JP2023505522A - Ｎａｄｈデヒドロゲナーゼタンパク質でレーベル遺伝性視神経症を治療するための組成物及び方法 - Google Patents

Ｎａｄｈデヒドロゲナーゼタンパク質でレーベル遺伝性視神経症を治療するための組成物及び方法 Download PDF

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JP2023505522A
JP2023505522A JP2022534671A JP2022534671A JP2023505522A JP 2023505522 A JP2023505522 A JP 2023505522A JP 2022534671 A JP2022534671 A JP 2022534671A JP 2022534671 A JP2022534671 A JP 2022534671A JP 2023505522 A JP2023505522 A JP 2023505522A
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nucleic acid
sequence
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リー，ビン
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Wuhan Neurophth Biotechnology Ltd Co
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JP2022534671A 2019-12-09 2020-12-09 Ｎａｄｈデヒドロゲナーゼタンパク質でレーベル遺伝性視神経症を治療するための組成物及び方法 Pending JP2023505522A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN201911250082.4A CN113025633B (zh) 2019-12-09 2019-12-09 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用
CN201911250082.4 2019-12-09
PCT/CN2020/134859 WO2021115317A1 (en) 2019-12-09 2020-12-09 Compositions and methods for treating leber's hereditary optic neuropathy with nadh dehydrogenase proteins

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JP2023505522A true JP2023505522A (ja) 2023-02-09
JP2023505522A5 JP2023505522A5 (https=) 2023-12-08

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US (2) US11357869B2 (https=)
EP (1) EP4073244A4 (https=)
JP (1) JP2023505522A (https=)
KR (1) KR20220112262A (https=)
CN (2) CN113025633B (https=)
AU (1) AU2020399898A1 (https=)
BR (1) BR112022010321A2 (https=)
CA (1) CA3160809A1 (https=)
MX (1) MX2022006949A (https=)
WO (1) WO2021115317A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113528510A (zh) 2018-06-29 2021-10-22 武汉纽福斯生物科技有限公司 治疗遗传性视神经病变的组合物和方法
SG11202101032VA (en) 2018-08-20 2021-02-25 Wuhan Neurophth Biotechnology Ltd Company Compositions and methods for treating leber's hereditary optic neuropathy
AR126727A1 (es) * 2021-08-06 2023-11-08 Wuhan Neurophth Biotechnology Ltd Company Composición y método para tratar la neuropatía óptica hereditaria de leber causada por la mutación nd4
CN115927401B (zh) * 2021-08-23 2025-05-16 纽福斯(苏州)生物科技有限公司 编码nadh泛醌氧化还原酶4号亚基的核酸及其用途
CN114213549B (zh) * 2021-12-24 2024-01-05 上海生物芯片有限公司 定位于线粒体的融合蛋白、接头及其用途
JP2026501611A (ja) * 2022-12-29 2026-01-16 エッジーン インコーポレイテッド レーベル遺伝性視神経症のミトコンドリア塩基変異矯正システム

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090306188A1 (en) * 2005-05-03 2009-12-10 Inserm (Institut National De Al Sante Et De Al Rec Importation of mitochondrial protein by an enhanced allotopic approach
WO2010040901A1 (en) * 2008-10-07 2010-04-15 Pierre Rustin Alternative oxidase and uses thereof
WO2013151663A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins

