ADENO-ASSOCIATED VIRAL VECTOR FOR EXON SKIPPING IN A GENE ENCODING A DISPENSIBLE-DOMAIN PROTEIN
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United States of America Patent
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app pub date -
Apr 27, 2012
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Aug 17, 2004
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Abstract
The invention concerns an adeno-associated viral vector comprising:
- a U7 type modified snRNA sequence;the native U7 promoter;at least one antisense sequence directed against at least one splice site of at least one exon, the said exon encoding a dispensable domain of dystrophin.
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