGVGIVDZLSHSEN-RYUDHWBXSA-N 0.000 description 1
- HUFCEIHAFNVSNR-IHRRRGAJSA-N Glu-Gln-Tyr Chemical compound OC(=O)CC[C@H](N)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@H](C(O)=O)CC1=CC=C(O)C=C1 HUFCEIHAFNVSNR-IHRRRGAJSA-N 0.000 description 1
- SXRSQZLOMIGNAQ-UHFFFAOYSA-N Glutaraldehyde Chemical compound O=CCCCC=O SXRSQZLOMIGNAQ-UHFFFAOYSA-N 0.000 description 1
- 108010024636 Glutathione Proteins 0.000 description 1
- 102000003886 Glycoproteins Human genes 0.000 description 1
- 108090000288 Glycoproteins Proteins 0.000 description 1
- 108010017213 Granulocyte-Macrophage Colony-Stimulating Factor Proteins 0.000 description 1
- 102100039620 Granulocyte-macrophage colony-stimulating factor Human genes 0.000 description 1
- 241000941423 Grom virus Species 0.000 description 1
- 241000282412 Homo Species 0.000 description 1
- 101000914514 Homo sapiens T-cell-specific surface glycoprotein CD28 Proteins 0.000 description 1
- 101000611183 Homo sapiens Tumor necrosis factor Proteins 0.000 description 1
- 241001135569 Human adenovirus 5 Species 0.000 description 1
- 102000013462 Interleukin-12 Human genes 0.000 description 1
- 108010065805 Interleukin-12 Proteins 0.000 description 1
- 108010002350 Interleukin-2 Proteins 0.000 description 1
- 102000000588 Interleukin-2 Human genes 0.000 description 1
- 241001484259 Lacuna Species 0.000 description 1
- KSZCCRIGNVSHFH-UWVGGRQHSA-N Leu-Arg-Gly Chemical compound [H]N[C@@H](CC(C)C)C(=O)N[C@@H](CCCNC(N)=N)C(=O)NCC(O)=O KSZCCRIGNVSHFH-UWVGGRQHSA-N 0.000 description 1
- 125000001429 N-terminal alpha-amino-acid group Chemical group 0.000 description 1
- 102000015336 Nerve Growth Factor Human genes 0.000 description 1
- 108010025020 Nerve Growth Factor Proteins 0.000 description 1
- 108091028043 Nucleic acid sequence Proteins 0.000 description 1
- 108091006629 SLC13A2 Proteins 0.000 description 1
- 108020004682 Single-Stranded DNA Proteins 0.000 description 1
- 108091081024 Start codon Proteins 0.000 description 1
- 108010090804 Streptavidin Proteins 0.000 description 1
- 230000024932 T cell mediated immunity Effects 0.000 description 1
- 102100027213 T-cell-specific surface glycoprotein CD28 Human genes 0.000 description 1
- WVVOFCVMHAXGLE-LFSVMHDDSA-N Thr-Phe-Ala Chemical compound [H]N[C@@H]([C@@H](C)O)C(=O)N[C@@H](CC1=CC=CC=C1)C(=O)N[C@@H](C)C(O)=O WVVOFCVMHAXGLE-LFSVMHDDSA-N 0.000 description 1
- 108700019146 Transgenes Proteins 0.000 description 1
- 102100040247 Tumor necrosis factor Human genes 0.000 description 1
- 102100040245 Tumor necrosis factor receptor superfamily member 5 Human genes 0.000 description 1
- 101150024766 VP1 gene Proteins 0.000 description 1
- 101150093578 VP2 gene Proteins 0.000 description 1
- 101150036700 VP3 gene Proteins 0.000 description 1
- 241000700618 Vaccinia virus Species 0.000 description 1
- 108700005077 Viral Genes Proteins 0.000 description 1
- 108010067390 Viral Proteins Proteins 0.000 description 1
- 101100072652 Xenopus laevis ins-b gene Proteins 0.000 description 1
- 239000000654 additive Substances 0.000 description 1
- 238000005377 adsorption chromatography Methods 0.000 description 1
- 229940019748 antifibrinolytic proteinase inhibitors Drugs 0.000 description 1
- 229960002685 biotin Drugs 0.000 description 1
- 235000020958 biotin Nutrition 0.000 description 1
- 239000011616 biotin Substances 0.000 description 1
- 108091006004 biotinylated proteins Proteins 0.000 description 1
- 210000004899 c-terminal region Anatomy 0.000 description 1
- 150000003943 catecholamines Chemical class 0.000 description 1
- 125000002091 cationic group Chemical group 0.000 description 1
- 239000013592 cell lysate Substances 0.000 description 1
- 210000000170 cell membrane Anatomy 0.000 description 1
