KR102684387B1 - 신규한 마이크로-디스트로핀 및 관련된 사용 방법 - Google Patents

신규한 마이크로-디스트로핀 및 관련된 사용 방법 Download PDF

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KR102684387B1
KR102684387B1 KR1020177022718A KR20177022718A KR102684387B1 KR 102684387 B1 KR102684387 B1 KR 102684387B1 KR 1020177022718 A KR1020177022718 A KR 1020177022718A KR 20177022718 A KR20177022718 A KR 20177022718A KR 102684387 B1 KR102684387 B1 KR 102684387B1
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제프리 에스. 챔벌린
줄리안 라모스
스티븐 디. 하우쉬카
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유니버시티 오브 워싱톤
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707Muscular dystrophy
    • C07K14/4708Duchenne dystrophy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707Muscular dystrophy
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    • C07ORGANIC CHEMISTRY
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    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4716Muscle proteins, e.g. myosin, actin
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Bioinformatics & Cheminformatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Physical Education & Sports Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
KR1020177022718A 2015-01-16 2016-01-15 신규한 마이크로-디스트로핀 및 관련된 사용 방법 Active KR102684387B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562104537P 2015-01-16 2015-01-16
US62/104,537 2015-01-16
PCT/US2016/013733 WO2016115543A2 (en) 2015-01-16 2016-01-15 Novel micro-dystrophins and related methods of use

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KR20170107481A KR20170107481A (ko) 2017-09-25
KR102684387B1 true KR102684387B1 (ko) 2024-07-11

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US (4) US10479821B2 (enExample)
EP (2) EP4599851A2 (enExample)
JP (4) JP6832280B2 (enExample)
KR (1) KR102684387B1 (enExample)
CN (1) CN107250364A (enExample)
AU (2) AU2016206518B2 (enExample)
CA (1) CA2972811A1 (enExample)
IL (4) IL315273A (enExample)
NZ (1) NZ734019A (enExample)
SG (1) SG11201705324UA (enExample)
WO (1) WO2016115543A2 (enExample)

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IL253246A0 (en) 2017-08-31
IL286316A (en) 2021-10-31
JP2018503374A (ja) 2018-02-08
EP4599851A2 (en) 2025-08-13
US20180148488A1 (en) 2018-05-31
JP6832280B2 (ja) 2021-02-24
IL297239A (en) 2022-12-01
US20250376496A1 (en) 2025-12-11
US11958888B2 (en) 2024-04-16
CN107250364A (zh) 2017-10-13
JP2024088814A (ja) 2024-07-02
KR20170107481A (ko) 2017-09-25
IL297239B1 (en) 2024-10-01
WO2016115543A3 (en) 2016-09-29
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IL297239B2 (en) 2025-02-01
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JP7164896B2 (ja) 2022-11-02
EP3245291A2 (en) 2017-11-22
JP2021072839A (ja) 2021-05-13
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US20200095298A1 (en) 2020-03-26
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AU2020203358A1 (en) 2020-06-11
US10479821B2 (en) 2019-11-19
SG11201705324UA (en) 2017-07-28
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