JP2018503374A5 - - Google Patents
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- JP2018503374A5 JP2018503374A5 JP2017536343A JP2017536343A JP2018503374A5 JP 2018503374 A5 JP2018503374 A5 JP 2018503374A5 JP 2017536343 A JP2017536343 A JP 2017536343A JP 2017536343 A JP2017536343 A JP 2017536343A JP 2018503374 A5 JP2018503374 A5 JP 2018503374A5
- Authority
- JP
- Japan
- Prior art keywords
- spectrin
- repeat
- promoter
- isolated
- nucleotide sequence
- 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
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562104537P | 2015-01-16 | 2015-01-16 | |
| US62/104,537 | 2015-01-16 | ||
| PCT/US2016/013733 WO2016115543A2 (en) | 2015-01-16 | 2016-01-15 | Novel micro-dystrophins and related methods of use |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021014384A Division JP7164896B2 (ja) | 2015-01-16 | 2021-02-01 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018503374A JP2018503374A (ja) | 2018-02-08 |
| JP2018503374A5 true JP2018503374A5 (enExample) | 2019-02-21 |
| JP6832280B2 JP6832280B2 (ja) | 2021-02-24 |
Family
ID=56406571
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017536343A Active JP6832280B2 (ja) | 2015-01-16 | 2016-01-15 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
| JP2021014384A Active JP7164896B2 (ja) | 2015-01-16 | 2021-02-01 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
| JP2022165111A Active JP7482549B2 (ja) | 2015-01-16 | 2022-10-14 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
| JP2024068905A Pending JP2024088814A (ja) | 2015-01-16 | 2024-04-22 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021014384A Active JP7164896B2 (ja) | 2015-01-16 | 2021-02-01 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
| JP2022165111A Active JP7482549B2 (ja) | 2015-01-16 | 2022-10-14 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
| JP2024068905A Pending JP2024088814A (ja) | 2015-01-16 | 2024-04-22 | 新規のマイクロジストロフィンおよび使用の関連する方法 |
Country Status (11)
| Country | Link |
|---|---|
| US (4) | US10479821B2 (enExample) |
| EP (2) | EP3245291B1 (enExample) |
| JP (4) | JP6832280B2 (enExample) |
| KR (1) | KR102684387B1 (enExample) |
| CN (1) | CN107250364A (enExample) |
| AU (2) | AU2016206518B2 (enExample) |
| CA (1) | CA2972811A1 (enExample) |
| IL (4) | IL297239B2 (enExample) |
| NZ (1) | NZ734019A (enExample) |
| SG (1) | SG11201705324UA (enExample) |
| WO (1) | WO2016115543A2 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11958888B2 (en) | 2015-01-16 | 2024-04-16 | University Of Washington | Micro-dystrophins and related methods of use |
Families Citing this family (51)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201507842D0 (en) * | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
| EP4279084B1 (en) | 2015-10-28 | 2025-06-11 | Vertex Pharmaceuticals Inc. | Materials and methods for treatment of duchenne muscular dystrophy |
| US11617783B2 (en) | 2015-11-16 | 2023-04-04 | Research Institute At Nationwide Children's Hospital | Repairing a mutant human titin gene using CRISPR technology |
| JP7153562B2 (ja) | 2016-04-15 | 2022-10-14 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 筋ジストロフィーを治療するためのマイクロｒｎａ－２９のアデノ随伴ウイルスベクター送達 |
| US20170362635A1 (en) * | 2016-06-20 | 2017-12-21 | University Of Washington | Muscle-specific crispr/cas9 editing of genes |
| RU2753194C2 (ru) | 2016-06-21 | 2021-08-12 | Бамбу Терапьютикс, Инк. | Оптимизированные гены и экспрессионные кассеты мини-дистрофина и их применение |
| JOP20190166A1 (ar) * | 2017-01-05 | 2019-07-02 | Univ Texas | استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي |
| BR112019015081A2 (pt) | 2017-01-23 | 2020-03-10 | Rutgers, The State University Of New Jersey | Composições para reduzir a expressão da sarcolipina e prevenir e tratar a distrofia muscular e a cardiomiopatia e os métodos de utilização |
| US10687520B2 (en) | 2017-03-07 | 2020-06-23 | The Board Of Regents Of The University Of Texas System | Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44 |
| WO2019012336A2 (en) | 2017-03-17 | 2019-01-17 | Newcastle University | ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY |
| JP7213238B2 (ja) * | 2017-10-18 | 2023-01-26 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| WO2019118806A1 (en) | 2017-12-14 | 2019-06-20 | Solid Biosciences Inc. | Non-viral production and delivery of genes |
