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.

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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
coding sequences
rpe65 promoter
optimized
optimized rpe65
promoter
Prior art date
Application number
MX2017010292A
Other languages
English (en)
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

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    • 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
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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
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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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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    • 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
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    • 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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    • 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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    • C12N2750/00011—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)
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  • 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)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Eye Examination Apparatus (AREA)
  • Percussion Or Vibration Massage (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. MX2017010292A (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 (1)

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

Family

ID=52746363

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2017010292A MX2017010292A (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 (2) US10568973B2 (es)
EP (2) EP3256169B1 (es)
JP (1) JP6963503B2 (es)
CN (1) CN107429252B (es)
AU (1) AU2016217654B2 (es)
BR (1) BR112017017060A2 (es)
CA (1) CA2975850A1 (es)
CY (1) CY1123947T1 (es)
DK (1) DK3256169T3 (es)
ES (1) ES2830030T3 (es)
GB (1) GB201502137D0 (es)
HK (1) HK1248525A1 (es)
HR (1) HRP20201831T1 (es)
HU (1) HUE052407T2 (es)
IL (2) IL287142B2 (es)
LT (1) LT3256169T (es)
MX (2) MX2017010292A (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)
WO (1) WO2016128722A1 (es)

Families Citing this family (9)

* 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
SG11201809388SA (en) * 2016-04-28 2018-11-29 Spark Therapeutics Inc Relative potency assay for viral vector encoding isomerohydrolases
BR112020010928A2 (pt) * 2017-11-30 2020-11-24 Friedrich Miescher Institute For Biomedical Research synpiii, um promotor para a expressão de gene específica em epitélio de pigmento retinal
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
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
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

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2005268221A1 (en) * 2004-08-06 2006-02-09 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
PL3418300T3 (pl) * 2011-07-18 2021-05-04 Institute For Research In Biomedicine Neutralizujące przeciwciała przeciwko wirusowi grypy A i ich zastosowania
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

Also Published As

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

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