DK2797613T3 - Vektorer, der koder for rod-derived cone viability-faktor - Google Patents

Vektorer, der koder for rod-derived cone viability-faktor Download PDF

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Publication number
DK2797613T3
DK2797613T3 DK12843778.7T DK12843778T DK2797613T3 DK 2797613 T3 DK2797613 T3 DK 2797613T3 DK 12843778 T DK12843778 T DK 12843778T DK 2797613 T3 DK2797613 T3 DK 2797613T3
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Prior art keywords
rod
vectors coding
viability factor
cone viability
derived cone
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DK12843778.7T
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English (en)
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Tianci Luo
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Wellstat Ophthalmics Corp
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44—Oxidoreductases (1)
    • A—HUMAN NECESSITIES
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    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0051—Oxidoreductases (1.) acting on a sulfur group of donors (1.8)
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

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DK12843778.7T 2011-10-27 2012-10-26 Vektorer, der koder for rod-derived cone viability-faktor DK2797613T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161552155P 2011-10-27 2011-10-27
PCT/US2012/062106 WO2013063383A2 (en) 2011-10-27 2012-10-26 Vectors encoding rod-derived cone viability factor

Publications (1)

Publication Number Publication Date
DK2797613T3 true DK2797613T3 (da) 2020-03-02

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DK12843778.7T DK2797613T3 (da) 2011-10-27 2012-10-26 Vektorer, der koder for rod-derived cone viability-faktor

Country Status (15)

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US (2) US9265813B2 (da)
EP (1) EP2797613B1 (da)
JP (1) JP6293664B2 (da)
KR (1) KR102000141B1 (da)
CN (2) CN104321069A (da)
AU (1) AU2012321102C1 (da)
BR (1) BR112014010091B1 (da)
CA (1) CA2853379C (da)
DK (1) DK2797613T3 (da)
ES (1) ES2774779T3 (da)
HK (1) HK1202049A1 (da)
MX (1) MX354516B (da)
PL (1) PL2797613T3 (da)
RU (1) RU2664673C2 (da)
WO (1) WO2013063383A2 (da)

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JP6684343B2 (ja) 2015-05-21 2020-04-22 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル ニューロン生存因子の相乗組み合わせ及びその使用
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JP7061067B2 (ja) 2015-12-14 2022-04-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア クリグラー・ナジャー症候群の処置のための組成物
US10857240B2 (en) 2016-01-05 2020-12-08 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of ocular disorders and blinding diseases
CN106148528B (zh) * 2016-07-11 2019-10-01 赵晨 一种遗传性Usher综合征的致病突变及其检测试剂
KR20230156440A (ko) * 2016-10-11 2023-11-14 웰스테이트 옵탈믹스 코퍼레이션 짧은 형태의 간상체 유래 원추체 생존능 인자와 친수성 펩타이드 사이의 융합 단백질
CN111742052B (zh) * 2017-12-22 2024-10-11 节约视觉公司 包含神经元活力因子的构建体及其用途
PL3746114T3 (pl) * 2018-01-30 2023-01-16 Universiteit Gent Kompozycje do zastosowania w leczeniu zaćmy
CN108504685A (zh) * 2018-03-27 2018-09-07 宜明细胞生物科技有限公司 一种利用CRISPR/Cas9系统同源重组修复IL-2RG缺陷基因的方法
MX2021005517A (es) * 2018-11-14 2021-06-18 Regenxbio Inc Terapia genica para lipofuscinosis neuronal ceroidea.
CN113454227A (zh) * 2018-12-19 2021-09-28 维萨梅布有限公司 编码蛋白质的rna
MX2022002747A (es) 2019-09-10 2022-04-06 Obsidian Therapeutics Inc Proteinas de fusion de ca2-il15 para regulacion ajustable.
CN113260704B (zh) * 2019-12-09 2022-06-28 北京中因科技有限公司 CYP4V2和RdCVF在制备药物中的用途
CN111733174B (zh) * 2020-08-07 2021-02-09 北京大学第三医院（北京大学第三临床医学院） 一种分离的核酸分子及其用途
US20240342313A1 (en) 2023-03-30 2024-10-17 Pharma Cinq, Llc Vector encoding rod-derived cone viability factor and human igk signal sequence

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RU2014121256A (ru) 2015-12-10
JP2015501156A (ja) 2015-01-15
AU2012321102B2 (en) 2016-07-14
PL2797613T3 (pl) 2020-06-15
AU2012321102A1 (en) 2013-05-16
MX2014004949A (es) 2014-09-12
CN104321069A (zh) 2015-01-28
EP2797613A4 (en) 2015-09-09
KR102000141B1 (ko) 2019-07-15
JP6293664B2 (ja) 2018-03-14
US20160108099A1 (en) 2016-04-21
ES2774779T3 (es) 2020-07-22
RU2664673C2 (ru) 2018-08-21
EP2797613B1 (en) 2019-12-04
MX354516B (es) 2018-03-08
CN110777124A (zh) 2020-02-11
US9265813B2 (en) 2016-02-23
CA2853379C (en) 2020-11-24
CA2853379A1 (en) 2013-05-02
WO2013063383A3 (en) 2013-06-20
BR112014010091B1 (pt) 2022-11-08
KR20140092320A (ko) 2014-07-23
HK1202049A1 (en) 2015-09-18
EP2797613A2 (en) 2014-11-05
US20140328821A1 (en) 2014-11-06
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AU2012321102C1 (en) 2016-11-10
BR112014010091A2 (pt) 2020-10-27

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