KR20210005994A - 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터 - Google Patents

유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터 Download PDF

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KR20210005994A
KR20210005994A KR1020217000034A KR20217000034A KR20210005994A KR 20210005994 A KR20210005994 A KR 20210005994A KR 1020217000034 A KR1020217000034 A KR 1020217000034A KR 20217000034 A KR20217000034 A KR 20217000034A KR 20210005994 A KR20210005994 A KR 20210005994A
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nucleic acid
sequence
seq
fxn
vector
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리차드 제이 새멀스키
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뱀부 테라퓨틱스 인코포레이티드
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
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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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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KR1020217000034A 2015-11-05 2016-11-01 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터 Withdrawn KR20210005994A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562251288P 2015-11-05 2015-11-05
US62/251,288 2015-11-05
US201662411980P 2016-10-24 2016-10-24
US62/411,980 2016-10-24
PCT/IB2016/056572 WO2017077451A1 (en) 2015-11-05 2016-11-01 Modified friedreich ataxia genes and vectors for gene therapy

Related Parent Applications (1)

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KR1020187015819A Division KR102200642B1 (ko) 2015-11-05 2016-11-01 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터

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KR20210005994A true KR20210005994A (ko) 2021-01-15

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KR1020217000034A Withdrawn KR20210005994A (ko) 2015-11-05 2016-11-01 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터
KR1020187015819A Expired - Fee Related KR102200642B1 (ko) 2015-11-05 2016-11-01 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터

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US (2) US10548947B2 (https=)
EP (1) EP3370778A1 (https=)
JP (2) JP6767483B2 (https=)
KR (2) KR20210005994A (https=)
CN (1) CN108348621A (https=)
AU (2) AU2016347887B2 (https=)
BR (1) BR112018007453A2 (https=)
CA (1) CA2947584A1 (https=)
CO (1) CO2018004564A2 (https=)
HK (1) HK1250924A1 (https=)
IL (1) IL259141A (https=)
MX (1) MX2018005084A (https=)
PE (1) PE20181156A1 (https=)
PH (1) PH12018500964A1 (https=)
RU (2) RU2021102893A (https=)
SG (2) SG10201912763QA (https=)
TW (2) TWI743442B (https=)
WO (1) WO2017077451A1 (https=)

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