- 230000010307 cell transformation Effects 0.000 description 1
- 230000008859 change Effects 0.000 description 1
- 239000013522 chelant Substances 0.000 description 1
- 238000011097 chromatography purification Methods 0.000 description 1
- 238000010367 cloning Methods 0.000 description 1
- 230000000093 cytochemical effect Effects 0.000 description 1
- 238000000432 density-gradient centrifugation Methods 0.000 description 1
- 238000011161 development Methods 0.000 description 1
- 238000003745 diagnosis Methods 0.000 description 1
- 238000009826 distribution Methods 0.000 description 1
- 239000012636 effector Substances 0.000 description 1
- 238000001493 electron microscopy Methods 0.000 description 1
- 230000012202 endocytosis Effects 0.000 description 1
- 210000002919 epithelial cell Anatomy 0.000 description 1
- -1 for example Proteins 0.000 description 1
- 230000002068 genetic effect Effects 0.000 description 1
- 108010078144 glutaminyl-glycine Proteins 0.000 description 1
- 229960003180 glutathione Drugs 0.000 description 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 description 1
- 230000028996 humoral immune response Effects 0.000 description 1
- 230000007062 hydrolysis Effects 0.000 description 1
- 238000006460 hydrolysis reaction Methods 0.000 description 1
- 210000000987 immune system Anatomy 0.000 description 1
- 238000003125 immunofluorescent labeling Methods 0.000 description 1
- 230000005847 immunogenicity Effects 0.000 description 1
- 230000002779 inactivation Effects 0.000 description 1
- 238000010348 incorporation Methods 0.000 description 1
- 238000011534 incubation Methods 0.000 description 1
- 238000009776 industrial production Methods 0.000 description 1
- 230000002458 infectious effect Effects 0.000 description 1
- 239000003112 inhibitor Substances 0.000 description 1
- 238000011835 investigation Methods 0.000 description 1
- 238000004255 ion exchange chromatography Methods 0.000 description 1
- 150000002500 ions Chemical class 0.000 description 1
- 238000007169 ligase reaction Methods 0.000 description 1
- 238000011068 loading method Methods 0.000 description 1
- 230000001926 lymphatic effect Effects 0.000 description 1
- 239000006166 lysate Substances 0.000 description 1
- 238000005259 measurement Methods 0.000 description 1
- 230000007246 mechanism Effects 0.000 description 1
- 230000001404 mediated effect Effects 0.000 description 1
- 108020004084 membrane receptors Proteins 0.000 description 1
- 229910052751 metal Inorganic materials 0.000 description 1
- 239000002184 metal Substances 0.000 description 1
- 244000005700 microbiome Species 0.000 description 1
- 238000007431 microscopic evaluation Methods 0.000 description 1
- 210000004897 n-terminal region Anatomy 0.000 description 1
- 239000002773 nucleotide Substances 0.000 description 1
- 125000003729 nucleotide group Chemical group 0.000 description 1
- 210000000056 organ Anatomy 0.000 description 1
- 238000004810 partition chromatography Methods 0.000 description 1
- 230000007918 pathogenicity Effects 0.000 description 1
- 239000000137 peptide hydrolase inhibitor Substances 0.000 description 1
- 239000000546 pharmaceutical excipient Substances 0.000 description 1
- 239000002504 physiological saline solution Substances 0.000 description 1
- 230000008569 process Effects 0.000 description 1
- 102000004196 processed proteins & peptides Human genes 0.000 description 1
- 108020001580 protein domains Proteins 0.000 description 1
- 108020001775 protein parts Proteins 0.000 description 1
- 238000001243 protein synthesis Methods 0.000 description 1
- 101150066583 rep gene Proteins 0.000 description 1
- 230000000717 retained effect Effects 0.000 description 1
