Chimeric U1 and U2 snRNA molecules carrying antisense sequences against the splice junctions of the dystrophin gene and their therapeutic applications

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专利权人:

发明人:

申请号：

EP09176221.1

公开号：

EP2151497B1

申请日:

2003.05.06

申请国别(地区):

EP

年份:

2014

代理人:

摘要：

A gene is described that encodes for a snRNA modified such that it contains an antisense sequence complementary to at least one of the two splice junctions at 5 and/or 3 of one of the exons encoding for a protein of therapeutic interest, whose sequence should be skipped during the splicing process that converts the pre-mRNA to the mature mRNA. The gene is particularly advantageous which it contain segments complementary to both splice junctions. If introduced into appropriate vectors, the gene is useful in gene therapy, particularly in the treatment of Duchenne Muscular Dystrophies. dystrophic diseases.

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