JP2015523379A5 - - Google Patents
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- Publication number
- JP2015523379A5 JP2015523379A5 JP2015521593A JP2015521593A JP2015523379A5 JP 2015523379 A5 JP2015523379 A5 JP 2015523379A5 JP 2015521593 A JP2015521593 A JP 2015521593A JP 2015521593 A JP2015521593 A JP 2015521593A JP 2015523379 A5 JP2015523379 A5 JP 2015523379A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- rpgr
- nucleic acid
- seq
- 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 |
|---|---|---|---|
| US201261670355P | 2012-07-11 | 2012-07-11 | |
| US61/670,355 | 2012-07-11 | ||
| PCT/US2013/022628 WO2014011210A1 (en) | 2012-07-11 | 2013-01-23 | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015523379A JP2015523379A (ja) | 2015-08-13 |
| JP2015523379A5 true JP2015523379A5 (enExample) | 2016-03-03 |
| JP6199965B2 JP6199965B2 (ja) | 2017-09-20 |
Family
ID=49916455
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015521593A Active JP6199965B2 (ja) | 2012-07-11 | 2013-01-23 | Ｒｐｇｒｘ連鎖性網膜変性のａａｖ媒介型遺伝子治療 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US9770491B2 (enExample) |
| EP (1) | EP2872183B1 (enExample) |
| JP (1) | JP6199965B2 (enExample) |
| CN (1) | CN105120901A (enExample) |
| AU (1) | AU2013287281B2 (enExample) |
| CA (1) | CA2878171C (enExample) |
| NZ (1) | NZ704275A (enExample) |
| WO (1) | WO2014011210A1 (enExample) |
Families Citing this family (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6199965B2 (ja) * | 2012-07-11 | 2017-09-20 | ザ・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 | Ｒｐｇｒｘ連鎖性網膜変性のａａｖ媒介型遺伝子治療 |
| EP3628334B1 (en) | 2014-03-21 | 2023-06-28 | Genzyme Corporation | Gene therapy for retinitis pigmentosa |
| US9534225B2 (en) * | 2014-04-15 | 2017-01-03 | Applied Genetic Technologies Corporation | Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (RPGR) |
| GB201412011D0 (en) * | 2014-07-04 | 2014-08-20 | Ucl Business Plc | Treatments |
| PT3191139T (pt) * | 2014-07-24 | 2020-11-05 | Massachusetts Eye & Ear Infirmary | Terapia do gene rpgr para retinite pigmentosa |
| PT3265568T (pt) * | 2015-03-06 | 2020-08-20 | Massachusetts Eye & Ear Infirmary | Terapias de aumento de genes para a degeneração hereditária da retina causada por mutações no gene prpf31 |
| US10646588B2 (en) * | 2015-03-11 | 2020-05-12 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | RP2 and RPGR vectors for treating X-linked retinitis pigmentosa |
| EP3302521A1 (en) * | 2015-06-04 | 2018-04-11 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Improved methods for treating ocular diseases by gene therapy |
| GB201516066D0 (en) | 2015-09-10 | 2015-10-28 | Young & Co Llp D | Treatment of retinitis pigmentosa |
| WO2017087900A1 (en) | 2015-11-19 | 2017-05-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods for correction of heritable ocular disease |
| JP7057281B2 (ja) | 2015-12-14 | 2022-04-19 | ザ・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 | 眼疾患のための遺伝子療法 |
| AU2017248656B2 (en) | 2016-04-15 | 2023-07-27 | The Trustees Of The University Of Pennsylvania | Novel AAV8 mutant capsids and compositions containing same |
| KR102526506B1 (ko) | 2016-07-08 | 2023-05-03 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물 |
| CN119752819A (zh) | 2017-03-01 | 2025-04-04 | 宾夕法尼亚州立大学托管会 | 用于眼部病症的基因疗法 |
| US11879133B2 (en) | 2017-04-24 | 2024-01-23 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| CA3098592A1 (en) | 2017-05-31 | 2018-12-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating peroxisomal disorders |
| WO2018232149A1 (en) | 2017-06-14 | 2018-12-20 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| WO2019099696A1 (en) * | 2017-11-15 | 2019-05-23 | The Regents Of The University Of Michigan | Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies |
| AU2019255708B2 (en) | 2018-04-17 | 2026-02-12 | Ascidian Therapeutics, Inc. | Trans-splicing molecules |
| CA3113648A1 (en) | 2018-09-21 | 2020-03-26 | University Of Connecticut | Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome |
