CN116113702A - 一种融合蛋白及其用途 - Google Patents

一种融合蛋白及其用途 Download PDF

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CN116113702A
CN116113702A CN202180058367.7A CN202180058367A CN116113702A CN 116113702 A CN116113702 A CN 116113702A CN 202180058367 A CN202180058367 A CN 202180058367A CN 116113702 A CN116113702 A CN 116113702A
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polynucleotide encoding
nucleic acid
rdcvf
acid molecule
polynucleotide
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陈邵宏
史天永
郝丹丹
和赛超
牛琳琳
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Beijing Chinagene Tech Co ltd
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Abstract

本申请涉及一种融合蛋白及其应用。所述融合蛋白包含RdCVF和信号肽,其中所述信号肽为SEQ ID NO:27‑31,SEQ ID NO:33中任一项所示的氨基酸序列。本申请还涉及分离的核酸分子,其包含a)编码RdCVF的多核苷酸以及b)编码信号肽的多核苷酸。本申请还提供了包含该核酸分子的载体、细胞和药物组合物等,并提供了它们的制药用途。本申请所述的融合蛋白和/或分离的核酸分子可以有效地提高RdCVF的分泌量。

Description

PCT国内申请,说明书已公开。

Claims (93)

  1. PCT国内申请,权利要求书已公开。
CN202180058367.7A 2020-08-07 2021-01-19 一种融合蛋白及其用途 Pending CN116113702A (zh)

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CN202010790496.2A CN111733174B (zh) 2020-08-07 2020-08-07 一种分离的核酸分子及其用途
CN2020107904962 2020-08-07
PCT/CN2021/072743 WO2022027933A1 (zh) 2020-08-07 2021-01-19 一种融合蛋白及其用途

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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
CN113015804B (zh) * 2020-10-13 2022-05-17 北京中因科技有限公司 用于治疗结晶样视网膜变性的核酸分子及其用途
WO2023284873A1 (en) * 2021-07-15 2023-01-19 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating bietti crystalline dystrophy
CN114875010B (zh) * 2022-04-21 2023-10-24 深圳大学 一种提升蛋白翻译效率的基因及其编码产物与应用
US12065663B2 (en) * 2022-07-13 2024-08-20 Chigenovo Co., Ltd Vector and method for treating bietti's crystalline dystrophy

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FR2823221B1 (fr) * 2001-04-06 2004-04-02 Univ Pasteur Sequences associees a la degenerescence retinienne et applications
WO2007078922A2 (en) * 2005-12-30 2007-07-12 Neurotech Usa Inc. Micronized device for the delivery of biologically active molecules and methods of use thereof
AU2012321102C1 (en) * 2011-10-27 2016-11-10 Pharma Cinq, Llc Vectors encoding rod-derived cone viability factor
TWI688401B (zh) 2013-09-13 2020-03-21 美商安進公司 用於治療骨髓性白血病的表觀遺傳因子與靶向cd33及cd3之雙特異性化合物的組合
CN104745594B (zh) * 2013-12-31 2019-10-25 中国人民解放军第三军医大学第一附属医院 Cyp4v2基因突变体及其应用
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
FI3445773T3 (fi) * 2016-05-13 2023-03-30 4D Molecular Therapeutics Inc Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä
CA3025977A1 (en) * 2016-10-11 2018-04-19 Wellstat Ophthalmics Corporation Fusion protein between short form rod-derived cone viability factor and a hydrophilic peptide
JP7065852B2 (ja) * 2016-12-01 2022-05-12 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 網膜変性疾患の処置のための医薬組成物
US20200255859A1 (en) * 2017-07-31 2020-08-13 Reflection Biotechnologies Limited Cellular models of and therapies for ocular diseases
CN109136266B (zh) * 2018-08-10 2022-02-18 深圳泓熙生物科技发展有限公司 用于治疗或预防结晶样视网膜色素变性的基因载体及其用途
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

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US20230399367A1 (en) 2023-12-14
CN111733174A (zh) 2020-10-02
CN111733174B (zh) 2021-02-09
EP4194556A1 (en) 2023-06-14

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