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
Authority
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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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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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    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/31Chemical structure of the backbone
    • 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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    • C12N2310/00Structure or type of the nucleic acid
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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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    • C12N2310/334Modified C
    • 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
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    • 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)
  • Chemical & Material Sciences (AREA)
  • 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)
  • Physical Education & Sports Medicine (AREA)
  • 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)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Plant Substances (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 (enExample)
EP (2) EP2935584A1 (enExample)
JP (3) JP2016502858A (enExample)
KR (2) KR20150099804A (enExample)
CN (2) CN111440796A (enExample)
AU (3) AU2013364158A1 (enExample)
BR (1) BR112015014987A2 (enExample)
CA (1) CA2894899A1 (enExample)
EA (1) EA201591178A1 (enExample)
HK (1) HK1216902A1 (enExample)
IL (1) IL239491A0 (enExample)
MX (2) MX2015008035A (enExample)
WO (1) WO2014100714A1 (enExample)

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AU2014236140B2 (en) 2013-03-14 2019-10-03 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
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JP2016502858A (ja) 2016-02-01
US20220259592A1 (en) 2022-08-18
EP3885439A1 (en) 2021-09-29
EA201591178A1 (ru) 2015-11-30
CN111440796A (zh) 2020-07-24
US20200339985A1 (en) 2020-10-29
MX2015008035A (es) 2016-03-17
KR20200143739A (ko) 2020-12-24
JP2019050834A (ja) 2019-04-04
WO2014100714A1 (en) 2014-06-26
BR112015014987A2 (pt) 2017-08-15
KR20150099804A (ko) 2015-09-01
EP2935584A1 (en) 2015-10-28
JP2018110601A (ja) 2018-07-19
AU2019253778A1 (en) 2019-11-07
HK1216902A1 (zh) 2016-12-09
CA2894899A1 (en) 2014-06-26
US20230104708A1 (en) 2023-04-06
AU2022201628A1 (en) 2022-03-31
MX2019008164A (es) 2019-09-13
AU2013364158A1 (en) 2015-07-09
US20170369875A1 (en) 2017-12-28
US20150361428A1 (en) 2015-12-17
CN106414739A (zh) 2017-02-15

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