MX2020013772A - Composiciones y métodos para el tratamiento de la neuropatía óptica hereditaria de leber. - Google Patents

Composiciones y métodos para el tratamiento de la neuropatía óptica hereditaria de leber.

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Publication number
MX2020013772A
MX2020013772A MX2020013772A MX2020013772A MX2020013772A MX 2020013772 A MX2020013772 A MX 2020013772A MX 2020013772 A MX2020013772 A MX 2020013772A MX 2020013772 A MX2020013772 A MX 2020013772A MX 2020013772 A MX2020013772 A MX 2020013772A
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MX
Mexico
Prior art keywords
optic neuropathy
hereditary optic
compositions
methods
nucleic acid
Prior art date
Application number
MX2020013772A
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English (en)
Inventor
Bin Li
Original Assignee
Wuhan Neurophth Biotechnology Ltd Company
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Priority claimed from CN201810702492.7A external-priority patent/CN110656117A/zh
Priority claimed from CN201810703168.7A external-priority patent/CN110724695A/zh
Priority claimed from PCT/CN2018/095023 external-priority patent/WO2020010491A1/zh
Priority claimed from CN201810948193.1A external-priority patent/CN110846392A/zh
Priority claimed from PCT/CN2018/103937 external-priority patent/WO2020000641A1/zh
Priority claimed from CN201811221305.XA external-priority patent/CN111073899B/zh
Priority claimed from CN201811230856.2A external-priority patent/CN111068071A/zh
Application filed by Wuhan Neurophth Biotechnology Ltd Company filed Critical Wuhan Neurophth Biotechnology Ltd Company
Priority claimed from PCT/CN2019/094136 external-priority patent/WO2020001657A1/en
Publication of MX2020013772A publication Critical patent/MX2020013772A/es

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Abstract

En la presente se describe un ácido nucleico recombinante, que comprende: una secuencia de direccionamiento mitocondrial; una secuencia codificante de la proteína mitocondrial, en donde dicha secuencia codificante de la proteína mitocondrial codifica un polipéptido que comprende una proteína mitocondrial; y una secuencia de ácido nucleico 3'UTR. También se describe una composición farmacéutica que comprende el ácido nucleico recombinante y un método para tratar la neuropatía óptica hereditaria de Leber (LHON) mediante el uso de la composición farmacéutica.
MX2020013772A 2018-06-29 2019-07-01 Composiciones y métodos para el tratamiento de la neuropatía óptica hereditaria de leber. MX2020013772A (es)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
CN201810702492.7A CN110656117A (zh) 2018-06-29 2018-06-29 一种编码人nadh脱氢酶亚单位6蛋白的核酸及其应用
CN201810703168.7A CN110724695A (zh) 2018-06-29 2018-06-29 一种编码人nadh脱氢酶亚单位1蛋白的核酸及其应用
PCT/CN2018/095023 WO2020010491A1 (zh) 2018-07-09 2018-07-09 编码人nadh脱氢酶亚单位4蛋白的核酸及其应用
CN201810948193.1A CN110846392A (zh) 2018-08-20 2018-08-20 一种重组腺相关病毒或含其的试剂盒及其应用
PCT/CN2018/103937 WO2020000641A1 (zh) 2018-06-29 2018-09-04 编码人nadh脱氢酶亚单位蛋白的核酸及其应用
CN201811221305.XA CN111073899B (zh) 2018-10-19 2018-10-19 一种编码人nadh脱氢酶亚单位4蛋白的核酸及其应用
CN201811230856.2A CN111068071A (zh) 2018-10-22 2018-10-22 基因治疗Leber遗传学视神经病变
PCT/CN2018/113799 WO2020082417A1 (zh) 2018-10-22 2018-11-02 基因治疗Leber遗传性视神经病变
PCT/CN2018/118662 WO2020077756A1 (zh) 2018-10-19 2018-11-30 Nd4蛋白的编码序列及其应用
PCT/CN2019/070461 WO2020037938A1 (zh) 2018-08-20 2019-01-04 一种重组腺相关病毒或含其的试剂盒及其应用
PCT/CN2019/094136 WO2020001657A1 (en) 2018-06-29 2019-07-01 Compositions and methods for treating leber's hereditary optic neuropathy

Publications (1)

Publication Number Publication Date
MX2020013772A true MX2020013772A (es) 2021-05-27

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MX2020013772A MX2020013772A (es) 2018-06-29 2019-07-01 Composiciones y métodos para el tratamiento de la neuropatía óptica hereditaria de leber.