- PYWVYCXTNDRMGF-UHFFFAOYSA-N rhodamine B Chemical compound [Cl-].C=12C=CC(=[N+](CC)CC)C=C2OC2=CC(N(CC)CC)=CC=C2C=1C1=CC=CC=C1C(O)=O PYWVYCXTNDRMGF-UHFFFAOYSA-N 0.000 description 1
- 238000004062 sedimentation Methods 0.000 description 1
- 238000013207 serial dilution Methods 0.000 description 1
- 239000012679 serum free medium Substances 0.000 description 1
- 239000007787 solid Substances 0.000 description 1
- 239000000243 solution Substances 0.000 description 1
- 230000000392 somatic effect Effects 0.000 description 1
- 239000002594 sorbent Substances 0.000 description 1
- 238000001179 sorption measurement Methods 0.000 description 1
- 239000003381 stabilizer Substances 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
- 230000001131 transforming effect Effects 0.000 description 1
- 230000014616 translation Effects 0.000 description 1
- 239000013603 viral vector Substances 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
-
- 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
-
- 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
- C12N2750/14145—Special targeting system for viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicinal Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| DE19933719.5 | 1999-07-19 | ||
| DE19933719A DE19933719A1 (de) | 1999-07-19 | 1999-07-19 | Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung |
| PCT/EP2000/006861 WO2001005991A1 (de) | 1999-07-19 | 2000-07-18 | Strukturprotein von adeno-assoziiertem virus mit veränderten chromatographischen eigenschaften, seine herstellung und verwendung |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CA2379564A1 CA2379564A1 (en) | 2001-01-25 |
| CA2379564C true CA2379564C (en) | 2012-03-20 |
Family
ID=7915238
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2379564A Expired - Fee Related CA2379564C (en) | 1999-07-19 | 2000-07-18 | Structural protein of adeno-associated virus with modified chromatographic properties, its production and use |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US7285381B1 (enExample) |
| EP (1) | EP1198580B1 (enExample) |
| JP (1) | JP4938189B2 (enExample) |
| AT (1) | ATE328101T1 (enExample) |
| AU (1) | AU777885B2 (enExample) |
| CA (1) | CA2379564C (enExample) |
| DE (2) | DE19933719A1 (enExample) |
| ES (1) | ES2263481T3 (enExample) |
| WO (1) | WO2001005991A1 (enExample) |
Families Citing this family (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6759237B1 (en) | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| US6491907B1 (en) | 1998-11-10 | 2002-12-10 | The University Of North Carolina At Chapel Hill | Recombinant parvovirus vectors and method of making |
| US7465566B2 (en) | 2000-02-23 | 2008-12-16 | The Trustees Of The University Of Pennsylvania | Regulation of genes via application of specific and selective electrical and electromagnetic signals |
| US7374916B2 (en) | 2000-02-23 | 2008-05-20 | The Trustees Of The University Of Pennsylvania | Regulation of aggrecan gene expression using specific and selective electrical and electromagnetic signals |
| US7429471B2 (en) | 2000-02-23 | 2008-09-30 | The Trustees Of The University Of Pennsylvania | Regulation of matrix metalloproteinase gene expression using specific and selective electrical and electromagnetic signals |
| US7981611B2 (en) | 2000-02-23 | 2011-07-19 | The Trustees Of The University Of Pennsylvania | Regulation of fibroblastic growth factor-2 (FGF-2) gene expression in living cells with the application of specific and selective electric and electromagnetic fields |
| US7022506B2 (en) | 2000-02-23 | 2006-04-04 | The Trustees Of The University Of Pennsylvania | Method and device for treating osteoarthritis, cartilage disease, defects and injuries in the human knee |