| US20220054655A1 (en) * | 2019-02-22 | 2022-02-24 | University Of Massachusetts | Oxr1 gene therapy |
| JP7637060B2 (ja) | 2019-03-04 | 2025-02-27 | ザ・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 | Ａｋｔ経路を標的とする神経保護遺伝子療法 |
| US20230139443A1 (en) * | 2020-02-28 | 2023-05-04 | The Trustees Of The University Of Pennsylvania | Treating autosomal dominant bestrophinopathies and methods for evaluating same |
| CN113952472A (zh) * | 2020-07-21 | 2022-01-21 | 英斯培瑞有限公司 | 用于治疗眼部疾病的组合物和方法 |
| KR102825852B1 (ko) * | 2020-09-02 | 2025-06-26 | 4디 몰레큘러 테라퓨틱스 아이엔씨. | 코돈 최적화된 rpgrorf15 유전자 및 이의 용도 |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| KR20250011939A (ko) | 2022-05-13 | 2025-01-22 | 아시디엔 테라퓨틱스, 인크. | Abca4 트랜스-스플라이싱 분자 |
| CN117165596A (zh) * | 2022-05-27 | 2023-12-05 | 武汉纽福斯生物科技有限公司 | 编码rpgr的核酸及其应用 |
| EP4555095A2 (en) * | 2022-07-14 | 2025-05-21 | Fondation Asile Des Aveugles | Gene therapy for fam161a-associated retinopathies and other ciliopathies |
| US12252518B2 (en) | 2023-01-06 | 2025-03-18 | Life Biosciences, Inc. | Methods of treating non-arteritic anterior ischemic optic neuropathy |
| CN117050951B (zh) * | 2023-08-24 | 2024-06-04 | 广州医药研究总院有限公司 | X连锁视网膜色素变性模型犬的构建方法 |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| WO1995018150A1 (en) | 1993-12-29 | 1995-07-06 | Sumitomo Pharmaceuticals Company, Limited | Novel human ciliary neurotrophic factor |
| JP3768257B2 (ja) * | 1993-12-29 | 2006-04-19 | 大日本住友製薬株式会社 | 改変タンパク質 |
| US6372250B1 (en) * | 2000-04-25 | 2002-04-16 | The Regents Of The University Of California | Non-invasive gene targeting to the brain |
| CA2442670A1 (en) * | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
| US20030003582A1 (en) * | 2001-05-08 | 2003-01-02 | Tranzyme, Inc. | Trans-viral vector mediated gene transfer to the retina |
| WO2003052051A2 (en) | 2001-12-17 | 2003-06-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences |
| PT2311848E (pt) | 2002-12-23 | 2013-10-03 | Vical Inc | Vacinas à base de polinucleótido optimizadas por codão contra a infecção do citomegalovírus humano |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| ES2331342B1 (es) | 2006-05-22 | 2010-10-13 | Consejo Superior Investg.Cientificas | Uso de la proinsulina para la elaboracion de una composicion farmaceutica neuroprotectora, composicion terapeutica que la contiene y sus aplicaciones. |
| WO2009100253A1 (en) * | 2008-02-07 | 2009-08-13 | Ceregene, Inc. | Rescue of photoreceptors by intravitreal administation of an expression vector encoding a therapeutic protein |
| US20100081707A1 (en) * | 2008-02-21 | 2010-04-01 | Ali Robin R | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| US7561973B1 (en) | 2008-07-31 | 2009-07-14 | Dna Twopointo, Inc. | Methods for determining properties that affect an expression property value of polynucleotides in an expression system |
| US7561972B1 (en) | 2008-06-06 | 2009-07-14 | Dna Twopointo, Inc. | Synthetic nucleic acids for expression of encoded proteins |
| ITMI20090284A1 (it) | 2009-02-26 | 2010-08-27 | Consiglio Nazionale Ricerche | Uso di inibitori della serina palmitoiltrasferasi per la prevenzione e il rallentamento delle degenerazioni retiniche ereditarie e relative composizioni |
| GB0911870D0 (en) * | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| JP6199965B2 (ja) * | 2012-07-11 | 2017-09-20 | ザ・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 | Ｒｐｇｒｘ連鎖性網膜変性のａａｖ媒介型遺伝子治療 |
-
2013
- 2013-01-23 JP JP2015521593A patent/JP6199965B2/ja active Active
- 2013-01-23 NZ NZ704275A patent/NZ704275A/en unknown
- 2013-01-23 US US14/413,884 patent/US9770491B2/en active Active
- 2013-01-23 WO PCT/US2013/022628 patent/WO2014011210A1/en not_active Ceased
- 2013-01-23 AU AU2013287281A patent/AU2013287281B2/en active Active
- 2013-01-23 EP EP13816245.8A patent/EP2872183B1/en active Active
- 2013-01-23 CN CN201380045636.1A patent/CN105120901A/zh active Pending
- 2013-01-23 CA CA2878171A patent/CA2878171C/en active Active
-
2017
- 2017-09-08 US US15/699,262 patent/US10383922B2/en active Active
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