JP2003533170A5 - - Google Patents
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- Publication number
- JP2003533170A5 JP2003533170A5 JP2001528616A JP2001528616A JP2003533170A5 JP 2003533170 A5 JP2003533170 A5 JP 2003533170A5 JP 2001528616 A JP2001528616 A JP 2001528616A JP 2001528616 A JP2001528616 A JP 2001528616A JP 2003533170 A5 JP2003533170 A5 JP 2003533170A5
- Authority
- JP
- Japan
- Prior art keywords
- dna segment
- aav
- recombinant
- raav
- terminal repeat
- 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.)
- Pending
Links
- 108020004414 DNA Proteins 0.000 description 72
- 108020004511 Recombinant DNA Proteins 0.000 description 37
- 102000053602 DNA Human genes 0.000 description 33
- 108700026244 Open Reading Frames Proteins 0.000 description 31
- 238000000034 method Methods 0.000 description 26
- 108090000623 proteins and genes Proteins 0.000 description 26
- 239000013598 vector Substances 0.000 description 15
- 108700029229 Transcriptional Regulatory Elements Proteins 0.000 description 12
- 230000001225 therapeutic effect Effects 0.000 description 12
- 241000702421 Dependoparvovirus Species 0.000 description 9
- 102000040430 polynucleotide Human genes 0.000 description 9
- 108091033319 polynucleotide Proteins 0.000 description 9
- 239000002157 polynucleotide Substances 0.000 description 9
- 102000004169 proteins and genes Human genes 0.000 description 9
- 239000003623 enhancer Substances 0.000 description 7
- 229920001184 polypeptide Polymers 0.000 description 5
- 102000004196 processed proteins & peptides Human genes 0.000 description 5
- 108090000765 processed proteins & peptides Proteins 0.000 description 5
- 230000035897 transcription Effects 0.000 description 5
- 238000013518 transcription Methods 0.000 description 5
- 230000002103 transcriptional effect Effects 0.000 description 5
- 239000013607 AAV vector Substances 0.000 description 4
- 239000003937 drug carrier Substances 0.000 description 4
- 239000003981 vehicle Substances 0.000 description 4
- 208000002267 Anti-neutrophil cytoplasmic antibody-associated vasculitis Diseases 0.000 description 3
- 230000002708 enhancing effect Effects 0.000 description 3
- 108020005067 RNA Splice Sites Proteins 0.000 description 1
- 239000013612 plasmid Substances 0.000 description 1
- 230000006798 recombination Effects 0.000 description 1
- 238000005215 recombination Methods 0.000 description 1
- 230000001105 regulatory effect Effects 0.000 description 1
- 239000000126 substance Substances 0.000 description 1
- 241000701161 unidentified adenovirus Species 0.000 description 1
- 239000013603 viral vector Substances 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US15820999P | 1999-10-07 | 1999-10-07 | |
| US60/158,209 | 1999-10-07 | ||
| PCT/US2000/027863 WO2001025465A1 (en) | 1999-10-07 | 2000-10-06 | Adeno-associated viruses and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2003533170A JP2003533170A (ja) | 2003-11-11 |
| JP2003533170A5 true JP2003533170A5 (https=) | 2007-11-22 |
Family
ID=22567109
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2001528616A Pending JP2003533170A (ja) | 1999-10-07 | 2000-10-06 | アデノ随伴ウイルスおよびその使用 |
Country Status (4)
| Country | Link |
|---|---|
| EP (1) | EP1224313A1 (https=) |
| JP (1) | JP2003533170A (https=) |
| CA (1) | CA2386546A1 (https=) |
| WO (1) | WO2001025465A1 (https=) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6924128B2 (en) | 1994-12-06 | 2005-08-02 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant AAV vectors |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6346415B1 (en) | 1997-10-21 | 2002-02-12 | Targeted Genetics Corporation | Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors |
| US6436392B1 (en) | 1998-05-20 | 2002-08-20 | University Of Iowa Research Foundation | Adeno-associated virus vectors |