| US7465546B2 (en) | 2000-02-23 | 2008-12-16 | The Trustees Of The University Of Pennsylvania | Regulation of transforming growth factor-beta (TGF-β) gene expression in living cells via the application of specific and selective electric and electromagnetic fields |
| US8313908B2 (en) | 2000-02-23 | 2012-11-20 | The Trustees Of The University Of Pennsylvania | Regulation of stem cell gene production with specific and selective electric and electromagnetic fields |
| WO2005070136A2 (en) | 2004-01-12 | 2005-08-04 | The Trustees Of The University Of Pennsylvania | Up-regulation of bone morphogenetic protein (bmp) gene expression in bone cells by electromagnetic signals |
| 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 |
| 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 |
| US7893985B1 (en) | 2004-03-15 | 2011-02-22 | Grandeye Ltd. | Wide angle electronic camera with improved peripheral vision |
| EP1828390B1 (en) | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| ES2602610T3 (es) | 2007-05-31 | 2017-02-21 | Medigene Ag | Proteína estructural mutada de un parvovirus |
| EP2012122A1 (en) | 2007-07-06 | 2009-01-07 | Medigene AG | Mutated parvovirus structural proteins as vaccines |
| 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 |
| US9464119B2 (en) | 2009-03-04 | 2016-10-11 | Deutsches Krebsforschungszentrum | Assembly activating protein (AAP) and its use for the manufacture of parvovirus particles essentially consisting of VP3 |
| 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 |
| SMT202400520T1 (it) | 2011-04-22 | 2025-01-14 | Univ California | Virioni di virus adeno-associato con capside variante e relativi metodi di utilizzo |
| US9821043B2 (en) | 2011-09-15 | 2017-11-21 | Medigene Ag | Anti-HER2 vaccine based upon AAV derived multimeric structures |
| CN105247044B (zh) | 2013-05-31 | 2021-05-07 | 加利福尼亚大学董事会 | 腺相关病毒变体及其使用方法 |
| EP3561062A1 (en) | 2013-09-13 | 2019-10-30 | California Institute of Technology | Selective recovery |
| CN106414474B (zh) | 2014-03-17 | 2021-01-15 | 阿德夫拉姆生物技术股份有限公司 | 用于视锥细胞中增强的基因表达的组合物和方法 |
| CA2978068C (en) | 2015-03-02 | 2024-05-28 | Adverum Biotechnologies, Inc. | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| 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 |
| ES2869284T3 (es) | 2015-12-11 | 2021-10-25 | California Inst Of Techn | Péptidos de diana para dirigir virus adenoasociados (AAV) |
| KR20230039779A (ko) | 2016-07-29 | 2023-03-21 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법 |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| RS65569B1 (sr) * | 2017-06-27 | 2024-06-28 | Regeneron Pharma | Rekombinantni virusni vektori modifikovani tropizmom i njihove upotrebe za ciljano uvođenje genetičkog materijala u humane ćelije |
| BR112019019015A2 (pt) | 2017-06-30 | 2020-04-14 | Univ California | vírions de vírus adeno-associado com capsídeos variantes e seus métodos de uso |
| KR20200039617A (ko) | 2017-08-28 | 2020-04-16 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 아데노-연관 바이러스 캡시드 변이체 및 이의 사용 방법 |
| CN113966399A (zh) | 2018-09-26 | 2022-01-21 | 加州理工学院 | 用于靶向基因疗法的腺相关病毒组合物 |
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0567578A1 (en) * | 1991-01-25 | 1993-11-03 | The Regents Of The University Of Minnesota | Laminin a chain domain vi polypeptides |
| FR2716893B1 (fr) | 1994-03-03 | 1996-04-12 | Rhone Poulenc Rorer Sa | Virus recombinants, leur préparation et leur utilisation thérapeutique. |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5837520A (en) * | 1995-03-07 | 1998-11-17 | Canji, Inc. | Method of purification of viral vectors |
