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 (33)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN105120901A (zh) * | 2012-07-11 | 2015-12-02 | 宾夕法尼亚大学托管会 | Rpgr x染色体连锁视网膜退化的aav介导的基因治疗 |
| PT3628334T (pt) | 2014-03-21 | 2023-09-26 | Genzyme Corp | Terapia genética para retinopatia pigmentar |
| EP3132051B1 (en) * | 2014-04-15 | 2019-03-20 | 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 |
| SMT202000715T1 (it) * | 2014-07-24 | 2021-01-05 | Massachusetts Eye & Ear Infirmary | Terapia genica rpgr per la retinite pigmentosa |
| US20180043034A1 (en) * | 2015-03-06 | 2018-02-15 | Massachusetts Eye And Ear Infirmary | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
| CA2979229A1 (en) * | 2015-03-11 | 2016-09-15 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Rp2 vectors for treating x-linked retinitis pigmentosa |
| HK1253299A1 (zh) * | 2015-06-04 | 2019-06-14 | INSERM (Institut National de la Santé et de la Recherche Médicale) | 一种通过基因治疗治疗眼疾病的改进方法 |
| GB201516066D0 (en) | 2015-09-10 | 2015-10-28 | Young & Co Llp D | Treatment of retinitis pigmentosa |
| AU2016355343C1 (en) | 2015-11-19 | 2024-10-03 | Lloyd G. Mitchell | Compositions and methods for correction of heritable ocular disease |
| CN109069668B (zh) | 2015-12-14 | 2023-04-18 | 宾夕法尼亚州大学信托人 | 用于眼病的基因疗法 |
| CN109661470A (zh) | 2016-04-15 | 2019-04-19 | 宾夕法尼亚州大学信托人 | 新型aav8突变衣壳和含有其的组合物 |
| US11197936B2 (en) | 2016-07-08 | 2021-12-14 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treatment of disorders and diseases involving RDH12 |
| WO2018160849A1 (en) | 2017-03-01 | 2018-09-07 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| US11879133B2 (en) | 2017-04-24 | 2024-01-23 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| US11793887B2 (en) | 2017-05-31 | 2023-10-24 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating peroxisomal disorders |
| US11827898B2 (en) | 2017-06-14 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| CA3071769A1 (en) | 2017-07-31 | 2019-02-07 | Reflection Biotechnologies Limited | Cellular models of and therapies for ocular diseases |
| SMT202400321T1 (it) * | 2017-11-15 | 2024-11-15 | Univ Michigan Regents | Vettori virali comprendenti regioni codificanti rdh12 e metodi di trattamento di distrofie retiniche |
| KR20250150157A (ko) | 2018-04-17 | 2025-10-17 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 트랜스-스플라이싱 분자 |
| EP3833398A4 (en) | 2018-09-21 | 2021-12-22 | University of Connecticut | COMPOSITIONS AND METHODS OF RESTORING PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN'S SYNDROME IN HUMANS |
| CA3131023A1 (en) * | 2019-02-22 | 2020-08-27 | Michael R. Volkert | Oxr1 gene therapy |
| AU2020232692A1 (en) | 2019-03-04 | 2021-09-30 | The Trustees Of The University Of Pennsylvania | Neuroprotective gene therapy targeting the AKT pathway |
| BR112022017182A2 (pt) * | 2020-02-28 | 2022-11-08 | Univ Pennsylvania | Tratamento de bestrofinopatias autossômicas recessivas e métodos para avaliação das mesmas |
| 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 |
| IL299927A (en) * | 2020-07-29 | 2023-03-01 | Univ Florida | Improved adeno-associated virus-mediated therapies for X-linked retinoschisis |
| CA3191540A1 (en) * | 2020-09-02 | 2022-03-10 | 4D Molecular Therapeutics Inc. | Codon optimized rpgrorf 15 genes and uses thereof |
| MX2023008826A (es) | 2021-02-01 | 2023-09-15 | Regenxbio Inc | Terapia génica para lipofuscinosis neuronal ceroidea. |
| 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 |
| US20260000786A1 (en) * | 2022-07-14 | 2026-01-01 | 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 |
| 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 |
| AU1282895A (en) | 1993-12-29 | 1995-07-17 | Sumitomo Pharmaceuticals Company, Limited | Novel human ciliary neurotrophic factor |
| 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 |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| WO2004058166A2 (en) | 2002-12-23 | 2004-07-15 | Vical Incorporated | Codon-optimized polynucleotide-based vaccines against human cytomegalovirus infection |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| EP1668143B1 (en) | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| 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. |
| CA2713755A1 (en) * | 2008-02-07 | 2009-08-13 | Ceregene, Inc. | Rescue of photoreceptors by intravitreal administration 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 |
| US7561972B1 (en) | 2008-06-06 | 2009-07-14 | Dna Twopointo, Inc. | Synthetic nucleic acids for expression of encoded proteins |
| 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 |
| 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 |
| CN105120901A (zh) * | 2012-07-11 | 2015-12-02 | 宾夕法尼亚大学托管会 | Rpgr x染色体连锁视网膜退化的aav介导的基因治疗 |
-
2013
- 2013-01-23 CN CN201380045636.1A patent/CN105120901A/zh active Pending
- 2013-01-23 NZ NZ704275A patent/NZ704275A/en unknown
- 2013-01-23 EP EP13816245.8A patent/EP2872183B1/en active Active
- 2013-01-23 AU AU2013287281A patent/AU2013287281B2/en active Active
- 2013-01-23 WO PCT/US2013/022628 patent/WO2014011210A1/en not_active Ceased
- 2013-01-23 JP JP2015521593A patent/JP6199965B2/ja active Active
- 2013-01-23 US US14/413,884 patent/US9770491B2/en active Active
- 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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