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MX2017010292A - Promotor de epitelio del pigmento retinal 65 (rpe65) optimizado y secuencias codificantes. - Google Patents

Promotor de epitelio del pigmento retinal 65 (rpe65) optimizado y secuencias codificantes.

Info

Publication number
MX2017010292A
MX2017010292A MX2017010292A MX2017010292A MX2017010292A MX 2017010292 A MX2017010292 A MX 2017010292A MX 2017010292 A MX2017010292 A MX 2017010292A MX 2017010292 A MX2017010292 A MX 2017010292A MX 2017010292 A MX2017010292 A MX 2017010292A
Authority
MX
Mexico
Prior art keywords
epetel
rpe65
promoter
optimized
coding sequences
Prior art date
Application number
MX2017010292A
Other languages
English (en)
Other versions
MX383316B (es
Inventor
Ali Robin
Smith Alexander
Original Assignee
Ucl Business Plc
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 Plc filed Critical Ucl Business Plc
Publication of MX2017010292A publication Critical patent/MX2017010292A/es
Publication of MX383316B publication Critical patent/MX383316B/es

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    • 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
    • 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
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    • C—CHEMISTRY; METALLURGY
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    • 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
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    • 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
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    • C12N15/86—Viral vectors
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    • 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)
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    • 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)
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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

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  • 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)
  • Wood Science & Technology (AREA)
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  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (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)
  • Urology & Nephrology (AREA)
  • Virology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Ophthalmology & Optometry (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Eye Examination Apparatus (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

La presente invención se refiere a la prevención y/o tratamiento de distrofia retinal en un paciente, que incluye amaurosis congénita de Leber (LCA).
MX2017010292A 2015-02-09 2016-02-08 Promotor de epitelio del pigmento retinal 65 (rpe65) optimizado y secuencias codificantes. MX383316B (es)

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 (2)

Publication Number Publication Date
MX2017010292A true MX2017010292A (es) 2018-03-23
MX383316B MX383316B (es) 2025-03-13

Family

ID=52746363

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2017010292A MX383316B (es) 2015-02-09 2016-02-08 Promotor de epitelio del pigmento retinal 65 (rpe65) optimizado y secuencias codificantes.
MX2021006762A MX2021006762A (es) 2015-02-09 2017-08-09 Promotor de epitelio del pigmento retinal 65 (rpe65) optimizado y secuencias codificantes.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2021006762A MX2021006762A (es) 2015-02-09 2017-08-09 Promotor de epitelio del pigmento retinal 65 (rpe65) optimizado y secuencias codificantes.

Country Status (24)

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

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
CN109689033A (zh) * 2016-04-28 2019-04-26 星火治疗有限公司 病毒载体编码异构水解醇的相对效力测定
US11591615B2 (en) * 2017-11-30 2023-02-28 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
WO2021055760A1 (en) * 2019-09-18 2021-03-25 Intergalactic Therapeutics, Inc. Synthetic dna vectors and methods of use
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
AU2021314400A1 (en) * 2020-07-21 2023-03-23 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
AU2022340595A1 (en) * 2021-09-06 2024-04-18 Huidagene Therapeutics (Singapore) Pte. Ltd. Treatment of rpe65-associated eye diseases and disorders
EP4646212A1 (en) * 2023-01-05 2025-11-12 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

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
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

Also Published As

Publication number Publication date
JP2018510620A (ja) 2018-04-19
BR112017017060A2 (pt) 2018-04-10
JP6963503B2 (ja) 2021-11-10
HUE052407T2 (hu) 2021-04-28
SG11201706520UA (en) 2017-09-28
HRP20201831T1 (hr) 2021-01-08
US20240293580A1 (en) 2024-09-05
EP3769790A1 (en) 2021-01-27
DK3256169T3 (da) 2020-11-23
CA2975850C (en) 2024-06-18
MX383316B (es) 2025-03-13
PH12017501430A1 (en) 2018-01-15
US10568973B2 (en) 2020-02-25
ES2830030T3 (es) 2021-06-02
GB201502137D0 (en) 2015-03-25
IL287142A (en) 2021-12-01
PT3256169T (pt) 2020-11-04
IL287142B1 (en) 2023-07-01
RS61079B1 (sr) 2020-12-31
US20200179535A1 (en) 2020-06-11
IL253915B (en) 2021-10-31
SI3256169T1 (sl) 2021-01-29
HK1248525A1 (zh) 2018-10-19
US11969478B2 (en) 2024-04-30
CA2975850A1 (en) 2016-08-18
SMT202000655T1 (it) 2021-01-05
PL3256169T3 (pl) 2021-05-04
US20180021458A1 (en) 2018-01-25
IL287142B2 (en) 2023-11-01
CY1123947T1 (el) 2022-03-24
AU2016217654B2 (en) 2021-12-09
AU2016217654A1 (en) 2017-09-28
NZ735329A (en) 2024-08-30
WO2016128722A1 (en) 2016-08-18
CN107429252B (zh) 2022-03-29
LT3256169T (lt) 2021-01-11
IL253915A0 (en) 2017-10-31
EP3256169B1 (en) 2020-09-02
MX2021006762A (es) 2021-07-15
CN107429252A (zh) 2017-12-01
EP3256169A1 (en) 2017-12-20

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