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CY1123947T1 - Βελτιστοποιημενος υποκινητης rpe65 και βελτιστοποιημενες κωδικοποιητικες αλληλουχιες - Google Patents

Βελτιστοποιημενος υποκινητης rpe65 και βελτιστοποιημενες κωδικοποιητικες αλληλουχιες

Info

Publication number
CY1123947T1
CY1123947T1 CY20201101129T CY201101129T CY1123947T1 CY 1123947 T1 CY1123947 T1 CY 1123947T1 CY 20201101129 T CY20201101129 T CY 20201101129T CY 201101129 T CY201101129 T CY 201101129T CY 1123947 T1 CY1123947 T1 CY 1123947T1
Authority
CY
Cyprus
Prior art keywords
optimized
submotor
rpe65
coding sequences
optimized coding
Prior art date
Application number
CY20201101129T
Other languages
English (en)
Inventor
Alexander Smith
Robin Ali
Original Assignee
Ucl Business Ltd
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Ucl Business Ltd filed Critical Ucl Business Ltd
Publication of CY1123947T1 publication Critical patent/CY1123947T1/el

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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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
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    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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  • Cardiology (AREA)
  • Urology & Nephrology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Ophthalmology & Optometry (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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Abstract

Η παρούσα εφεύρεση σχετίζεται με την πρόληψη και/ή τη θεραπευτική αντιμετώπιση της δυστροφίας του αμφιβληστροειδούς σε ένα ασθενή, συμπεριλαμβανομένης της συγγενούς, αμαύρωσης του Leber.
CY20201101129T 2015-02-09 2020-11-26 Βελτιστοποιημενος υποκινητης rpe65 και βελτιστοποιημενες κωδικοποιητικες αλληλουχιες CY1123947T1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
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 (1)

Publication Number Publication Date
CY1123947T1 true CY1123947T1 (el) 2022-03-24

Family

ID=52746363

Family Applications (1)

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CY20201101129T CY1123947T1 (el) 2015-02-09 2020-11-26 Βελτιστοποιημενος υποκινητης rpe65 και βελτιστοποιημενες κωδικοποιητικες αλληλουχιες

Country Status (24)

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

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* 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
NZ748179A (en) * 2016-04-28 2025-08-29 Spark Therapeutics Inc Relative potency assay for viral vector encoding isomerohydrolases
AU2018376259A1 (en) 2017-11-30 2020-06-11 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
MX2021009305A (es) 2019-02-04 2021-11-04 Freeline Therapeutics Ltd Polinucleotidos.
TW202124722A (zh) * 2019-09-18 2021-07-01 美商英特佳樂帝克醫療公司 合成dna載體及其使用方法
GB202002202D0 (en) 2020-02-18 2020-04-01 Ucl Business Plc Gene therapy
EP4185335A1 (en) * 2020-07-21 2023-05-31 Frontera Therapeutics, Inc. Compositions and methods for the treatment of ocular diseases
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
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
EP4646212A1 (en) * 2023-01-05 2025-11-12 Opus Genetics, Inc. Gene therapy for ocular disease
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

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JP4819683B2 (ja) * 2004-08-06 2011-11-24 一般財団法人化学及血清療法研究所 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
HUE044089T2 (hu) 2011-07-18 2019-09-30 Inst Res Biomedicine Neutralizáló anti-influenza A antitestek és alkalmazásaik
US9163259B2 (en) * 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
TWI775096B (zh) 2012-05-15 2022-08-21 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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IL287142B1 (en) 2023-07-01
SG11201706520UA (en) 2017-09-28
SMT202000655T1 (it) 2021-01-05
IL253915B (en) 2021-10-31
US20180021458A1 (en) 2018-01-25
US20200179535A1 (en) 2020-06-11
CN107429252B (zh) 2022-03-29
MX2021006762A (es) 2021-07-15
MX383316B (es) 2025-03-13
EP3256169A1 (en) 2017-12-20
SI3256169T1 (sl) 2021-01-29
MX2017010292A (es) 2018-03-23
IL253915A0 (en) 2017-10-31
IL287142B2 (en) 2023-11-01
WO2016128722A1 (en) 2016-08-18
JP2018510620A (ja) 2018-04-19
EP3256169B1 (en) 2020-09-02
GB201502137D0 (en) 2015-03-25
AU2016217654B2 (en) 2021-12-09
CN107429252A (zh) 2017-12-01
HUE052407T2 (hu) 2021-04-28
IL287142A (en) 2021-12-01
NZ735329A (en) 2024-08-30
ES2830030T3 (es) 2021-06-02
US10568973B2 (en) 2020-02-25
PH12017501430A1 (en) 2018-01-15
AU2016217654A1 (en) 2017-09-28
JP6963503B2 (ja) 2021-11-10
LT3256169T (lt) 2021-01-11
DK3256169T3 (da) 2020-11-23
BR112017017060A2 (pt) 2018-04-10
CA2975850C (en) 2024-06-18
CA2975850A1 (en) 2016-08-18
EP3769790A1 (en) 2021-01-27
US20240293580A1 (en) 2024-09-05
RS61079B1 (sr) 2020-12-31
PT3256169T (pt) 2020-11-04
HK1248525A1 (zh) 2018-10-19
US11969478B2 (en) 2024-04-30
PL3256169T3 (pl) 2021-05-04
HRP20201831T1 (hr) 2021-01-08

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