JP2018510620A5 - - Google Patents

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Publication number
JP2018510620A5
JP2018510620A5 JP2017541749A JP2017541749A JP2018510620A5 JP 2018510620 A5 JP2018510620 A5 JP 2018510620A5 JP 2017541749 A JP2017541749 A JP 2017541749A JP 2017541749 A JP2017541749 A JP 2017541749A JP 2018510620 A5 JP2018510620 A5 JP 2018510620A5
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JP
Japan
Prior art keywords
sequence
rpe
vector
seq
specific promoter
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JP2017541749A
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English (en)
Japanese (ja)
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JP6963503B2 (ja
JP2018510620A (ja
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Priority claimed from GBGB1502137.1A external-priority patent/GB201502137D0/en
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Publication of JP2018510620A5 publication Critical patent/JP2018510620A5/ja
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Publication of JP6963503B2 publication Critical patent/JP6963503B2/ja
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JP2017541749A 2015-02-09 2016-02-08 最適化されたrpe65プロモーター及びコード配列 Active JP6963503B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1502137.1A GB201502137D0 (en) 2015-02-09 2015-02-09 Treatment
GB1502137.1 2015-02-09
PCT/GB2016/050289 WO2016128722A1 (en) 2015-02-09 2016-02-08 Optimized rpe65 promoter and coding sequences

Publications (3)

Publication Number Publication Date
JP2018510620A JP2018510620A (ja) 2018-04-19
JP2018510620A5 true JP2018510620A5 (enExample) 2019-03-22
JP6963503B2 JP6963503B2 (ja) 2021-11-10

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ID=52746363

Family Applications (1)

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JP2017541749A Active JP6963503B2 (ja) 2015-02-09 2016-02-08 最適化されたrpe65プロモーター及びコード配列

Country Status (24)

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US (3) US10568973B2 (enExample)
EP (2) EP3256169B1 (enExample)
JP (1) JP6963503B2 (enExample)
CN (1) CN107429252B (enExample)
AU (1) AU2016217654B2 (enExample)
BR (1) BR112017017060A2 (enExample)
CA (1) CA2975850C (enExample)
CY (1) CY1123947T1 (enExample)
DK (1) DK3256169T3 (enExample)
ES (1) ES2830030T3 (enExample)
GB (1) GB201502137D0 (enExample)
HR (1) HRP20201831T1 (enExample)
HU (1) HUE052407T2 (enExample)
IL (2) IL287142B2 (enExample)
LT (1) LT3256169T (enExample)
MX (2) MX383316B (enExample)
PH (1) PH12017501430A1 (enExample)
PL (1) PL3256169T3 (enExample)
PT (1) PT3256169T (enExample)
RS (1) RS61079B1 (enExample)
SG (1) SG11201706520UA (enExample)
SI (1) SI3256169T1 (enExample)
SM (1) SMT202000655T1 (enExample)
WO (1) WO2016128722A1 (enExample)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201502137D0 (en) * 2015-02-09 2015-03-25 Ucl Business Plc Treatment
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
MX2018013205A (es) * 2016-04-28 2019-06-24 Spark Therapeutics Inc Ensayo de potencia relativa para un vector viral que codifica isomero hidrolasas.
WO2019106035A1 (en) * 2017-11-30 2019-06-06 Friedrich Miescher Institute For Biomedical Research Synpiii, a promoter for the specific expression of genes in retinal pigment epithelium
CN111118017B (zh) * 2018-11-01 2023-10-03 上海朗昇生物科技有限公司 治疗Leber先天性黑蒙症的载体及其应用
PT3850089T (pt) 2019-02-04 2024-01-24 Freeline Therapeutics Ltd Polinucleótidos
GB202002202D0 (en) 2020-02-18 2020-04-01 Ucl Business Plc Gene therapy
CN113952471A (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
EP4185335A1 (en) * 2020-07-21 2023-05-31 Frontera Therapeutics, Inc. Compositions and methods for the treatment of ocular diseases
CN111826378B (zh) * 2020-08-05 2022-05-27 武汉纽福斯生物科技有限公司 一种编码人受体酪氨酸激酶Mer的核苷酸序列及其应用
CN112522292B (zh) * 2020-10-29 2023-05-02 南京启真基因工程有限公司 一种用于构建先天性黑蒙症克隆猪核供体细胞的CRISPR/Cas9系统及其应用
EP4399314A4 (en) * 2021-09-06 2025-09-10 Huidagene Therapeutics Singapore Pte Ltd TREATMENT OF EYE DISEASES AND DISORDERS ASSOCIATED WITH RPE65
CN120882416A (zh) * 2023-01-05 2025-10-31 欧普斯遗传学股份有限公司 用于眼部疾病的基因疗法
CN117018235A (zh) * 2023-06-20 2023-11-10 浙江大学 一种可用于活体的可视化神经环路的方法及工具病毒载体
CN116898991A (zh) * 2023-06-20 2023-10-20 浙江大学 一种可用于活体的可视化的神经活动状态评估方法及工具病毒载体

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080064061A1 (en) 2004-08-06 2008-03-13 Juridical Foundation The Chemo-Sero-Therapeutic Research Institute Yeast Promoter
US7740836B2 (en) 2006-05-03 2010-06-22 Fondazione Telethon Methods and compositions for recovering or improving visual function
US20100081707A1 (en) * 2008-02-21 2010-04-01 Ali Robin R Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
LT3418300T (lt) 2011-07-18 2021-01-11 Institute For Research In Biomedicine Neutralizuojantys antikūnai prieš gripo virusą a ir jų panaudojimas
US9163259B2 (en) * 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
TWI698240B (zh) * 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
GB201502137D0 (en) * 2015-02-09 2015-03-25 Ucl Business Plc Treatment
GB202002073D0 (en) * 2020-02-14 2020-04-01 Ucl Business Ltd Horizontal cells
GB202002202D0 (en) * 2020-02-18 2020-04-01 Ucl Business Plc Gene therapy

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