JP2016502858A - 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 - Google Patents

筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 Download PDF

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JP2016502858A
JP2016502858A JP2015549818A JP2015549818A JP2016502858A JP 2016502858 A JP2016502858 A JP 2016502858A JP 2015549818 A JP2015549818 A JP 2015549818A JP 2015549818 A JP2015549818 A JP 2015549818A JP 2016502858 A JP2016502858 A JP 2016502858A
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antisense
antisense oligonucleotide
oligonucleotide
exon
dystrophin
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JP2016502858A5 (OSRAM
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リチャード ケー． ベストウィック，
リチャード ケー． ベストウィック，
ダイアン エリザベス フランク，
ダイアン エリザベス フランク，
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サレプタ セラピューティクス， インコーポレイテッド
サレプタ セラピューティクス， インコーポレイテッド
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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
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/3513—Protein; Peptide
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Epidemiology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Plant Substances (AREA)
JP2015549818A 2012-12-20 2013-12-20 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 Withdrawn JP2016502858A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261739968P 2012-12-20 2012-12-20
US61/739,968 2012-12-20
PCT/US2013/077216 WO2014100714A1 (en) 2012-12-20 2013-12-20 Improved exon skipping compositions for treating muscular dystrophy

Related Child Applications (1)

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JP2018081272A Division JP2018110601A (ja) 2012-12-20 2018-04-20 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物

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JP2016502858A true JP2016502858A (ja) 2016-02-01
JP2016502858A5 JP2016502858A5 (OSRAM) 2017-02-02

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JP2015549818A Withdrawn JP2016502858A (ja) 2012-12-20 2013-12-20 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物
JP2018081272A Pending JP2018110601A (ja) 2012-12-20 2018-04-20 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物
JP2019003308A Pending JP2019050834A (ja) 2012-12-20 2019-01-11 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物

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JP2018081272A Pending JP2018110601A (ja) 2012-12-20 2018-04-20 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物
JP2019003308A Pending JP2019050834A (ja) 2012-12-20 2019-01-11 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物

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US (5) US20150361428A1 (OSRAM)
EP (2) EP2935584A1 (OSRAM)
JP (3) JP2016502858A (OSRAM)
KR (2) KR20200143739A (OSRAM)
CN (2) CN106414739A (OSRAM)
AU (3) AU2013364158A1 (OSRAM)
BR (1) BR112015014987A2 (OSRAM)
CA (1) CA2894899A1 (OSRAM)
EA (1) EA201591178A1 (OSRAM)
HK (1) HK1216902A1 (OSRAM)
IL (1) IL239491A0 (OSRAM)
MX (2) MX2015008035A (OSRAM)
WO (1) WO2014100714A1 (OSRAM)

Cited By (2)

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Publication number Priority date Publication date Assignee Title
JP2016521119A (ja) * 2013-03-14 2016-07-21 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィを処置するためのエキソンスキッピング組成物
JP2020537501A (ja) * 2017-09-22 2020-12-24 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーのためのエクソンスキッピングオリゴマーコンジュゲート

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