发明授权
- 专利标题： Exon skipping therapy for dysferlinopathies
- 专利标题（中）： 外显子跳跃治疗偏头痛
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申请号： US13501892申请日： 2010-10-18
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公开(公告)号： US08586560B2公开(公告)日： 2013-11-19
- 发明人: Nicolas Levy , Martin Krahn , Marc Bartoli , Luis Garcia
- 申请人： Nicolas Levy , Martin Krahn , Marc Bartoli , Luis Garcia
- 申请人地址： FR Paris FR Marseilles
- 专利权人： Institut National de la Sante et de la Recherche Medicale (INSERM),Universite de la Mediterranee—Aix Marseille II
- 当前专利权人： Institut National de la Sante et de la Recherche Medicale (INSERM),Universite de la Mediterranee—Aix Marseille II
- 当前专利权人地址： FR Paris FR Marseilles
- 代理机构： Whitham, Curtis, Christofferson & Cook, P.C.
- 国际申请： PCT/EP2010/065606 WO 20101018
- 国际公布： WO2011/054659 WO 20110512
- 主分类号： C12N15/11
- IPC分类号： C12N15/11 ; C12N15/00
摘要：
The present invention relates to methods for restoring the function of a mutated dysferlin comprising the step of preventing splicing of one or more exons which encode amino acid sequences that cause said dysferlin dysfunction. Particularly, the splicing of exon 32 is prevented. The present invention also relates to a method for treating a dysferlinopathy in a patient in need thereof, comprising the step of administering to said patient antisense oligonucleotides complementary to nucleic acid sequences that are necessary for correct splicing of one or more exons which encode amino acid sequences that cause said dysfunction. Particularly, the splicing of exon 32 is prevented.
公开/授权文献
- US20120208865A1 EXON SKIPPING THERAPY FOR DYSFERLINOPATHIES 公开/授权日：2012-08-16
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