Jean-Michel Rozet;Antoine Kichler;Isabelle Perrault;Xavier Gerard;Daniel Scherman;Josseline Kaplan;M. Arnold Munnich
发明人:
Jean-Michel Rozet,Antoine Kichler,Isabelle Perrault,Josseline Kaplan,Xavier Gerard,Daniel Scherman,M. Arnold Munnich
申请号：
US14546124
公开号：
US09487782B2
申请日:
2014.11.18
申请国别(地区):
US
年份:
2016
代理人:
摘要：
The present invention relates to a method for treating a Leber congenital amaurosis in a patient harboring the mutation c.2991+1655 A>；G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c.2291+1655 A>；G CEP290 mRNA.
