JP2003505033A5 - - Google Patents
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- Publication number
- JP2003505033A5 JP2003505033A5 JP2001511201A JP2001511201A JP2003505033A5 JP 2003505033 A5 JP2003505033 A5 JP 2003505033A5 JP 2001511201 A JP2001511201 A JP 2001511201A JP 2001511201 A JP2001511201 A JP 2001511201A JP 2003505033 A5 JP2003505033 A5 JP 2003505033A5
- Authority
- JP
- Japan
- Prior art keywords
- mutation
- capsid
- structural protein
- sequence
- aav
- 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.)
- Granted
Links
- 150000001413 amino acids Chemical class 0.000 description 22
- 101710172711 Structural protein Proteins 0.000 description 21
- 230000035772 mutation Effects 0.000 description 17
- 108090000765 processed proteins & peptides Proteins 0.000 description 14
- 241000702423 Adeno-associated virus - 2 Species 0.000 description 12
- 230000037430 deletion Effects 0.000 description 12
- 238000012217 deletion Methods 0.000 description 12
- 230000037431 insertion Effects 0.000 description 12
- 238000003780 insertion Methods 0.000 description 12
- 210000000234 capsid Anatomy 0.000 description 10
- 238000003776 cleavage reaction Methods 0.000 description 9
- 150000007523 nucleic acids Chemical class 0.000 description 9
- 230000007017 scission Effects 0.000 description 9
- 241000700605 Viruses Species 0.000 description 8
- 108020004707 nucleic acids Proteins 0.000 description 8
- 102000039446 nucleic acids Human genes 0.000 description 8
- 239000002245 particle Substances 0.000 description 7
- 101000805768 Banna virus (strain Indonesia/JKT-6423/1980) mRNA (guanine-N(7))-methyltransferase Proteins 0.000 description 6
- 241000701931 Canine parvovirus Species 0.000 description 6
- 101000686790 Chaetoceros protobacilladnavirus 2 Replication-associated protein Proteins 0.000 description 6
- 101000864475 Chlamydia phage 1 Internal scaffolding protein VP3 Proteins 0.000 description 6
- 101000803553 Eumenes pomiformis Venom peptide 3 Proteins 0.000 description 6
- 101000583961 Halorubrum pleomorphic virus 1 Matrix protein Proteins 0.000 description 6
- 239000003446 ligand Substances 0.000 description 6
- 108090000623 proteins and genes Proteins 0.000 description 5
- 108090000565 Capsid Proteins Proteins 0.000 description 4
- 101710132601 Capsid protein Proteins 0.000 description 4
- 101710197658 Capsid protein VP1 Proteins 0.000 description 4
- 102100023321 Ceruloplasmin Human genes 0.000 description 4
- 101710118046 RNA-directed RNA polymerase Proteins 0.000 description 4
- 101710108545 Viral protein 1 Proteins 0.000 description 4
- 102000006495 integrins Human genes 0.000 description 4
- 108010044426 integrins Proteins 0.000 description 4
- 238000000746 purification Methods 0.000 description 4
- 102000005962 receptors Human genes 0.000 description 4
- 108020003175 receptors Proteins 0.000 description 4
- 101710081079 Minor spike protein H Proteins 0.000 description 3
- 239000000427 antigen Substances 0.000 description 3
- 102000036639 antigens Human genes 0.000 description 3
- 108091007433 antigens Proteins 0.000 description 3
- 238000000034 method Methods 0.000 description 3
- 102000004169 proteins and genes Human genes 0.000 description 3
- 230000003612 virological effect Effects 0.000 description 3
- 241001655883 Adeno-associated virus - 1 Species 0.000 description 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 description 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 description 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 description 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 description 2
- 102000004127 Cytokines Human genes 0.000 description 2
- 108090000695 Cytokines Proteins 0.000 description 2
- 108090000790 Enzymes Proteins 0.000 description 2
- 102000004190 Enzymes Human genes 0.000 description 2
- 241000702617 Human parvovirus B19 Species 0.000 description 2
