RU2734678C2 - Генетическая конструкция - Google Patents

Генетическая конструкция Download PDF

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RU2734678C2
RU2734678C2 RU2018115760A RU2018115760A RU2734678C2 RU 2734678 C2 RU2734678 C2 RU 2734678C2 RU 2018115760 A RU2018115760 A RU 2018115760A RU 2018115760 A RU2018115760 A RU 2018115760A RU 2734678 C2 RU2734678 C2 RU 2734678C2
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seq
sequence
leu
recombinant vector
gly
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RU2018115760A
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RU2018115760A3 (OSRAM
RU2018115760A (ru
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Питер ВИДДОУСОН
Кейт МАРТИН
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Кетера Лимитед
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RU2018115760A 2015-10-26 2016-10-25 Генетическая конструкция RU2734678C2 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1518911.1 2015-10-26
GB1518911.1A GB2547179A (en) 2015-10-26 2015-10-26 Genetic construct
PCT/GB2016/053319 WO2017072498A1 (en) 2015-10-26 2016-10-25 Genetic construct

Publications (3)

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RU2018115760A RU2018115760A (ru) 2019-11-28
RU2018115760A3 RU2018115760A3 (OSRAM) 2020-03-19
RU2734678C2 true RU2734678C2 (ru) 2020-10-21

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US (2) US10342880B2 (OSRAM)
EP (2) EP3985119A1 (OSRAM)
JP (2) JP7073264B2 (OSRAM)
KR (1) KR102819739B1 (OSRAM)
CN (1) CN108350467B (OSRAM)
AU (1) AU2016344508B2 (OSRAM)
CY (1) CY1124588T1 (OSRAM)
DK (1) DK3368678T3 (OSRAM)
ES (1) ES2886009T3 (OSRAM)
GB (1) GB2547179A (OSRAM)
HR (1) HRP20211407T1 (OSRAM)
HU (1) HUE056911T2 (OSRAM)
LT (1) LT3368678T (OSRAM)
MX (1) MX2018005286A (OSRAM)
PL (1) PL3368678T3 (OSRAM)
PT (1) PT3368678T (OSRAM)
RS (1) RS62460B1 (OSRAM)
RU (1) RU2734678C2 (OSRAM)
SI (1) SI3368678T1 (OSRAM)
SM (1) SMT202100520T1 (OSRAM)
WO (1) WO2017072498A1 (OSRAM)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2825840C2 (ru) * 2019-04-15 2024-09-02 Лоджикбайо Терапьютикс, Инк. Мониторинг генной терапии

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017058926A1 (en) 2015-10-01 2017-04-06 Goleini Inc. Targeted expression of chloride channels and methods of use thereof
GB201705484D0 (en) * 2017-04-05 2017-05-17 Quethera Ltd Genetic construct
CN111372612A (zh) * 2017-09-27 2020-07-03 西吉隆医疗股份有限公司 包含活性细胞的方法、组合物和可植入元件
IL321310A (en) 2017-10-20 2025-08-01 Res Inst Nationwide Childrens Hospital Methods and materials for NT-3 gene therapy
EP3720460A1 (en) * 2017-12-05 2020-10-14 University of Washington Compositions and methods for enhancing functional expression of therapeutic genes in photoreceptors
CN108250288A (zh) * 2018-01-17 2018-07-06 吉林省农业科学院 一种叶绿体表达的脑源性神经营养因子hBDNFb蛋白及其制备方法
CN109022487A (zh) * 2018-08-15 2018-12-18 上海市第人民医院 一种表达p65的基因载体及其用于治疗视网膜神经节细胞变性的基因治疗药物
WO2020077295A1 (en) * 2018-10-11 2020-04-16 Decibel Therapeutics, Inc. Aav1 vectors and uses thereof for treatment of otic indications
SG11202105267VA (en) * 2018-12-19 2021-07-29 Versameb Ag Rna encoding a protein
CN113557301B (zh) * 2019-03-04 2025-03-07 公益财团法人东京都医学综合研究所 编码Trk片段的核酸构建体及其利用
MA55223A (fr) * 2019-03-06 2022-01-12 Generation Bio Co Adn à extrémité fermée (cedna) et composés de modulation immunitaire
US20230123802A1 (en) * 2019-03-27 2023-04-20 Sigilon Therapeutics, Inc. Compositions, devices, and methods for factor vii therapy
US20230038502A1 (en) 2019-06-11 2023-02-09 Shire Human Genetic Therapies, Inc. Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein
EP3999647A1 (en) * 2019-07-18 2022-05-25 Lysogene Compositions and methods for the treatment of sanfilippo disease and other disorders
RU2719013C1 (ru) * 2019-09-25 2020-04-16 Федеральное государственное бюджетное образовательное учреждение высшего образования "Московский государственный университет имени М.В. Ломоносова" (МГУ) Генно-инженерная конструкция для стимуляции посттравматической регенерации периферических нервов
WO2021067448A1 (en) 2019-09-30 2021-04-08 Applied Genetic Technologies Corporation Adeno-associated virus (aav) systems for treatment of genetic hearing loss
EP4048800A1 (en) 2019-10-23 2022-08-31 Shire Human Genetic Therapies, Inc. Adeno-associated virus vectors based gene therapy for hereditary angioedema
US20220186252A1 (en) 2020-12-16 2022-06-16 Takeda Pharmaceutical Company Limited Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein
MX2023009922A (es) * 2021-02-26 2023-10-25 Icahn School Med Mount Sinai Método para reducir la degeneración de células ganglionares retinianas.
CN116179495B (zh) * 2022-11-28 2025-06-06 上海恩凯细胞技术有限公司 转基因免疫细胞及其应用
CN118256562A (zh) * 2022-12-26 2024-06-28 科辉智药(深圳)新药研究中心有限公司 一种用于中枢神经系统疾病治疗的基因序列构建体及其应用
WO2024161142A1 (en) 2023-02-02 2024-08-08 Quethera Limited Recombinant adeno-associated virus vector
CN118534135A (zh) * 2024-06-07 2024-08-23 中国人民解放军联勤保障部队第九二四医院 一种中枢性肥胖抵抗标志物及其检测方法和应用

