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CHIMERIC SNRNA MOLECULES CARRYING ANTISENSE SEQUENCES AGAINST THE SPLICE JUNCTIONS OF THE DYSTROPHIN GENE AND THEIR THERAPEUTIC APPLICATIONS
CHIMERIC SNRNA MOLECULES CARRYING ANTISENSE SEQUENCES AGAINST THE SPLICE JUNCTIONS OF THE DYSTROPHIN GENE AND THEIR THERAPEUTIC APPLICATIONS
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机译:带有反义序列的营养不良基因的嵌合SNRNA分子及其在治疗中的应用
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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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