| WO1997008298A1 (en) * | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
| EP0927044A4 (en) * | 1996-04-16 | 1999-09-08 | Immusol Inc | DEFINED TARGET VIRAL VECTORS |
| US20010031463A1 (en) | 1997-09-02 | 2001-10-18 | Jurgen Kleinschmidt | Method for purifying and concentrating AAV-2 and antigen portions thereof |
| DE19827457C1 (de) | 1998-06-19 | 2000-03-02 | Medigene Ag | Strukturprotein von AAV, seine Herstellung und Verwendung |
| US6491907B1 (en) * | 1998-11-10 | 2002-12-10 | The University Of North Carolina At Chapel Hill | Recombinant parvovirus vectors and method of making |
| US6962815B2 (en) | 2001-01-05 | 2005-11-08 | Children's Hopital Inc. | AAV2 vectors and methods |
-
1999
- 1999-07-19 DE DE19933719A patent/DE19933719A1/de not_active Ceased
-
2000
- 2000-07-18 JP JP2001511201A patent/JP4938189B2/ja not_active Expired - Fee Related
- 2000-07-18 DE DE50012863T patent/DE50012863D1/de not_active Expired - Lifetime
- 2000-07-18 AT AT00951400T patent/ATE328101T1/de not_active IP Right Cessation
- 2000-07-18 CA CA2379564A patent/CA2379564C/en not_active Expired - Fee Related
- 2000-07-18 WO PCT/EP2000/006861 patent/WO2001005991A1/de not_active Ceased
- 2000-07-18 US US10/031,187 patent/US7285381B1/en not_active Expired - Fee Related
- 2000-07-18 EP EP00951400A patent/EP1198580B1/de not_active Expired - Lifetime
- 2000-07-18 ES ES00951400T patent/ES2263481T3/es not_active Expired - Lifetime
- 2000-07-18 AU AU64354/00A patent/AU777885B2/en not_active Ceased
-
2007
- 2007-09-24 US US11/903,662 patent/US20100105123A2/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| EP1198580A1 (de) | 2002-04-24 |
| WO2001005991A1 (de) | 2001-01-25 |
| US7285381B1 (en) | 2007-10-23 |
| US20090098634A2 (en) | 2009-04-16 |
| ATE328101T1 (de) | 2006-06-15 |
| US20090246854A2 (en) | 2009-10-01 |
| US20080241906A1 (en) | 2008-10-02 |
| JP2003505033A (ja) | 2003-02-12 |
| US20100105123A2 (en) | 2010-04-29 |
| DE50012863D1 (de) | 2006-07-06 |
| CA2379564A1 (en) | 2001-01-25 |
| EP1198580B1 (de) | 2006-05-31 |
| ES2263481T3 (es) | 2006-12-16 |
| DE19933719A1 (de) | 2001-01-25 |
| AU6435400A (en) | 2001-02-05 |
| AU777885B2 (en) | 2004-11-04 |
| JP4938189B2 (ja) | 2012-05-23 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CA2379564C (en) | Structural protein of adeno-associated virus with modified chromatographic properties, its production and use | |
| US7314912B1 (en) | AAv scleroprotein, production and use thereof | |
| CA2332115C (en) | Aav structural protein, its preparation and use | |
| US11261463B2 (en) | Methods for producing preparations of recombinant AAV virions substantially free of empty capsids | |
| CA2379220C (en) | Structural protein of adeno-associated virus with modified antigenicity, its production and its use | |
| Lee et al. | Adeno-associated virus (AAV) vectors: rational design strategies for capsid engineering | |
| Rabinowitz et al. | Insertional mutagenesis of AAV2 capsid and the production of recombinant virus | |
| AU780231B2 (en) | Virus vectors and methods of making and administering the same | |
| Büning et al. | Recent developments in adeno‐associated virus vector technology | |
| Wu et al. | Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism | |
| Zhang et al. | Addition of six-His-tagged peptide to the C terminus of adeno-associated virus VP3 does not affect viral tropism or production | |
| CN116249771A (zh) | 改进的腺相关病毒基因治疗载体 | |
| EP3773746A1 (en) | Aav6 variants | |
| Bhrigu | Replication of adeno-associated virus in murine fibroblasts with mouse adenovirus provided helper functions |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request | ||
| MKLA | Lapsed |
Effective date: 20180718 |
|
| MKLA | Lapsed |
Effective date: 20180718 |