CN105392884B - 用于治疗肌肉萎缩的外显子跳跃组合物 - Google Patents

用于治疗肌肉萎缩的外显子跳跃组合物 Download PDF

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CN105392884B
CN105392884B CN201480027371.7A CN201480027371A CN105392884B CN 105392884 B CN105392884 B CN 105392884B CN 201480027371 A CN201480027371 A CN 201480027371A CN 105392884 B CN105392884 B CN 105392884B
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antisense
acid
dystrophin
oligomer
exon
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CN105392884A (zh
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R.K.贝斯特维克
D.E.弗兰克
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Sarepta Therapeutics Inc
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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
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • 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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    • C12N2310/00—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
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    • C12N2310/3513—Protein; Peptide
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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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CN201480027371.7A 2013-03-14 2014-03-14 用于治疗肌肉萎缩的外显子跳跃组合物 Active CN105392884B (zh)

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US201361782706P 2013-03-14 2013-03-14
US61/782706 2013-03-14
PCT/US2014/029766 WO2014153240A2 (en) 2013-03-14 2014-03-14 Exon skipping compositions for treating muscular dystrophy

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