CN116782886A - 密码子优化的rpgrorf15基因及其用途 - Google Patents

密码子优化的rpgrorf15基因及其用途 Download PDF

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Publication number
CN116782886A
CN116782886A CN202180072680.6A CN202180072680A CN116782886A CN 116782886 A CN116782886 A CN 116782886A CN 202180072680 A CN202180072680 A CN 202180072680A CN 116782886 A CN116782886 A CN 116782886A
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seq
sequence
nucleic acid
vector
nucleotide sequence
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Chinese (zh)
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D·H·基恩
M·A·科特尔曼
D·沙菲尔
P·弗朗西斯
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4D Molecular Therapeutics Inc
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4D Molecular Therapeutics Inc
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    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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CN202180072680.6A 2020-09-02 2021-08-30 密码子优化的rpgrorf15基因及其用途 Pending CN116782886A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063073843P 2020-09-02 2020-09-02
US63/073843 2020-09-02
PCT/US2021/048267 WO2022051232A1 (en) 2020-09-02 2021-08-30 Codon optimized rpgrorf 15 genes and uses thereof

Publications (1)

Publication Number Publication Date
CN116782886A true CN116782886A (zh) 2023-09-19

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US (3) US20230340042A1 (https=)
EP (1) EP4208156A4 (https=)
JP (1) JP2023539368A (https=)
KR (1) KR102825852B1 (https=)
CN (1) CN116782886A (https=)
AU (1) AU2021337530B2 (https=)
BR (1) BR112023003834A2 (https=)
CA (1) CA3191540A1 (https=)
IL (1) IL300785B2 (https=)
WO (1) WO2022051232A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL300785B2 (en) * 2020-09-02 2025-04-01 4D Molecular Therapeutics Inc RPGRORF 15-codon-specific genes and their uses
CN117165596A (zh) * 2022-05-27 2023-12-05 武汉纽福斯生物科技有限公司 编码rpgr的核酸及其应用
EP4696540A1 (en) 2023-05-11 2026-02-18 LG Energy Solution, Ltd. Computing system for providing energy management service connected with autonomous driving, operation method of computing system and vehicle
CN121693566A (zh) * 2023-07-28 2026-03-17 瑞泽恩制药公司 使用bGH-SV40L串联PolyA在单向基因插入期间增强转基因表达

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015160893A1 (en) * 2014-04-15 2015-10-22 Applied Genetic Technologies Corporation Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (rpgr)
WO2018167510A1 (en) * 2017-03-16 2018-09-20 Nightstarx Limited Treatment of retinitis pigmentosa
CN108699118A (zh) * 2015-09-10 2018-10-23 牛津大学创新有限公司 色素性视网膜炎的治疗
US20190255192A1 (en) * 2016-05-13 2019-08-22 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
WO2020061574A1 (en) * 2018-09-21 2020-03-26 Nightstarx Limited Compositions and methods for treating retinitis pigmentosa

Family Cites Families (5)

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Publication number Priority date Publication date Assignee Title
WO2003031569A2 (en) * 2001-10-10 2003-04-17 Centocor, Inc. Nucleic acid vaccines using tumor antigen encoding nucleic acids with cytokine adjuvant encoding nucleic acid
US9163259B2 (en) * 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
EP2872183B1 (en) * 2012-07-11 2018-09-26 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
CN113952472A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
IL300785B2 (en) * 2020-09-02 2025-04-01 4D Molecular Therapeutics Inc RPGRORF 15-codon-specific genes and their uses

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015160893A1 (en) * 2014-04-15 2015-10-22 Applied Genetic Technologies Corporation Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (rpgr)
CN108699118A (zh) * 2015-09-10 2018-10-23 牛津大学创新有限公司 色素性视网膜炎的治疗
US20190255192A1 (en) * 2016-05-13 2019-08-22 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
WO2018167510A1 (en) * 2017-03-16 2018-09-20 Nightstarx Limited Treatment of retinitis pigmentosa
WO2020061574A1 (en) * 2018-09-21 2020-03-26 Nightstarx Limited Compositions and methods for treating retinitis pigmentosa

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
蒋鹏飞 等: "视网膜色素变性的实验研究进展", 国际眼科杂志, no. 6, 8 June 2020 (2020-06-08), pages 52 - 55 *

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IL300785A (en) 2023-04-01
IL300785B2 (en) 2025-04-01
AU2021337530B2 (en) 2023-08-24
KR102825852B1 (ko) 2025-06-26
US20220290181A1 (en) 2022-09-15
EP4208156A1 (en) 2023-07-12
EP4208156A4 (en) 2025-01-15
US20220064673A1 (en) 2022-03-03
AU2021337530A1 (en) 2023-04-06
BR112023003834A2 (pt) 2023-03-28
IL300785B1 (en) 2024-12-01
JP2023539368A (ja) 2023-09-13
US11345930B2 (en) 2022-05-31
CA3191540A1 (en) 2022-03-10
KR20230061372A (ko) 2023-05-08
WO2022051232A1 (en) 2022-03-10
US20230340042A1 (en) 2023-10-26

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