BR112015023038A2 - composições para salto de exon para o tratamento da distrofia muscular - Google Patents

composições para salto de exon para o tratamento da distrofia muscular

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Publication number
BR112015023038A2
BR112015023038A2 BR112015023038A BR112015023038A BR112015023038A2 BR 112015023038 A2 BR112015023038 A2 BR 112015023038A2 BR 112015023038 A BR112015023038 A BR 112015023038A BR 112015023038 A BR112015023038 A BR 112015023038A BR 112015023038 A2 BR112015023038 A2 BR 112015023038A2
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BR
Brazil
Prior art keywords
muscular dystrophy
treating muscular
compositions
exon
exon jump
Prior art date
Application number
BR112015023038A
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English (en)
Inventor
Elizabeth Frank Diane
K Bestwick Richard
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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 BR112015023038A2 publication Critical patent/BR112015023038A2/pt

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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
    • C12N15/113—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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • 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
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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
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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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
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Abstract

resumo “composições para salto de exon para o tratamento da distrofia muscular” moléculas antissenso capazes de se ligarem a um sítio alvo selecionado no gene daistrofna huanapara induzir o salto do exon 53 são descritas.
BR112015023038A 2013-03-14 2014-03-14 composições para salto de exon para o tratamento da distrofia muscular BR112015023038A2 (pt)

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
BR112015023038A2 true BR112015023038A2 (pt) 2017-11-14

Family

ID=50694008

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112015023038A BR112015023038A2 (pt) 2013-03-14 2014-03-14 composições para salto de exon para o tratamento da distrofia muscular

Country Status (26)

Country Link
US (5) US20140315977A1 (pt)
EP (3) EP3760720A1 (pt)
JP (4) JP2016521119A (pt)
KR (5) KR20220061280A (pt)
CN (2) CN110066793A (pt)
AU (3) AU2014236160A1 (pt)
BR (1) BR112015023038A2 (pt)
CA (1) CA2903872A1 (pt)
CY (1) CY1121876T1 (pt)
DK (1) DK2970964T3 (pt)
EA (1) EA201591694A1 (pt)
ES (1) ES2714290T3 (pt)
HK (1) HK1218138A1 (pt)
HR (1) HRP20190382T1 (pt)
HU (1) HUE042218T2 (pt)
IL (2) IL241047B (pt)
LT (1) LT2970964T (pt)
MX (2) MX366274B (pt)
NZ (1) NZ631132A (pt)
PL (1) PL2970964T3 (pt)
PT (1) PT2970964T (pt)
RS (1) RS58529B1 (pt)
SA (1) SA515361121B1 (pt)
SI (1) SI2970964T1 (pt)
TR (1) TR201903009T4 (pt)
WO (1) WO2014153240A2 (pt)

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