JP6953131B2 - 筋強直性ジストロフィーの処置 - Google Patents

筋強直性ジストロフィーの処置 Download PDF

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Publication number
JP6953131B2
JP6953131B2 JP2016562263A JP2016562263A JP6953131B2 JP 6953131 B2 JP6953131 B2 JP 6953131B2 JP 2016562263 A JP2016562263 A JP 2016562263A JP 2016562263 A JP2016562263 A JP 2016562263A JP 6953131 B2 JP6953131 B2 JP 6953131B2
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mbnl
mbnl1
δct3
splicing
modified
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JP2016562263A
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English (en)
Japanese (ja)
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JP2017511145A (ja
Inventor
ファーリング，ドゥニ
セルジュアン，ニコラ
カイエ−ブーダン，マリー−ローレ
アランデル，リュドヴィック
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Centre National de la Recherche Scientifique
Institute of Myology
Sorbonne Université
Original Assignee
Centre National de la Recherche Scientifique
Institute of Myology
Sorbonne Université
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Priority to JP2021042924A priority Critical patent/JP7739017B2/ja
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
JP2016562263A 2014-04-14 2015-04-14 筋強直性ジストロフィーの処置 Active JP6953131B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2021042924A JP7739017B2 (ja) 2014-04-14 2021-03-16 筋強直性ジストロフィーの処置

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EPPCT/EP2014/057553 2014-04-14
PCT/EP2014/057553 WO2015158365A1 (en) 2014-04-14 2014-04-14 Treatment of myotonic dystrophy
PCT/EP2015/058111 WO2015158740A1 (en) 2014-04-14 2015-04-14 Treatment of myotonic dystrophy

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2021042924A Division JP7739017B2 (ja) 2014-04-14 2021-03-16 筋強直性ジストロフィーの処置

Publications (2)

Publication Number Publication Date
JP2017511145A JP2017511145A (ja) 2017-04-20
JP6953131B2 true JP6953131B2 (ja) 2021-10-27

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ID=50588655

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2016562263A Active JP6953131B2 (ja) 2014-04-14 2015-04-14 筋強直性ジストロフィーの処置
JP2021042924A Active JP7739017B2 (ja) 2014-04-14 2021-03-16 筋強直性ジストロフィーの処置
JP2023072627A Active JP7752148B2 (ja) 2014-04-14 2023-04-26 筋強直性ジストロフィーの処置

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2021042924A Active JP7739017B2 (ja) 2014-04-14 2021-03-16 筋強直性ジストロフィーの処置
JP2023072627A Active JP7752148B2 (ja) 2014-04-14 2023-04-26 筋強直性ジストロフィーの処置

Country Status (6)

Country Link
US (1) US10799556B2 (cg-RX-API-DMAC7.html)
EP (1) EP3145529B1 (cg-RX-API-DMAC7.html)
JP (3) JP6953131B2 (cg-RX-API-DMAC7.html)
CA (1) CA2944606C (cg-RX-API-DMAC7.html)
ES (1) ES2970841T3 (cg-RX-API-DMAC7.html)
WO (2) WO2015158365A1 (cg-RX-API-DMAC7.html)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015158365A1 (en) * 2014-04-14 2015-10-22 Association Institut De Myologie Treatment of myotonic dystrophy
AU2020347503B2 (en) * 2019-09-12 2026-07-16 Association Institut De Myologie Nucleic acid sequence for regulation of transgene expression
WO2024249893A1 (en) * 2023-05-31 2024-12-05 The Trustees Of Columbia University In The City Of New York Generation of mature neurons differentiated from pluripotent stem cells

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2019143A1 (en) * 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
CA2777486A1 (en) * 2008-10-15 2010-04-22 4S3 Bioscience Inc. Methods and compositions for treatment of myotonic dystrophy
WO2015158365A1 (en) 2014-04-14 2015-10-22 Association Institut De Myologie Treatment of myotonic dystrophy

Also Published As

Publication number Publication date
WO2015158740A1 (en) 2015-10-22
US20170035841A1 (en) 2017-02-09
CA2944606A1 (en) 2015-10-22
WO2015158365A1 (en) 2015-10-22
CA2944606C (en) 2023-06-27
ES2970841T3 (es) 2024-05-30
JP2023099082A (ja) 2023-07-11
JP7739017B2 (ja) 2025-09-16
EP3145529B1 (en) 2023-11-15
EP3145529A1 (en) 2017-03-29
JP2021101713A (ja) 2021-07-15
US10799556B2 (en) 2020-10-13
JP2017511145A (ja) 2017-04-20
JP7752148B2 (ja) 2025-10-09

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