JP6293664B2 - 桿体由来錐体生存因子をコードするベクター - Google Patents

桿体由来錐体生存因子をコードするベクター Download PDF

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JP6293664B2
JP6293664B2 JP2014539038A JP2014539038A JP6293664B2 JP 6293664 B2 JP6293664 B2 JP 6293664B2 JP 2014539038 A JP2014539038 A JP 2014539038A JP 2014539038 A JP2014539038 A JP 2014539038A JP 6293664 B2 JP6293664 B2 JP 6293664B2
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cells
nucleic acid
rdcvf
protein
cell
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JP2015501156A (ja
JP2015501156A5 (enExample
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ティアンシー ルオ,
ティアンシー ルオ,
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ウェルスタット オフサルミクス コーポレイション
ウェルスタット オフサルミクス コーポレイション
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    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • 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)
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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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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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • 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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    • C12N2830/42—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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JP2014539038A 2011-10-27 2012-10-26 桿体由来錐体生存因子をコードするベクター Expired - Fee Related JP6293664B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161552155P 2011-10-27 2011-10-27
US61/552,155 2011-10-27
PCT/US2012/062106 WO2013063383A2 (en) 2011-10-27 2012-10-26 Vectors encoding rod-derived cone viability factor

Publications (3)

Publication Number Publication Date
JP2015501156A JP2015501156A (ja) 2015-01-15
JP2015501156A5 JP2015501156A5 (enExample) 2015-12-17
JP6293664B2 true JP6293664B2 (ja) 2018-03-14

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US (2) US9265813B2 (enExample)
EP (1) EP2797613B1 (enExample)
JP (1) JP6293664B2 (enExample)
KR (1) KR102000141B1 (enExample)
CN (2) CN110777124A (enExample)
AU (1) AU2012321102C1 (enExample)
BR (1) BR112014010091B1 (enExample)
CA (1) CA2853379C (enExample)
DK (1) DK2797613T3 (enExample)
ES (1) ES2774779T3 (enExample)
MX (1) MX354516B (enExample)
PL (1) PL2797613T3 (enExample)
RU (1) RU2664673C2 (enExample)
WO (1) WO2013063383A2 (enExample)

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US10004780B2 (en) 2012-10-17 2018-06-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for the treatment of age-related macular degeneration (AMD)
ES2744565T3 (es) 2014-04-25 2020-02-25 Genethon Tratamiento de la hiperbilirrubinemia
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
GB201516066D0 (en) 2015-09-10 2015-10-28 Young & Co Llp D Treatment of retinitis pigmentosa
AU2016370590B2 (en) 2015-12-14 2023-11-02 The Trustees Of The University Of Pennsylvania Composition for treatment of Crigler-Najjar syndrome
WO2017120294A1 (en) * 2016-01-05 2017-07-13 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
KR20260085174A (ko) * 2016-10-11 2026-06-08 파마 싱크, 엘엘씨 짧은 형태의 간상체 유래 원추체 생존능 인자와 친수성 펩타이드 사이의 융합 단백질
AU2018350700B2 (en) * 2017-10-16 2025-04-24 VeonGen Therapeutics GmbH AAV vectors
WO2019122403A1 (en) * 2017-12-22 2019-06-27 Sparingvision Constructs comprising neuronal viability factors and uses thereof
WO2019149648A1 (en) * 2018-01-30 2019-08-08 Universiteit Gent Compositions for use to treat cataract
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
KR20250058061A (ko) * 2018-09-07 2025-04-29 유니버시티 오브 써던 캘리포니아 세포 시딩된 기질로부터의 치료적 상층액 및 관련 방법
KR20260086827A (ko) * 2018-11-14 2026-06-10 리젠엑스바이오 인크. 신경원성 세로이드 리포푸신증에 대한 유전자 요법
JP2022514863A (ja) * 2018-12-19 2022-02-16 ヴェルサメブ アーゲー タンパク質をコードするrna
CN114651003A (zh) 2019-09-10 2022-06-21 黑曜石疗法公司 用于可调调节的ca2-il15融合蛋白
JP7495756B2 (ja) * 2019-12-09 2024-06-05 北京中因科技有限公司 医薬の製造におけるCYP4V2およびRdCVFの使用
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
IL300738A (en) * 2020-08-28 2023-04-01 Biocad Joint Stock Co A vaccine for SARS-COV-2 based on AAV5
CN121285568A (zh) * 2023-03-30 2026-01-06 制药五有限责任公司 编码视杆细胞源性视锥细胞活力因子和人IgK信号序列的载体
WO2026072752A1 (en) 2024-09-27 2026-04-02 Pharma Cinq, Llc Rod-derived cone viability factor fusion protein

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US9265813B2 (en) 2016-02-23
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US20160108099A1 (en) 2016-04-21
CN104321069A (zh) 2015-01-28
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DK2797613T3 (da) 2020-03-02
US20140328821A1 (en) 2014-11-06
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AU2012321102C1 (en) 2016-11-10
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RU2664673C2 (ru) 2018-08-21
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WO2013063383A3 (en) 2013-06-20
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US10040835B2 (en) 2018-08-07

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