Family Cites Families (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3625214A (en) 1970-05-18 1971-12-07 Alza Corp Drug-delivery device
US4906474A (en) 1983-03-22 1990-03-06 Massachusetts Institute Of Technology Bioerodible polyanhydrides for controlled drug delivery
US4789734A (en) 1985-08-06 1988-12-06 La Jolla Cancer Research Foundation Vitronectin specific cell receptor derived from mammalian mesenchymal tissue
AU610083B2 (en) 1986-08-18 1991-05-16 Clinical Technologies Associates, Inc. Delivery systems for pharmacological agents
US6024983A (en) 1986-10-24 2000-02-15 Southern Research Institute Composition for delivering bioactive agents for immune response and its preparation
US5075109A (en) 1986-10-24 1991-12-24 Southern Research Institute Method of potentiating an immune response
US4897268A (en) 1987-08-03 1990-01-30 Southern Research Institute Drug delivery system and method of making the same
DK0678034T3 (da) 1993-01-11 1999-11-08 Dana Farber Cancer Inst Inc Induktion af cytotoksiske T-lymfocytreaktioner
AU2668897A (en) 1996-04-19 1997-11-12 Genetic Therapy, Inc. Gene therapy involving concurrent and repeated administration of adenoviruses and immunosuppressive agents
US5814451A (en) * 1997-01-17 1998-09-29 Incyte Pharmaceuticals, Inc. Subunits of NADH dehydrogenase
US6207392B1 (en) 1997-11-25 2001-03-27 The Regents Of The University Of California Semiconductor nanocrystal probes for biological applications and process for making and using such probes
CN1270222A (zh) * 2000-03-17 2000-10-18 国家人类基因组南方研究中心 一种新的人辅酶ⅰ亚基异构体蛋白及其编码序列
US20020132978A1 (en) 2000-03-21 2002-09-19 Hans-Peter Gerber VEGF-modulated genes and methods employing them
WO2002082904A2 (en) 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
BRPI0511764B8 (pt) 2004-06-01 2021-05-25 Avigen Inc método de prevenção de agregação de vírions de vírus adeno-associado recombinante (raav) em uma preparação purificada de virions raav
EP1877559B1 (en) * 2005-04-18 2010-10-13 Mitomics Inc. Mitochondrial mutations and rearrangements as a diagnostic tool for the detection of sun exposure, prostate cancer and other cancers
US20130022979A1 (en) 2005-04-18 2013-01-24 Genesis Genomics Inc. 3.4kb MITOCHONDRIAL DNA DELETION FOR USE IN THE DETECTION OF CANCER
AU2007324073A1 (en) 2006-10-23 2008-05-29 John Guy Suppression of mitochondrial oxidative stress
WO2008155397A2 (en) 2007-06-20 2008-12-24 Galapagos N.V. Molecular targets and compounds, and methods to identify the same, useful in the treatment of bone and joint degenerative diseases
CA2962219C (en) 2008-10-22 2020-08-25 Quark Pharmaceuticals, Inc. Methods for treating eye disorders
RU2011137486A (ru) * 2009-03-27 2013-05-10 Митомикс Инк. Аберрантная митохондриальная днк, ассоциированные с ней слитые транскрипты, продукты трансляции и гибрадизационные зонды
WO2011094198A1 (en) 2010-01-28 2011-08-04 The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
CN102517304B (zh) 2011-12-16 2013-08-28 中国农业科学院生物技术研究所 重组葡萄糖氧化酶的优化基因及其表达载体和应用
US20160289674A1 (en) 2012-04-02 2016-10-06 Moderna Therapeutics, Inc. Modified polynucleotides for the production of membrane proteins
CN102634527B (zh) 2012-04-11 2013-11-06 华中科技大学同济医学院附属同济医院 重组人nadh脱氢酶亚单位4基因及其表达载体构建方法
TWI690322B (zh) 2012-10-02 2020-04-11 法商傳斯堅公司 含病毒的調配物及其使用
SG11201702116VA (en) 2014-09-17 2017-04-27 Univ Iowa Res Found Viral rna segments as immunomodulatory agents and vaccine components
CN104450747B (zh) * 2014-09-23 2018-02-09 武汉纽福斯生物科技有限公司 用于治疗Leber遗传性视神经病变的重组腺相关病毒‑NADH脱氢酶亚单位4基因全长以及药剂
AU2016291778B2 (en) 2015-07-13 2021-05-06 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
EP3548039B1 (en) 2016-12-04 2023-07-19 Alavi Khorassani Moghadam, Marcel Victor Ribavirin for use in the treatment of a mitochondrial disease
US20180207293A1 (en) 2017-01-25 2018-07-26 2C Tech Corp. Nanoparticles for sustained ophthalmic drug delivery and methods of use
WO2019033119A1 (en) 2017-08-11 2019-02-14 Unity Biotechnology, Inc. TREATMENT OF OPHTHALMIC CONDITIONS SUCH AS MACULAR DEGENERATION, GLAUCOMA AND DIABETIC RETINOPATHY USING PHARMACEUTICAL AGENTS THAT ELIMINATE SENESCENT CELLS
CN109970861B (zh) * 2017-12-27 2021-06-11 武汉纽福斯生物科技有限公司 一种靶向线粒体的nd4融合蛋白及其制备方法和应用
US20210163898A1 (en) 2018-04-16 2021-06-03 The Children's Hospital Of Philadelphia Mitochondrial rna import for treating mitochondrial disease
CA3103231A1 (en) 2018-06-11 2019-12-19 Serge FITOUSSI Recombinant aav vectors and methods of using the same
WO2020000641A1 (zh) 2018-06-29 2020-01-02 武汉纽福斯生物科技有限公司 编码人nadh脱氢酶亚单位蛋白的核酸及其应用
WO2020010491A1 (zh) 2018-07-09 2020-01-16 武汉纽福斯生物科技有限公司 编码人nadh脱氢酶亚单位4蛋白的核酸及其应用
CN110846392A (zh) 2018-08-20 2020-02-28 武汉纽福斯生物科技有限公司 一种重组腺相关病毒或含其的试剂盒及其应用
CN111068071A (zh) 2018-10-22 2020-04-28 武汉纽福斯生物科技有限公司 基因治疗Leber遗传学视神经病变
CN113528510A (zh) * 2018-06-29 2021-10-22 武汉纽福斯生物科技有限公司 治疗遗传性视神经病变的组合物和方法
CN111073899B (zh) 2018-10-19 2021-01-01 武汉纽福斯生物科技有限公司 一种编码人nadh脱氢酶亚单位4蛋白的核酸及其应用
SG11202101032VA (en) * 2018-08-20 2021-02-25 Wuhan Neurophth Biotechnology Ltd Company Compositions and methods for treating leber's hereditary optic neuropathy

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090306188A1 (en) * 2005-05-03 2009-12-10 Inserm (Institut National De Al Sante Et De Al Rec Importation of mitochondrial protein by an enhanced allotopic approach
WO2010040901A1 (en) * 2008-10-07 2010-04-15 Pierre Rustin Alternative oxidase and uses thereof
WO2013151663A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
HOMO SAPIENS MITOCHONDRION, COMPLETE GENOME, GENBANK[ONLINE], JPN6025003162, 31 October 2014 (2014-10-31), ISSN: 0005651246 *

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