EA201591694A1 - Композиции для пропуска экзонов для лечения мышечной дистрофии - Google Patents

Композиции для пропуска экзонов для лечения мышечной дистрофии

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Publication number
EA201591694A1
EA201591694A1 EA201591694A EA201591694A EA201591694A1 EA 201591694 A1 EA201591694 A1 EA 201591694A1 EA 201591694 A EA201591694 A EA 201591694A EA 201591694 A EA201591694 A EA 201591694A EA 201591694 A1 EA201591694 A1 EA 201591694A1
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EA
Eurasian Patent Office
Prior art keywords
exons
admission
compositions
treatment
muscular dystrophy
Prior art date
Application number
EA201591694A
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English (en)
Inventor
Ричард К. Бестуик
Дайан Элизабет Франк
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Сарепта Терапьютикс, Инк.
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Application filed by Сарепта Терапьютикс, Инк. filed Critical Сарепта Терапьютикс, Инк.
Publication of EA201591694A1 publication Critical patent/EA201591694A1/ru

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3513—Protein; Peptide
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3535—Nitrogen
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)

Abstract

Раскрыты антисмысловые молекулы, способные связываться с сайтом-мишенью в гене дистрофина человека для индуцирования пропуска экзона 53.
EA201591694A 2013-03-14 2014-03-14 Композиции для пропуска экзонов для лечения мышечной дистрофии EA201591694A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361782706P 2013-03-14 2013-03-14
PCT/US2014/029766 WO2014153240A2 (en) 2013-03-14 2014-03-14 Exon skipping compositions for treating muscular dystrophy

Publications (1)

Publication Number Publication Date
EA201591694A1 true EA201591694A1 (ru) 2016-06-30

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
EA201591694A EA201591694A1 (ru) 2013-03-14 2014-03-14 Композиции для пропуска экзонов для лечения мышечной дистрофии

Country Status (26)

Country Link
US (5) US20140315977A1 (ru)
EP (3) EP3760720A1 (ru)
JP (3) JP2016521119A (ru)
KR (4) KR20150133752A (ru)
CN (2) CN110066793A (ru)
AU (3) AU2014236160A1 (ru)
BR (1) BR112015023038A2 (ru)
CA (1) CA2903872A1 (ru)
CY (1) CY1121876T1 (ru)
DK (1) DK2970964T3 (ru)
EA (1) EA201591694A1 (ru)
ES (1) ES2714290T3 (ru)
HK (1) HK1218138A1 (ru)
HR (1) HRP20190382T1 (ru)
HU (1) HUE042218T2 (ru)
IL (2) IL241047B (ru)
LT (1) LT2970964T (ru)
MX (2) MX366274B (ru)
NZ (1) NZ631132A (ru)
PL (1) PL2970964T3 (ru)
PT (1) PT2970964T (ru)
RS (1) RS58529B1 (ru)
SA (1) SA515361121B1 (ru)
SI (1) SI2970964T1 (ru)
TR (1) TR201903009T4 (ru)
WO (1) WO2014153240A2 (ru)

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