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999053959A2 (en) * 1998-04-20 1999-10-28 Koninklijke Nederlandse Akademie Van Wetenschappen Use of viral vectors for treatment of the injured peripheral and central nervous system
RU2288742C2 (ru) * 2000-12-19 2006-12-10 Рисерч Дивелопмент Фаундейшн Перенос генов, опосредованный лентивирусным вектором, и их использование
WO2009048605A1 (en) * 2007-10-11 2009-04-16 Biogen Idec Ma Inc. Methods for treating pressure induced optic neuropathy, preventing neuronal degeneration and promoting neuronal cell, survival via administration of lingo-1 antagonists and trkb agonists
WO2009120978A2 (en) * 2008-03-27 2009-10-01 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor
RU2515904C2 (ru) * 2006-09-28 2014-05-20 Пьер Фабр Медикамент Способ генерирования активных антител к антигену устойчивости, антитела, полученные этим способом, и их применения

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5229500A (en) 1989-08-30 1993-07-20 Regeneron Pharmaceuticals, Inc. Brain derived neurotrophic factor
US6225282B1 (en) * 1996-01-05 2001-05-01 Genentech, Inc. Treatment of hearing impairments
ATE258804T1 (de) * 1996-10-16 2004-02-15 Regeneron Pharma Verfahren zur verabreichung von wachstumsfaktoren
US7060429B2 (en) * 2001-02-22 2006-06-13 University Of Maryland, Baltimore Treatment of neurodegenerative diseases by altering levels of TrkB isoforms and/or TrkC isoforms
CN103614416B (zh) * 2013-09-30 2016-09-07 中国人民解放军第二军医大学东方肝胆外科医院 一种携带人穿膜肽p53与GM-CSF基因的重组溶瘤腺病毒及其用途

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999053959A2 (en) * 1998-04-20 1999-10-28 Koninklijke Nederlandse Akademie Van Wetenschappen Use of viral vectors for treatment of the injured peripheral and central nervous system
RU2288742C2 (ru) * 2000-12-19 2006-12-10 Рисерч Дивелопмент Фаундейшн Перенос генов, опосредованный лентивирусным вектором, и их использование
RU2515904C2 (ru) * 2006-09-28 2014-05-20 Пьер Фабр Медикамент Способ генерирования активных антител к антигену устойчивости, антитела, полученные этим способом, и их применения
WO2009048605A1 (en) * 2007-10-11 2009-04-16 Biogen Idec Ma Inc. Methods for treating pressure induced optic neuropathy, preventing neuronal degeneration and promoting neuronal cell, survival via administration of lingo-1 antagonists and trkb agonists
WO2009120978A2 (en) * 2008-03-27 2009-10-01 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2825840C2 (ru) * 2019-04-15 2024-09-02 Лоджикбайо Терапьютикс, Инк. Мониторинг генной терапии

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