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US (3) US11034954B2 (es)
EP (1) EP3814492A4 (es)
JP (2) JP2021529001A (es)
KR (2) KR102627561B1 (es)
AU (3) AU2019296451B2 (es)
BR (1) BR112020026361A2 (es)
CA (1) CA3103740A1 (es)
MX (1) MX2020013772A (es)
SG (1) SG11202012044QA (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11202012044QA (en) 2018-06-29 2021-01-28 Wuhan Neurophth Biological Tech Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
CA3109432A1 (en) * 2018-08-20 2020-02-27 Wuhan Neurophth Biotechnology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
CN113025633A (zh) 2019-12-09 2021-06-25 武汉纽福斯生物科技有限公司 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用

Family Cites Families (49)

* 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
JP2876058B2 (ja) 1986-08-18 1999-03-31 エミスフィア・テクノロジーズ・インコーポレイテッド 薬物送達システム
US5075109A (en) 1986-10-24 1991-12-24 Southern Research Institute Method of potentiating an immune response
US6024983A (en) 1986-10-24 2000-02-15 Southern Research Institute Composition for delivering bioactive agents for immune response and its preparation
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
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
US20010006948A1 (en) * 1998-11-25 2001-07-05 James D. Kang Gene transfer to intervertebral disc cells
JP2004501608A (ja) 2000-03-21 2004-01-22 キュラゲン　コーポレイション Ｖｅｇｆ調節遺伝子及びそれらを利用する方法
CA2442670A1 (en) 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
US20040072774A1 (en) * 2002-02-23 2004-04-15 Giovanni Manfredi Methods for expressing and targeting mitochondrial-DNA-encoded peptides and uses thereof
US7405284B2 (en) * 2002-10-18 2008-07-29 University Of Florida Research Foundation, Inc. Reducing cellular dysfunction caused by mitochondrial gene mutations
ES2647477T3 (es) * 2004-06-01 2017-12-21 Genzyme Corporation Composiciones y métodos para prevenir la agregación del vector AAV
US20130022979A1 (en) 2005-04-18 2013-01-24 Genesis Genomics Inc. 3.4kb MITOCHONDRIAL DNA DELETION FOR USE IN THE DETECTION OF CANCER
WO2006117250A2 (en) 2005-05-03 2006-11-09 Institut National De La Sante Et De La Recherche Medicale (Inserm) Importation of mitochondrial protein by an enhanced allotopic approach
EP2089412A4 (en) 2006-10-23 2010-01-27 John Guy SUPPRESSION OF OXIDATIVE STRESS OF MITOCHONDRIEN
US20100022517A1 (en) * 2006-12-18 2010-01-28 Richards Lori A Ophthalmic formulation of rho kinase inhibitor compound
JP2010538244A (ja) 2007-06-20 2010-12-09 ガラパゴス・ナムローゼ・フェンノートシャップ 骨及び関節の変性疾患の治療に有用な分子標的及び化合物並びにその同定方法
DK2350279T3 (en) 2008-10-22 2016-02-15 Quark Pharmaceuticals Inc METHODS OF TREATING EYE DISEASES
BR112012018899A2 (pt) 2010-01-28 2015-09-15 Philadelphia Children Hospital "método para purificar partículas de vetor de vírus adeno-associado."
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
WO2013151669A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of cytoplasmic and cytoskeletal 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
US9066966B2 (en) * 2013-02-01 2015-06-30 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
US9567376B2 (en) * 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
EP2992892A1 (en) * 2014-09-05 2016-03-09 Universität zu Köln Fusion protein for use in the treatment of mitochondrial diseases
JP2017530968A (ja) 2014-09-17 2017-10-19 ユニバーシティー オブ アイオワ リサーチ ファンデーション 免疫調整剤およびワクチン構成要素としてのウイルスｒｎａセグメント
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
JP2018521000A (ja) * 2015-05-29 2018-08-02 シドネキシス，インク． Ｄ２ｏで安定化された医薬製剤
WO2017011519A1 (en) 2015-07-13 2017-01-19 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
WO2018102672A1 (en) 2016-12-04 2018-06-07 Alavi Khorassani Moghadam Marcel Victor Methods for treating diseases related to mitochondrial stress
US20180207293A1 (en) 2017-01-25 2018-07-26 2C Tech Corp. Nanoparticles for sustained ophthalmic drug delivery and methods of use
KR20230093072A (ko) * 2017-03-01 2023-06-26 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 안구 장애에 대한 유전자 치료
AU2018314280B2 (en) 2017-08-11 2023-07-20 Rsem, Société En Commandite Treatment of ophthalmic conditions such as macular degeneration, glaucoma, and diabetic retinopathy using pharmaceutical agents that eliminate senescent cells
WO2019204304A2 (en) 2018-04-16 2019-10-24 The Children's Hospital Of Philadelphia Mitochondrial rna import for treating mitochondrial disease
WO2019241206A1 (en) 2018-06-11 2019-12-19 Gensight Biologics Sa Recombinant aav vectors and methods of using the same
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
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
SG11202012044QA (en) 2018-06-29 2021-01-28 Wuhan Neurophth Biological Tech 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
CA3109432A1 (en) 2018-08-20 2020-02-27 Wuhan Neurophth Biotechnology Limited 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
CN113025633A (zh) 2019-12-09 2021-06-25 武汉纽福斯生物科技有限公司 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用

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