| AU2019216257B2 (en) | 2018-01-31 | 2025-11-20 | Research Institute At Nationwide Children's Hospital | Gene therapy for limb-girdle muscular dystrophy type 2C |
| US12460226B2 (en) | 2018-04-16 | 2025-11-04 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating duchenne muscular dystrophy |
| EP4591888A3 (en) | 2018-06-18 | 2026-01-21 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| US12391928B2 (en) | 2018-06-29 | 2025-08-19 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A |
| KR20210102870A (ko) | 2018-08-30 | 2021-08-20 | 테나야 테라퓨틱스, 인코포레이티드 | 미오카르딘 및 ascl1을 사용한 심장 세포 재프로그래밍 |
| PE20211110A1 (es) * | 2018-10-24 | 2021-06-17 | Bristol Myers Squibb Co | Distrofinas miniaturizadas y usos de las mismas |
| AU2019395388A1 (en) * | 2018-12-12 | 2021-07-29 | Solid Biosciences Inc. | Combination therapy for treating muscular dystrophy |
| CN113766935A (zh) | 2019-02-26 | 2021-12-07 | 全国儿童医院研究所 | β-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗 |
| JP7635139B2 (ja) * | 2019-03-25 | 2025-02-25 | ジェネトン | オーバーラップａａｖベクターを使用する大きいサイズのクアシジストロフィンの産生 |
| AU2020284255A1 (en) * | 2019-05-30 | 2022-01-06 | Solid Biosciences Inc. | Recombinant herpesvirales vector |
| US20200405824A1 (en) * | 2019-06-26 | 2020-12-31 | University Of Washington | Use of ribonucleotide reductase alone or in combination with micro-dystrophin to treat duchenne muscular dystrophy striated muscle disease |
| SI4017871T1 (sl) | 2019-08-21 | 2024-06-28 | Research Institute At Nationwide Children's Hospital | Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije |
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| EP4065596A2 (en) * | 2019-11-28 | 2022-10-05 | RegenxBio Inc. | Microdystrophin gene therapy constructs and uses thereof |
| WO2021163357A2 (en) * | 2020-02-13 | 2021-08-19 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| BR112022015979A2 (pt) * | 2020-02-13 | 2022-10-11 | Tenaya Therapeutics Inc | Vetores de terapia genética para tratamento de doenças cardíacas |
| WO2021222332A1 (en) | 2020-04-29 | 2021-11-04 | Bristol-Myers Squibb Company | Miniaturized dystrophins having spectrin fusion domains and uses thereof |
| JP2023530974A (ja) | 2020-06-15 | 2023-07-20 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 筋ジストロフィーのためのアデノ随伴ウイルスベクター送達 |
| CN115768430A (zh) * | 2020-06-30 | 2023-03-07 | 东丽株式会社 | 伴随代谢异常的疾病或综合征中的肌力下降症状的改善剂或预防剂 |
| WO2022076750A2 (en) | 2020-10-07 | 2022-04-14 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns or muscle delivery |
| US20240003898A1 (en) | 2020-10-30 | 2024-01-04 | Pfizer Inc. | Methods for measuring dystrophin in tissue samples |
| RU2767335C1 (ru) * | 2021-03-02 | 2022-03-17 | Общество с ограниченной ответственностью «Марлин Биотех» | Химерные белки на основе утрофина и дистрофина человека и их применение для лечения миодистрофии Дюшенна |
| CA3218333A1 (en) | 2021-04-05 | 2022-10-13 | Solid Biosciences Inc. | Recombinant herpesvirales vector |
| EP4707401A2 (en) * | 2021-04-09 | 2026-03-11 | University of Washington | Artificial regulatory cassettes for muscle-specific gene expression |
| EP4329821A1 (en) * | 2021-04-26 | 2024-03-06 | RegenxBio Inc. | Microdystrophin gene therapy administration for treatment of dystrophinopathies |
| US20240358857A1 (en) * | 2021-05-11 | 2024-10-31 | Regenxbio Inc. | Treatment of duchenne muscular dystrophy and combinations thereof |
| EP4108263A3 (en) | 2021-06-02 | 2023-03-22 | Research Institute at Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
| US20240269329A1 (en) * | 2021-06-07 | 2024-08-15 | University Of Florida Research Foundation, Incorporated | Gene therapy for duchenne muscular dystrophy |
| IL310725A (en) | 2021-08-11 | 2024-04-01 | Solid Biosciences Inc | Treatment of muscular dystrophy |
| JP2024531138A (ja) * | 2021-08-11 | 2024-08-29 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | 筋ジストロフィーを処置するための組成物および方法 |
| US20250001006A1 (en) | 2021-10-07 | 2025-01-02 | Regenxbio Inc. | Recombinant adeno-associated viruses for targeted delivery |
| EP4219726A1 (en) | 2021-10-15 | 2023-08-02 | Research Institute at Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| WO2023081850A1 (en) | 2021-11-04 | 2023-05-11 | Regeneron Pharmaceuticals, Inc. | Viral particles retargeted to skeletal muscle |
