发明名称 |
EXON SKIPPING THERAPY FOR DYSTROPHIC EPIDERMOLYSIS BULLOSA |
摘要 |
The present invention also relates to an antisense oligonucleotide complementary to a nucleic acid sequence of COL7A1 gene that is necessary for correct splicing of one or more exons which encode amino acid sequence of type VII collagen implicated in dysfunction of a mutated type VII collagen wherein said exons are selected from the group consisting of exon 73, 74 or 80 of the COL7A1 gene. The present invention also relates to a method for the treatment of a patient suffering from Dystrophic Epidermolysis Bullosa caused by a dysfunction of a mutated type VII collagen, comprising the step of administering to said patient a least one antisense oligonucleotide according to the invention. |
申请公布号 |
EP2766480(A1) |
申请公布日期 |
2014.08.20 |
申请号 |
EP20120772927 |
申请日期 |
2012.10.11 |
申请人 |
INSERM - INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE;UNIVERSITÉ PARIS DESCARTES |
发明人 |
HOVNANIAN, ALAIN;TITEUX, MATTHIAS;TURCZYNSKI, SANDRINA |
分类号 |
C12N15/113;A61P17/00 |
主分类号 |
C12N15/113 |
代理机构 |
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代理人 |
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主权项 |
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