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 CNRS
Association Institut de Myologie
Sorbonne Universite
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Centre National de la Recherche Scientifique CNRS
Association Institut de Myologie
Sorbonne Universite
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Publication of JP2017511145A publication Critical patent/JP2017511145A/ja
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 (https=)
EP (1) EP3145529B1 (https=)
JP (3) JP6953131B2 (https=)
CA (1) CA2944606C (https=)
ES (1) ES2970841T3 (https=)
WO (2) WO2015158365A1 (https=)

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
CA3150413A1 (en) * 2019-09-12 2021-03-18 Denis Furling NUCLEIC ACID SEQUENCE FOR THE 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
WO2010044894A1 (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
JP2021101713A (ja) 2021-07-15
WO2015158740A1 (en) 2015-10-22
ES2970841T3 (es) 2024-05-30
US10799556B2 (en) 2020-10-13
JP2023099082A (ja) 2023-07-11
JP7752148B2 (ja) 2025-10-09
EP3145529A1 (en) 2017-03-29
EP3145529B1 (en) 2023-11-15
CA2944606A1 (en) 2015-10-22
US20170035841A1 (en) 2017-02-09
WO2015158365A1 (en) 2015-10-22
JP7739017B2 (ja) 2025-09-16
CA2944606C (en) 2023-06-27
JP2017511145A (ja) 2017-04-20

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