| CN114316070B (zh) * | 2021-12-29 | 2022-11-15 | 上海勉亦生物科技有限公司 | 用于治疗肌营养不良症的转基因表达盒 |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| US20250288641A1 (en) * | 2022-04-27 | 2025-09-18 | The Curators Of The University Of Missouri | Micro-dystrophin for heart protection |
| WO2024138158A2 (en) | 2022-12-23 | 2024-06-27 | Ultragenyx Pharmaceutical Inc. | Method of producing soluble microdystrophin protein and uses of the same |
| KR20250150011A (ko) | 2023-02-13 | 2025-10-17 | 리제너론 파마슈티칼스 인코포레이티드 | 항-인간 cacng1 항체를 이용한 근육 관련 장애 치료 |
| WO2024216244A2 (en) | 2023-04-13 | 2024-10-17 | Regenxbio Inc. | Targeting aav capsids, methods of manufacturing and using same |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE60134786D1 (de) | 2000-10-06 | 2008-08-21 | Univ Michigan | Mini-dystrophin nukleinsäure- und peptidsequenzen |
| US20070202587A1 (en) | 2002-09-23 | 2007-08-30 | Applied Genetic Technologies Corporation | Recombinant AAV production in mammalian cells |
| DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| US20080044393A1 (en) * | 2004-07-16 | 2008-02-21 | White Robert L | Retinal dystrophin transgene and methods of use thereof |
| US7436622B2 (en) * | 2006-07-31 | 2008-10-14 | Imation Corp. | Concurrent servo and data track writing |
| WO2008088895A2 (en) | 2007-01-18 | 2008-07-24 | University Of Missouri-Columbia | Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma |
| AU2009209408B2 (en) | 2008-01-29 | 2015-06-11 | Beacon Therapeutics Limited | Recombinant virus production using mammalian cells in suspension |
| WO2010055781A1 (ja) * | 2008-11-11 | 2010-05-20 | インターナショナル・ビジネス・マシーンズ・コーポレーション | 提供装置、システム、サーバ装置、プログラム及び方法 |
| BR112013030209A2 (pt) * | 2011-05-26 | 2017-06-13 | Univ Washington | métodos baseados em célula e gene para melhorar função cardíaca |
| US20130136729A1 (en) * | 2011-11-11 | 2013-05-30 | University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group | Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors |
| US9624282B2 (en) * | 2012-11-26 | 2017-04-18 | The Curators Of The University Of Missouri | Microdystrophin peptides and methods for treating muscular dystrophy using the same |
| US20150329609A1 (en) * | 2014-03-03 | 2015-11-19 | Sujata Acharjee | Chimeric dystrophin proteins to treat dystrophinopathies |
| EP2960336A1 (en) | 2014-06-27 | 2015-12-30 | Genethon | Efficient systemic treatment of dystrophic muscle pathologies |
| US10479821B2 (en) | 2015-01-16 | 2019-11-19 | University Of Washington | Micro-dystrophins and related methods of use |
-
2016
- 2016-01-15 US US15/541,870 patent/US10479821B2/en active Active
- 2016-01-15 EP EP16738022.9A patent/EP3245291B1/en active Active
- 2016-01-15 EP EP25169094.7A patent/EP4599851A3/en active Pending
- 2016-01-15 KR KR1020177022718A patent/KR102684387B1/ko active Active
- 2016-01-15 JP JP2017536343A patent/JP6832280B2/ja active Active
- 2016-01-15 WO PCT/US2016/013733 patent/WO2016115543A2/en not_active Ceased
- 2016-01-15 IL IL297239A patent/IL297239B2/en unknown
- 2016-01-15 IL IL286316A patent/IL286316B2/en unknown
- 2016-01-15 NZ NZ734019A patent/NZ734019A/en unknown
- 2016-01-15 IL IL315273A patent/IL315273A/en unknown
- 2016-01-15 CN CN201680005836.8A patent/CN107250364A/zh active Pending
- 2016-01-15 SG SG11201705324UA patent/SG11201705324UA/en unknown
- 2016-01-15 AU AU2016206518A patent/AU2016206518B2/en active Active
- 2016-01-15 CA CA2972811A patent/CA2972811A1/en active Pending
-
2017
- 2017-06-29 IL IL253246A patent/IL253246B/en unknown
-
2019
- 2019-10-04 US US16/593,840 patent/US12421285B2/en active Active
- 2019-10-04 US US16/593,833 patent/US11958888B2/en active Active
-
2020
- 2020-05-22 AU AU2020203358A patent/AU2020203358B2/en active Active
-
2021
- 2021-02-01 JP JP2021014384A patent/JP7164896B2/ja active Active
-
2022
- 2022-10-14 JP JP2022165111A patent/JP7482549B2/ja active Active
-
2024
- 2024-04-22 JP JP2024068905A patent/JP2024088814A/ja active Pending
-
2025
- 2025-08-22 US US19/307,571 patent/US20250376496A1/en active Pending
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11958888B2 (en) | 2015-01-16 | 2024-04-16 | University Of Washington | Micro-dystrophins and related methods of use |
| US12421285B2 (en) | 2015-01-16 | 2025-09-23 | University Of Washington | Micro-dystrophins and related methods of use |
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