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Chimeric U2 snRNA molecules carrying antisense sequences against the splice junctions of the dystrophin gene and their therapeutic applications
Chimeric U2 snRNA molecules carrying antisense sequences against the splice junctions of the dystrophin gene and their therapeutic applications
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机译:带有抗肌萎缩蛋白基因剪接点的反义序列的嵌合U2 snRNA分子及其治疗应用
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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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