专利名称：TRICYCLO-DNA ANTISENSE
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发明人：Daniel SCHUMPERLI,Christian
LEUMANN,Denis FURLING,Luis
GARCIA,Thomas VOIT
美国姓名
申请号：US15428385
申请日：20170209
公开号：US20170260524A1
公开日：
20170914
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摘要：Provided are tricyclo-DNA (tc-DNA) AON and methods employing tc-DNA AON for modifying splicing events that occur during pre-mRNA processing. Tricyclo-DNA (tc-DNA) AON are described that may be ud to facilitate exon skipping or to mask intronic silencer quences and/or terminal stein-loop quences during pre-mRNA processing and to target RNa-mediated destruction of procesd mRNA. Tc-DNA AON described herein may be ud in methods for the treatment of Duchenne Muscular Dystrophy by skipping a mutated exon 23 or exon 51 within a dystrophin gene to restore functionality of a dystrophin protein; in methods for the treatment of Spinal Muscular Atrophy by masking an intronic silencing quence and/or a terminal stem-loop quence within an SMN2 gene to yield modified functional SMN2 protein, including an amino acid quence encoded by exon 7, which is capable of at least partially complementing a non-functional SMN1 protein; and in methods for the treatment of Steinert's Myotonic Dystrophy by targeting the destruction of a mutated DM1 mRNA comprising 3′-terminal CUG repeats.
申请人：ASSOCIATION INSTITUT DE MYOLOGIE,UNIVERSITE PIERRE ET MARIE
CURIE,UNIVERSITAT BERN,CENTRE NATIONAL DE LA RECHERCHE
SCIENTIFIQUE,INSTITUT NATIONAL DE LA SANTE DE LA RECHERCHE MEDICALE 地址：Paris FR,Paris FR,Bern CH,Paris FR,Paris FR
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国籍：FR,FR,CH,FR,FR
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