CN113260704A - CYP4V2和RdCVF在制备药物中的用途 - Google Patents

CYP4V2和RdCVF在制备药物中的用途 Download PDF

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CN113260704A
CN113260704A CN202080001822.5A CN202080001822A CN113260704A CN 113260704 A CN113260704 A CN 113260704A CN 202080001822 A CN202080001822 A CN 202080001822A CN 113260704 A CN113260704 A CN 113260704A
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vector
polynucleotide encoding
cyp4v2
rdcvf
seq
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CN113260704B (zh
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杨丽萍
陈邵宏
贾睿璇
张天赋
张凡
曾巧莉
和赛超
史天永
郝丹丹
姜尚伟
裴红杰
王旭东
曾露颖
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Beijing Chinagene Tech Co ltd
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Beijing Chinagene Tech Co ltd
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Abstract

本申请涉及CYP4V2和RdCVF在制备药物中的用途，所述药物用于治疗、缓解和/或预防与视网膜色素上皮(RPE)萎缩相关的疾病或病症。本申请还涉及一种分离的核酸分子，其包含编码CYP4V2的多核苷酸以及编码RdCVF的多核苷酸。本申请还涉及所述分离的核酸分子编码的氨基酸序列、包含所述分离的核酸分子的载体、包含所述核酸或所述载体的细胞及其在制备用于治疗、缓解和/或预防与视网膜色素上皮(RPE)萎缩相关的疾病或病症的药物中的用途。

Description

PCT国内申请，说明书已公开。

Claims (77)

  1. PCT国内申请，权利要求书已公开。
CN202080001822.5A 2019-12-09 2020-07-16 CYP4V2和RdCVF在制备药物中的用途 Active CN113260704B (zh)

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CN201911255192X 2019-12-09
CN201911255192 2019-12-09
PCT/CN2020/102292 WO2021114662A1 (zh) 2019-12-09 2020-07-16 CYP4V2和RdCVF在制备药物中的用途

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US (2) US20230146121A1 (zh)
EP (2) EP4268895A3 (zh)
JP (2) JP2023506458A (zh)
CN (1) CN113260704B (zh)
WO (1) WO2021114662A1 (zh)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1529753A (zh) * 2001-04-06 2004-09-15 ��˹��ŵ�� 疾病相关蛋白质
CN109136266A (zh) * 2018-08-10 2019-01-04 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
WO2019025984A1 (en) * 2017-07-31 2019-02-07 Reflection Biotechnologies Limited CELLULAR MODELS AND THERAPIES FOR OCULAR DISEASES

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP4693244B2 (ja) * 1999-03-18 2011-06-01 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 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
JP5913984B2 (ja) * 2009-12-03 2016-05-11 タカラバイオ株式会社 多能性幹細胞の製造のための核酸
RU2664673C2 (ru) * 2011-10-27 2018-08-21 Веллстат Офтэлмикс Корпорэйшн Векторы, кодирующие фактор жизнеспособности колбочек, полученный из палочек
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
EP3233093B1 (en) * 2014-12-18 2022-06-29 Sorbonne Universite Transgenic rpe cells overexpressing otx2 for the treatment of retinal degeneration
WO2016185242A1 (en) * 2015-05-21 2016-11-24 INSERM (Institut National de la Santé et de la Recherche Médicale) Synergistic combination of neuronal viability factors and uses thereof
AU2017344059B2 (en) * 2016-10-11 2021-12-23 Pharma Cinq, Llc Fusion protein between short form rod-derived cone viability factor and a hydrophilic peptide
WO2018199287A1 (ja) * 2017-04-28 2018-11-01 国立大学法人京都大学 クリスタリン網膜症の処置および／または予防方法

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1529753A (zh) * 2001-04-06 2004-09-15 ��˹��ŵ�� 疾病相关蛋白质
WO2019025984A1 (en) * 2017-07-31 2019-02-07 Reflection Biotechnologies Limited CELLULAR MODELS AND THERAPIES FOR OCULAR DISEASES
CN109136266A (zh) * 2018-08-10 2019-01-04 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

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JP2023139317A (ja) 2023-10-03
US20240024433A1 (en) 2024-01-25
EP4074828A4 (en) 2023-12-27
EP4268895A2 (en) 2023-11-01
WO2021114662A1 (zh) 2021-06-17
CN113260704B (zh) 2022-06-28
EP4074828A1 (en) 2022-10-19
US20230146121A1 (en) 2023-05-11
EP4268895A3 (en) 2024-01-10
JP2023506458A (ja) 2023-02-16

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