IL239491A0 - Dystrophin Antisense Oligonucleotides Exon Skipping Factors, Vectors and Preparations Containing Them and Their Uses - Google Patents

Dystrophin Antisense Oligonucleotides Exon Skipping Factors, Vectors and Preparations Containing Them and Their Uses

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Publication number
IL239491A0
IL239491A0 IL239491A IL23949115A IL239491A0 IL 239491 A0 IL239491 A0 IL 239491A0 IL 239491 A IL239491 A IL 239491A IL 23949115 A IL23949115 A IL 23949115A IL 239491 A0 IL239491 A0 IL 239491A0
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IL
Israel
Prior art keywords
dystrophin
vectors
compositions
same
antisense oligonucleotides
Prior art date
Application number
IL239491A
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English (en)
Hebrew (he)
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Sarepta Therapeutics Inc
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Publication date
Application filed by Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Publication of IL239491A0 publication Critical patent/IL239491A0/en

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    • CCHEMISTRY; METALLURGY
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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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/31Chemical structure of the backbone
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/314Phosphoramidates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181Peptide nucleic acid, PNA
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    • C12N2310/32Chemical structure of the sugar
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/33415-Methylcytosine
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3513Protein; Peptide
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
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  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Plant Substances (AREA)
  • Peptides Or Proteins (AREA)
IL239491A 2012-12-20 2015-06-18 Dystrophin Antisense Oligonucleotides Exon Skipping Factors, Vectors and Preparations Containing Them and Their Uses IL239491A0 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261739968P 2012-12-20 2012-12-20
PCT/US2013/077216 WO2014100714A1 (en) 2012-12-20 2013-12-20 Improved exon skipping compositions for treating muscular dystrophy

Publications (1)

Publication Number Publication Date
IL239491A0 true IL239491A0 (en) 2015-08-31

Family

ID=49950080

Family Applications (1)

Application Number Title Priority Date Filing Date
IL239491A IL239491A0 (en) 2012-12-20 2015-06-18 Dystrophin Antisense Oligonucleotides Exon Skipping Factors, Vectors and Preparations Containing Them and Their Uses

Country Status (13)

Country Link
US (5) US20150361428A1 (https=)
EP (2) EP2935584A1 (https=)
JP (3) JP2016502858A (https=)
KR (2) KR20150099804A (https=)
CN (2) CN111440796A (https=)
AU (3) AU2013364158A1 (https=)
BR (1) BR112015014987A2 (https=)
CA (1) CA2894899A1 (https=)
EA (1) EA201591178A1 (https=)
HK (1) HK1216902A1 (https=)
IL (1) IL239491A0 (https=)
MX (2) MX2015008035A (https=)
WO (1) WO2014100714A1 (https=)

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TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
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RS58529B1 (sr) * 2013-03-14 2019-04-30 Sarepta Therapeutics Inc Sastavi koji preskaču egzon za tretiranje mišićne distrofije
NZ731587A (en) 2013-03-14 2021-07-30 Sarepta Therapeutics Inc Exon skipping compositions for treating muscular dystrophy
CA2906812A1 (en) 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
TN2017000427A1 (en) 2015-04-08 2019-04-12 Univ Northwestern Compositions and methods for correcting limb girdle muscular dystrophy type 2c using exon skipping
EP3858993A1 (en) 2015-10-09 2021-08-04 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
FR3044926B1 (fr) * 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
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CA2894899A1 (en) 2014-06-26
US20200339985A1 (en) 2020-10-29
CN106414739A (zh) 2017-02-15
US20230104708A1 (en) 2023-04-06
AU2019253778A1 (en) 2019-11-07
US20170369875A1 (en) 2017-12-28
JP2018110601A (ja) 2018-07-19
EP2935584A1 (en) 2015-10-28
HK1216902A1 (zh) 2016-12-09
AU2013364158A1 (en) 2015-07-09
US20220259592A1 (en) 2022-08-18
AU2022201628A1 (en) 2022-03-31
JP2016502858A (ja) 2016-02-01
BR112015014987A2 (pt) 2017-08-15
JP2019050834A (ja) 2019-04-04
MX2015008035A (es) 2016-03-17
WO2014100714A1 (en) 2014-06-26
MX2019008164A (es) 2019-09-13
KR20200143739A (ko) 2020-12-24
CN111440796A (zh) 2020-07-24
KR20150099804A (ko) 2015-09-01
EA201591178A1 (ru) 2015-11-30
US20150361428A1 (en) 2015-12-17
EP3885439A1 (en) 2021-09-29

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