- 108700026244 Open Reading Frames Proteins 0.000 description 2
- 210000004027 cell Anatomy 0.000 description 2
- 239000013078 crystal Substances 0.000 description 2
- 238000004806 packaging method and process Methods 0.000 description 2
- 239000013612 plasmid Substances 0.000 description 2
- IIZPXYDJLKNOIY-JXPKJXOSSA-N 1-palmitoyl-2-arachidonoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCC\C=C/C\C=C/C\C=C/C\C=C/CCCCC IIZPXYDJLKNOIY-JXPKJXOSSA-N 0.000 description 1
- 102000000844 Cell Surface Receptors Human genes 0.000 description 1
- 108010001857 Cell Surface Receptors Proteins 0.000 description 1
- 108091026890 Coding region Proteins 0.000 description 1
- 241000702421 Dependoparvovirus Species 0.000 description 1
- 108010070675 Glutathione transferase Proteins 0.000 description 1
- 102000005720 Glutathione transferase Human genes 0.000 description 1
- 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 1
- 108060003951 Immunoglobulin Proteins 0.000 description 1
- 102000004856 Lectins Human genes 0.000 description 1
- 108090001090 Lectins Proteins 0.000 description 1
- 108091028043 Nucleic acid sequence Proteins 0.000 description 1
- 241000125945 Protoparvovirus Species 0.000 description 1
- 108091081024 Start codon Proteins 0.000 description 1
- 101710120037 Toxin CcdB Proteins 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
- 239000002253 acid Substances 0.000 description 1
- 150000007513 acids Chemical class 0.000 description 1
- 238000001042 affinity chromatography Methods 0.000 description 1
- 230000008901 benefit Effects 0.000 description 1
- 150000001768 cations Chemical class 0.000 description 1
- 230000008859 change Effects 0.000 description 1
- 125000004122 cyclic group Chemical group 0.000 description 1
- 230000000694 effects Effects 0.000 description 1
- 230000012202 endocytosis Effects 0.000 description 1
- 239000003102 growth factor Substances 0.000 description 1
- 229960002897 heparin Drugs 0.000 description 1
- 229920000669 heparin Polymers 0.000 description 1
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Natural products OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 description 1
- 229940088597 hormone Drugs 0.000 description 1
- 239000005556 hormone Substances 0.000 description 1
- 108091008039 hormone receptors Proteins 0.000 description 1
- 230000028996 humoral immune response Effects 0.000 description 1
- 102000018358 immunoglobulin Human genes 0.000 description 1
- 229940067606 lecithin Drugs 0.000 description 1
- 235000010445 lecithin Nutrition 0.000 description 1
- 239000000787 lecithin Substances 0.000 description 1
- 239000002523 lectin Substances 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 210000004897 n-terminal region Anatomy 0.000 description 1
- 102000004196 processed proteins & peptides Human genes 0.000 description 1
- 108020001580 protein domains Proteins 0.000 description 1
- 230000009467 reduction Effects 0.000 description 1
- 101150066583 rep gene Proteins 0.000 description 1
- 238000000926 separation method Methods 0.000 description 1
- 238000012916 structural analysis Methods 0.000 description 1
- 239000000758 substrate Substances 0.000 description 1
- 241000701161 unidentified adenovirus Species 0.000 description 1
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 (3)
| Publication Number | Publication Date |
|---|---|
| JP2003505033A JP2003505033A (ja) | 2003-02-12 |
| JP2003505033A5 true JP2003505033A5 (enExample) | 2007-09-06 |
| JP4938189B2 JP4938189B2 (ja) | 2012-05-23 |
Family
ID=7915238
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2001511201A Expired - Fee Related JP4938189B2 (ja) | 1999-07-19 | 2000-07-18 | 修飾されたクロマトグラフィー特性を有するアデノ随伴ウイルスの構造タンパク質と、その製造及び使用 |
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
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