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BR112015014987A2 - Aprimoradas composições que pulam éxon para o tratamento da distrofia muscular - Google Patents

Aprimoradas composições que pulam éxon para o tratamento da distrofia muscular

Info

Publication number
BR112015014987A2
BR112015014987A2 BR112015014987A BR112015014987A BR112015014987A2 BR 112015014987 A2 BR112015014987 A2 BR 112015014987A2 BR 112015014987 A BR112015014987 A BR 112015014987A BR 112015014987 A BR112015014987 A BR 112015014987A BR 112015014987 A2 BR112015014987 A2 BR 112015014987A2
Authority
BR
Brazil
Prior art keywords
treatment
muscular dystrophy
skipping
exon
improved
Prior art date
Application number
BR112015014987A
Other languages
English (en)
Inventor
K Bestwick Richard
Elizabeth Frank Diane
Original Assignee
Sarepta Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Publication of BR112015014987A2 publication Critical patent/BR112015014987A2/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
    • 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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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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)
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  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
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  • 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)

Abstract

APRIMORADAS COMPOSIÇÕES QUE PULAM ÉXON PARA O TRATAMENTO DA DISTROFIA MUSCULAR. Moléculas antissentido capazes de se ligar a um sítio alvo selecionado no gene da distrofina humana para induzir o salto do éxon 53 são descritas.
BR112015014987A 2012-12-20 2013-12-20 Aprimoradas composições que pulam éxon para o tratamento da distrofia muscular BR112015014987A2 (pt)

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
BR112015014987A2 true BR112015014987A2 (pt) 2017-08-15

Family

ID=49950080

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112015014987A BR112015014987A2 (pt) 2012-12-20 2013-12-20 Aprimoradas composições que pulam éxon para o tratamento da distrofia muscular

Country Status (13)

Country Link
US (5) US20150361428A1 (pt)
EP (2) EP3885439A1 (pt)
JP (3) JP2016502858A (pt)
KR (2) KR20200143739A (pt)
CN (2) CN106414739A (pt)
AU (3) AU2013364158A1 (pt)
BR (1) BR112015014987A2 (pt)
CA (1) CA2894899A1 (pt)
EA (1) EA201591178A1 (pt)
HK (1) HK1216902A1 (pt)
IL (1) IL239491A0 (pt)
MX (2) MX2015008035A (pt)
WO (1) WO2014100714A1 (pt)

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KR20150099804A (ko) 2015-09-01
EA201591178A1 (ru) 2015-11-30
US20230104708A1 (en) 2023-04-06
MX2015008035A (es) 2016-03-17
US20200339985A1 (en) 2020-10-29
CN106414739A (zh) 2017-02-15
CA2894899A1 (en) 2014-06-26
JP2019050834A (ja) 2019-04-04
HK1216902A1 (zh) 2016-12-09
AU2022201628A1 (en) 2022-03-31
AU2019253778A1 (en) 2019-11-07
JP2016502858A (ja) 2016-02-01
EP2935584A1 (en) 2015-10-28
AU2013364158A1 (en) 2015-07-09
CN111440796A (zh) 2020-07-24
US20170369875A1 (en) 2017-12-28
IL239491A0 (en) 2015-08-31
JP2018110601A (ja) 2018-07-19
MX2019008164A (es) 2019-09-13
EP3885439A1 (en) 2021-09-29
US20220259592A1 (en) 2022-08-18
US20150361428A1 (en) 2015-12-17
KR20200143739A (ko) 2020-12-24
WO2014100714A1 (en) 2014-06-26

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