BR112022010321A2 - Composições e métodos para tratamento de neuropatia óptica hereditária de leber com proteínas nadh desidrogenase - Google Patents

Composições e métodos para tratamento de neuropatia óptica hereditária de leber com proteínas nadh desidrogenase

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Publication number
BR112022010321A2
BR112022010321A2 BR112022010321A BR112022010321A BR112022010321A2 BR 112022010321 A2 BR112022010321 A2 BR 112022010321A2 BR 112022010321 A BR112022010321 A BR 112022010321A BR 112022010321 A BR112022010321 A BR 112022010321A BR 112022010321 A2 BR112022010321 A2 BR 112022010321A2
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leber
compositions
treatment
methods
nadh dehydrogenase
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BR112022010321A
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English (en)
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Li Bin
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Wuhan Neurophth Biotechnology Ltd Company
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Publication of BR112022010321A2 publication Critical patent/BR112022010321A2/pt

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Abstract

COMPOSIÇÕES E MÉTODOS PARA TRATAMENTO DE NEUROPATIA ÓPTICA HEREDITÁRIA DE LEBER COM PROTEÍNAS NADH DESIDROGENASE. A presente invenção refere-se a um ácido nucleico recombinante,compreendendo: uma sequência de direcionamento mitocondrial; uma sequência de codificação de proteína mitocondrial, em que a referida sequência de codificação de proteína mitocondrial codifica um polipeptídeo compreendendo uma proteína mitocondrial; e uma sequência de ácido nucleico 3?UTR. Também refere-se a uma composição farmacêutica compreendendo o ácido nucleico recombinante e um método de tratamento da neuropatia óptica hereditária de Leber (LHON) usando a composição farmacêutica.
BR112022010321A 2019-12-09 2020-12-09 Composições e métodos para tratamento de neuropatia óptica hereditária de leber com proteínas nadh desidrogenase BR112022010321A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CN201911250082.4A CN113025633A (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
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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BR112022010321A2 true BR112022010321A2 (pt) 2022-08-16

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US (2) US11357869B2 (pt)
EP (1) EP4073244A4 (pt)
JP (1) JP2023505522A (pt)
KR (1) KR20220112262A (pt)
CN (2) CN113025633A (pt)
AU (1) AU2020399898A1 (pt)
BR (1) BR112022010321A2 (pt)
CA (1) CA3160809A1 (pt)
MX (1) MX2022006949A (pt)
WO (1) WO2021115317A1 (pt)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240014102A (ko) 2018-06-29 2024-01-31 우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법
SG11202101032VA (en) 2018-08-20 2021-02-25 Wuhan Neurophth Biotechnology Ltd Company Compositions and methods for treating leber's hereditary optic neuropathy
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

Family Cites Families (48)

* 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
US4925673A (en) 1986-08-18 1990-05-15 Clinical Technologies Associates, Inc. Delivery systems for pharmacological agents encapsulated with proteinoids
US5075109A (en) 1986-10-24 1991-12-24 Southern Research Institute Method of potentiating an immune response
US5811128A (en) 1986-10-24 1998-09-22 Southern Research Institute Method for oral or rectal delivery of microencapsulated vaccines and compositions therefor
US4897268A (en) 1987-08-03 1990-01-30 Southern Research Institute Drug delivery system and method of making the same
EP0678034B1 (en) 1993-01-11 1999-05-26 Dana Farber Cancer Institute Inducing cytotoxic t lymphocyte responses
WO1997039776A1 (en) 1996-04-19 1997-10-30 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
CA2403804A1 (en) 2000-03-21 2001-09-27 Curagen Corporation Vegf-modulated genes and methods employing them
EP1381276A4 (en) 2001-04-13 2005-02-02 Univ Pennsylvania METHOD FOR TREATMENT OR DEVELOPMENT SLUDGE DEGRADATION
DK1751275T3 (da) 2004-06-01 2017-11-27 Avigen Inc Sammensætninger og fremgangsmåder til forebyggelse af aggregering af aav-vektorer
CA2606156C (en) * 2005-04-18 2013-01-08 Ryan Parr 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
PT1880008E (pt) 2005-05-03 2015-08-27 Inserm Inst Nat De La Santé Et De La Rech Médicale Expressão de proteína mitocondrial através de uma abordagem alotópica melhorada
EP2089412A4 (en) 2006-10-23 2010-01-27 John Guy SUPPRESSION OF OXIDATIVE STRESS OF MITOCHONDRIEN
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
WO2010040901A1 (en) * 2008-10-07 2010-04-15 Pierre Rustin Alternative oxidase and uses thereof
WO2010048352A2 (en) 2008-10-22 2010-04-29 Quark Pharmaceuticals, Inc. Methods for treating eye disorders
JP5734947B2 (ja) * 2009-03-27 2015-06-17 ミトミクス インコーポレイテッドＧｅｎｅｓｉｓ Ｇｅｎｏｍｉｃｓ Ｉｎｃ． 異常型ミトコンドリアｄｎａ、関連融合転写物および翻訳産物、並びにそのハイブリダイゼーションプローブ
CA2787827C (en) 2010-01-28 2020-11-10 The Children's Hospital Of Philadelphia 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
AU2013243953A1 (en) * 2012-04-02 2014-10-30 Modernatx, Inc. Modified polynucleotides for the production of nuclear 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
KR20170066433A (ko) 2014-09-17 2017-06-14 더 유니버시티 오브 아이오와 리서치 파운데이션 면역조절제 및 백신 성분으로서 바이러스 rna 분절
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
US10450585B2 (en) 2015-07-13 2019-10-22 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
CN110049769A (zh) 2016-12-04 2019-07-23 阿拉维·霍拉萨尼·默哈达姆·马塞尔·维克托 治疗与线粒体应激相关的疾病的方法
US20180207293A1 (en) 2017-01-25 2018-07-26 2C Tech Corp. Nanoparticles for sustained ophthalmic drug delivery and methods of use
EP4234025A3 (en) 2017-08-11 2024-04-24 Unity Biotechnology, Inc. Treatment of 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
KR20240014102A (ko) 2018-06-29 2024-01-31 우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법
WO2020001657A1 (en) 2018-06-29 2020-01-02 Wuhan Neurophth Biological Technology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
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
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
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
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
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
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
WO2020038352A1 (en) 2018-08-20 2020-02-27 Wuhan Neurophth Biological Technology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy

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