IL287142B2 - Optimized RPE65 promoter and coding sequences - Google Patents
Optimized RPE65 promoter and coding sequencesInfo
- Publication number
- IL287142B2 IL287142B2 IL287142A IL28714221A IL287142B2 IL 287142 B2 IL287142 B2 IL 287142B2 IL 287142 A IL287142 A IL 287142A IL 28714221 A IL28714221 A IL 28714221A IL 287142 B2 IL287142 B2 IL 287142B2
- Authority
- IL
- Israel
- Prior art keywords
- sequence
- rpe65
- aav
- seq
- vector
- Prior art date
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/18—Carboxylic ester hydrolases (3.1.1)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
- A61P27/02—Ophthalmic agents
-
- 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
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/861—Adenoviral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/01—Carboxylic ester hydrolases (3.1.1)
- C12Y301/01064—Retinoid isomerohydrolase (3.1.1.64)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
- C12N2015/8518—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic expressing industrially exogenous proteins, e.g. for pharmaceutical use, human insulin, blood factors, immunoglobulins, pseudoparticles
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Microbiology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Dermatology (AREA)
- Vascular Medicine (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Ophthalmology & Optometry (AREA)
- Virology (AREA)
- Urology & Nephrology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Eye Examination Apparatus (AREA)
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 (3)
| Publication Number | Publication Date |
|---|---|
| IL287142A IL287142A (en) | 2021-12-01 |
| IL287142B1 IL287142B1 (en) | 2023-07-01 |
| IL287142B2 true IL287142B2 (en) | 2023-11-01 |
Family
ID=52746363
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL287142A IL287142B2 (en) | 2015-02-09 | 2016-02-08 | Optimized RPE65 promoter and coding sequences |
| IL253915A IL253915B (en) | 2015-02-09 | 2017-08-08 | Optimized rpe65 promoter and coding sequences |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL253915A IL253915B (en) | 2015-02-09 | 2017-08-08 | Optimized rpe65 promoter and coding sequences |
Country Status (24)
| Country | Link |
|---|---|
| US (3) | US10568973B2 (enExample) |
| EP (2) | EP3769790A1 (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 (16)
| 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 |
| EP3448371A4 (en) * | 2016-04-28 | 2019-09-11 | Spark Therapeutics, Inc. | TESTING THE RELATIVE EFFECTIVENESS OF ISOMERHYDROLASES FOR THE CODING OF VIRAL VECTORS |
| CN111542611A (zh) * | 2017-11-30 | 2020-08-14 | 弗里德里克·米谢尔生物医学研究所 | 用于使基因在视网膜色素上皮中特异性表达的启动子SynPIII |
| 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 |
| ES2968922T3 (es) | 2019-02-04 | 2024-05-14 | Freeline Therapeutics Ltd | Polinucleótidos |
| MX2022003291A (es) * | 2019-09-18 | 2022-09-09 | Intergalactic Therapeutics Inc | Vectores de adn sinteticos y metodos de uso. |
| 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 |
| WO2022017363A1 (en) * | 2020-07-21 | 2022-01-27 | Inspirar Limited | 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 |
| WO2024148175A1 (en) * | 2023-01-05 | 2024-07-11 | Opus Genetics Inc. | Gene therapy for ocular disease |
| 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 |
| 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 |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009105690A2 (en) * | 2008-02-21 | 2009-08-27 | Targeted Genetics Corporation | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| WO2013173129A2 (en) * | 2012-05-15 | 2013-11-21 | Avalanche Australia Pty Ltd. | Treatment of amd using aav sflt-1 |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101035895A (zh) | 2004-08-06 | 2007-09-12 | 财团法人化学及血清疗法研究所 | 酵母启动子 |
| US7740836B2 (en) | 2006-05-03 | 2010-06-22 | Fondazione Telethon | Methods and compositions for recovering or improving visual function |
| CA2841551C (en) * | 2011-07-18 | 2020-07-28 | Institute For Research In Biomedicine | Neutralizing anti-influenza a virus antibodies and uses thereof |
| US9163259B2 (en) | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
| 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 |
-
2015
- 2015-02-09 GB GBGB1502137.1A patent/GB201502137D0/en not_active Ceased
-
2016
- 2016-02-08 RS RS20201400A patent/RS61079B1/sr unknown
- 2016-02-08 MX MX2017010292A patent/MX383316B/es unknown
- 2016-02-08 PT PT167038504T patent/PT3256169T/pt unknown
- 2016-02-08 SI SI201630984T patent/SI3256169T1/sl unknown
- 2016-02-08 PL PL16703850T patent/PL3256169T3/pl unknown
- 2016-02-08 ES ES16703850T patent/ES2830030T3/es active Active
- 2016-02-08 AU AU2016217654A patent/AU2016217654B2/en active Active
- 2016-02-08 SM SM20200655T patent/SMT202000655T1/it unknown
- 2016-02-08 BR BR112017017060-4A patent/BR112017017060A2/pt active Search and Examination
- 2016-02-08 EP EP20193488.2A patent/EP3769790A1/en active Pending
- 2016-02-08 EP EP16703850.4A patent/EP3256169B1/en active Active
- 2016-02-08 CN CN201680020425.6A patent/CN107429252B/zh active Active
- 2016-02-08 HU HUE16703850A patent/HUE052407T2/hu unknown
- 2016-02-08 CA CA2975850A patent/CA2975850C/en active Active
- 2016-02-08 US US15/549,549 patent/US10568973B2/en active Active
- 2016-02-08 JP JP2017541749A patent/JP6963503B2/ja active Active
- 2016-02-08 WO PCT/GB2016/050289 patent/WO2016128722A1/en not_active Ceased
- 2016-02-08 LT LTEP16703850.4T patent/LT3256169T/lt unknown
- 2016-02-08 SG SG11201706520UA patent/SG11201706520UA/en unknown
- 2016-02-08 IL IL287142A patent/IL287142B2/en unknown
- 2016-02-08 HR HRP20201831TT patent/HRP20201831T1/hr unknown
- 2016-02-08 DK DK16703850.4T patent/DK3256169T3/da active
-
2017
- 2017-08-08 IL IL253915A patent/IL253915B/en unknown
- 2017-08-09 PH PH12017501430A patent/PH12017501430A1/en unknown
- 2017-08-09 MX MX2021006762A patent/MX2021006762A/es unknown
-
2020
- 2020-02-20 US US16/796,439 patent/US11969478B2/en active Active
- 2020-11-26 CY CY20201101129T patent/CY1123947T1/el unknown
-
2024
- 2024-04-25 US US18/646,126 patent/US20240293580A1/en active Pending
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009105690A2 (en) * | 2008-02-21 | 2009-08-27 | Targeted Genetics Corporation | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| WO2013173129A2 (en) * | 2012-05-15 | 2013-11-21 | Avalanche Australia Pty Ltd. | Treatment of amd using aav sflt-1 |
Non-Patent Citations (2)
| Title |
|---|
| BAINBRIDGE, JAMES WB, ET AL., EFFECT OF GENE THERAPY ON VISUAL FUNCTION IN LEBER'S CONGENITALAMAUROSIS., 31 December 2008 (2008-12-31) * |
| DATABASE GENESEQ (ONLINE): TREATMENT-RELATED PROMOTER SEQUENCE, SEQ ID NO: 30., ", 16 January 2014 (2014-01-16) * |
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