| US6893865B1 (en) | 1999-04-28 | 2005-05-17 | Targeted Genetics Corporation | Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles |
| JP4969002B2 (ja) | 1999-06-08 | 2012-07-04 | ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン | ｒＡＡＶ形質導入を増加するための化合物および方法 |
| US7241447B1 (en) | 1999-10-07 | 2007-07-10 | University Of Iowa Research Foundation | Adeno-associated virus vectors and uses thereof |
| US8067156B2 (en) | 2001-05-31 | 2011-11-29 | The Rockefeller University | Method for generating replication defective viral vectors that are helper free |
| CA2453405A1 (en) * | 2001-07-13 | 2003-01-23 | University Of Iowa Research Foundation | Pseudotyped adeno-associated viruses and uses thereof |
| AU2004227358B8 (en) | 2003-03-31 | 2009-12-10 | Targeted Genetics Corporation | Compounds and methods to enhance rAAV transduction |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| EP3384035A4 (en) | 2015-12-02 | 2019-08-07 | Voyager Therapeutics, Inc. | ASSAYS FOR THE DETECTION OF AAV-NEUTRALIZING ANTIBODIES |
| WO2017139381A1 (en) | 2016-02-08 | 2017-08-17 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
| CA3016985C (en) | 2016-03-07 | 2023-07-04 | University Of Iowa Research Foundation | Aav-mediated expression using a synthetic promoter and enhancer |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| KR102427379B1 (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| WO2017205739A1 (en) | 2016-05-26 | 2017-11-30 | University Of Iowa Research Foundation | cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1 |
| WO2018044933A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| US11142775B2 (en) | 2017-01-13 | 2021-10-12 | University Of Iowa Research Foundation | Bocaparvovirus small noncoding RNA and uses thereof |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | ハンチントン病治療組成物および方法 |
| JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CA3070087A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| EP3808849A1 (en) | 2017-08-03 | 2021-04-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| EP3697908A1 (en) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| US20210002667A1 (en) * | 2018-03-15 | 2021-01-07 | Intergalactic Therapeutics, Inc. | Synthetic dna vectors and methods of use |
| US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| CN112703016B (zh) | 2018-04-27 | 2024-11-22 | 分贝治疗公司 | 肌球蛋白15启动子及其用途 |
| KR20210019996A (ko) | 2018-05-15 | 2021-02-23 | 보이저 테라퓨틱스, 인크. | 파킨슨병의 치료를 위한 조성물 및 방법 |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| EP3921032A4 (en) | 2019-02-08 | 2022-11-30 | Decibel Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS |
| CA3129422A1 (en) | 2019-02-08 | 2020-08-13 | Decibel Therapeutics, Inc. | Myosin 15 promoters and uses thereof |
| FI3953483T3 (fi) | 2019-04-11 | 2023-11-30 | Regenxbio Inc | Kokoekskluusiokromatografiamenetelmiä rekombinanttien adenoassosioituneiden viruskoostumusten karakterisoimiseksi |
| KR20220128607A (ko) * | 2019-09-18 | 2022-09-21 | 인터갈락틱 테라퓨틱스, 인크. | 합성 dna 벡터 및 사용 방법 |
| KR20220130093A (ko) | 2019-10-30 | 2022-09-26 | 데시벨 테라퓨틱스, 인크. | 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법 |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) * | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| JP2001500497A (ja) * | 1996-09-06 | 2001-01-16 | トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法 |
| US6436392B1 (en) * | 1998-05-20 | 2002-08-20 | University Of Iowa Research Foundation | Adeno-associated virus vectors |
-
2000
- 2000-10-06 EP EP00970689A patent/EP1224313A1/en not_active Withdrawn
- 2000-10-06 JP JP2001528616A patent/JP2003533170A/ja active Pending
- 2000-10-06 WO PCT/US2000/027863 patent/WO2001025465A1/en not_active Ceased
- 2000-10-06 CA CA002386546A patent/CA2386546A1/en not_active Abandoned
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