JP2018510620A5 - - Google Patents

Download PDF

Info

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
Authority
JP
Japan
Prior art keywords
sequence
rpe
vector
seq
specific promoter
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2017541749A
Other languages
English (en)
Japanese (ja)
Other versions
JP6963503B2 (ja
JP2018510620A (ja
Filing date
Publication date
Priority claimed from GBGB1502137.1A external-priority patent/GB201502137D0/en
Application filed filed Critical
Publication of JP2018510620A publication Critical patent/JP2018510620A/ja
Publication of JP2018510620A5 publication Critical patent/JP2018510620A5/ja
Application granted granted Critical
Publication of JP6963503B2 publication Critical patent/JP6963503B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
GB1502137.1 2015-02-09
GBGB1502137.1A GB201502137D0 (en) 2015-02-09 2015-02-09 Treatment
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 (cg-RX-API-DMAC7.html) 2019-03-22
JP6963503B2 JP6963503B2 (ja) 2021-11-10

Family

ID=52746363

Family Applications (1)

Application Number Title Priority Date Filing Date
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)

Country Link
US (3) US10568973B2 (cg-RX-API-DMAC7.html)
EP (2) EP3769790A1 (cg-RX-API-DMAC7.html)
JP (1) JP6963503B2 (cg-RX-API-DMAC7.html)
CN (1) CN107429252B (cg-RX-API-DMAC7.html)
AU (1) AU2016217654B2 (cg-RX-API-DMAC7.html)
BR (1) BR112017017060A2 (cg-RX-API-DMAC7.html)
CA (1) CA2975850C (cg-RX-API-DMAC7.html)
CY (1) CY1123947T1 (cg-RX-API-DMAC7.html)
DK (1) DK3256169T3 (cg-RX-API-DMAC7.html)
ES (1) ES2830030T3 (cg-RX-API-DMAC7.html)
GB (1) GB201502137D0 (cg-RX-API-DMAC7.html)
HR (1) HRP20201831T1 (cg-RX-API-DMAC7.html)
HU (1) HUE052407T2 (cg-RX-API-DMAC7.html)
IL (2) IL287142B2 (cg-RX-API-DMAC7.html)
LT (1) LT3256169T (cg-RX-API-DMAC7.html)
MX (2) MX383316B (cg-RX-API-DMAC7.html)
PH (1) PH12017501430A1 (cg-RX-API-DMAC7.html)
PL (1) PL3256169T3 (cg-RX-API-DMAC7.html)
PT (1) PT3256169T (cg-RX-API-DMAC7.html)
RS (1) RS61079B1 (cg-RX-API-DMAC7.html)
SG (1) SG11201706520UA (cg-RX-API-DMAC7.html)
SI (1) SI3256169T1 (cg-RX-API-DMAC7.html)
SM (1) SMT202000655T1 (cg-RX-API-DMAC7.html)
WO (1) WO2016128722A1 (cg-RX-API-DMAC7.html)

Families Citing this family (16)

* 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
AU2017258776B2 (en) * 2016-04-28 2023-07-13 Spark Therapeutics, Inc. Relative potency assay for viral vector encoding isomerohydrolases
EP3717651A1 (en) * 2017-11-30 2020-10-07 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
JP7600124B2 (ja) 2019-02-04 2024-12-16 フリーライン セラピューティクス リミテッド ポリヌクレオチド
JP2022549138A (ja) * 2019-09-18 2022-11-24 インターガラクティック セラピューティクス インコーポレイテッド 合成dnaベクターおよびその使用法
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
CN116368228A (zh) * 2020-07-21 2023-06-30 方拓生物科技公司 用于治疗眼部疾病的组合物和方法
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
CA3231028A1 (en) * 2021-09-06 2023-03-09 Shaoran Wang Treatment of rpe65-associated eye diseases and disorders
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
WO2006013859A1 (ja) * 2004-08-06 2006-02-09 Juridical Foundation The Chemo-Sero-Therapeutic Research Institute 酵母プロモーター
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
ES2841899T3 (es) * 2011-07-18 2021-07-12 Inst Res Biomedicine Anticuerpos neutralizantes del virus de la influenza A y usos de los mismos
US9163259B2 (en) * 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
TWI702955B (zh) 2012-05-15 2020-09-01 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

Similar Documents

Publication Publication Date Title
JP2018510620A5 (cg-RX-API-DMAC7.html)
JP2022141670A5 (cg-RX-API-DMAC7.html)
JP6831779B2 (ja) 修飾された第ix因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用
JP2014512171A5 (cg-RX-API-DMAC7.html)
IL273261B2 (en) Adeno-associated virus variant capsids and methods of using them
PH12020550706A1 (en) Adeno-associated virus variant capsids and use for inhibiting angiogenesis
RU2018132517A (ru) Aav-idua вектор для лечения ассоциированной с mps i слепоты
RU2765826C2 (ru) Векторная система на основе аденоассоциированного вируса
RU2018119710A (ru) Кодирующие фактор viii варианты с пониженным содержанием cpg, композиции и способы, и применение в лечении нарушений гемостаза
JP2017510296A5 (cg-RX-API-DMAC7.html)
TW201629225A (zh) 第九因子基因療法
HRP20201831T1 (hr) Optimizirani promoter rpe65 i kodirajuće sekvence
EP3697448A1 (en) Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant aav9-derived vector
RU2019103488A (ru) Способы и композиции для лечения нарушений и заболеваний, связанных с rdh12
JP2019524090A5 (cg-RX-API-DMAC7.html)
WO2020039425A1 (en) Vectors expressing klotho for treating cancer
RU2019126509A (ru) Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
JP7289306B2 (ja) 網膜障害を治療するための組成物及び方法
JP2020533968A (ja) 組換えアデノ随伴ベクター
JP2020527167A5 (cg-RX-API-DMAC7.html)
JP2018509154A5 (cg-RX-API-DMAC7.html)
CN112029773B (zh) 编码bdnf的核酸及其应用
JP2020510433A5 (cg-RX-API-DMAC7.html)
RU2019130004A (ru) Генная терапия при глазных заболеваниях
AU2018395121B2 (en) Adeno-associated virus (AAV) vector having hybrid HGF gene introduced thereto