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 |
|---|---|---|---|
| DE19933719A DE19933719A1 (de) | 1999-07-19 | 1999-07-19 | Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung |
| DE19933719.5 | 1999-07-19 | ||
| 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 (35)
| 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 |
| EP1135468B1 (en) | 1998-11-10 | 2010-01-06 | University Of North Carolina At Chapel Hill | Virus vectors and methods of making and administering the same |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| CN1918285B (zh) | 2004-01-12 | 2013-09-25 | 宾夕法尼亚大学理事会 | 通过施加由特异性和选择性的电以及电磁信号产生的场上调骨细胞中骨形态发生蛋白(bmp)基因表达的系统以及方法 |
| 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 |
| EP2158211B1 (en) | 2007-05-31 | 2016-08-10 | Medigene AG | Mutated structural protein of a 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 |
| CA2754335C (en) | 2009-03-04 | 2019-12-24 | Medigene Ag | 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 |
| 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 |
| US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| EP3564379A1 (en) | 2013-09-13 | 2019-11-06 | California Institute of Technology | Selective recovery |
| ES2822551T3 (es) | 2014-03-17 | 2021-05-04 | Adverum Biotechnologies Inc | Composiciones y métodos para la expresión génica mejorada en células de los conos |
| 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 |
| AU2016366549B2 (en) | 2015-12-11 | 2022-11-10 | California Institute Of Technology | Targeting peptides for directing adeno-associated viruses (AAVs) |
| 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 |
| AU2017345470B2 (en) | 2016-10-19 | 2023-08-03 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
| SI3645551T1 (sl) * | 2017-06-27 | 2024-06-28 | Regeneron Pharmaceuticals, Inc. | S tropizmi spremenjeni rekombinantni virusni vektorji in njihova uporaba za tarčni vnos genetskega materiala v človeške celice |
| 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. |
| US11149256B2 (en) | 2018-09-26 | 2021-10-19 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
| 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 |
| US12668814B2 (en) | 2020-07-22 | 2026-06-30 | The Broad Institute, Inc. | Engineered muscle targeting compositions |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPH06507383A (ja) * | 1991-01-25 | 1994-08-25 | リージェンツ オブ ザ ユニバーシティ オブ ミネソタ | ラミニンのａ鎖のドメイン６のポリペプチド |
| 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 |
| JPH11511326A (ja) | 1995-08-30 | 1999-10-05 | ジエンザイム コーポレイション | アデノウィルスおよびａａｖの精製 |
| WO1997038723A1 (en) * | 1996-04-16 | 1997-10-23 | Immusol Incorporated | Targeted 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 |
| EP1135468B1 (en) * | 1998-11-10 | 2010-01-06 | University Of North Carolina At Chapel Hill | Virus vectors and methods of making and administering the same |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, 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 ES ES00951400T patent/ES2263481T3/es not_active Expired - Lifetime
- 2000-07-18 US US10/031,187 patent/US7285381B1/en not_active Expired - Fee Related
- 2000-07-18 AT AT00951400T patent/ATE328101T1/de not_active IP Right Cessation
- 2000-07-18 EP EP00951400A patent/EP1198580B1/de not_active Expired - Lifetime
- 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 DE DE50012863T patent/DE50012863D1/de 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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