Adeno-associated viral vectors for exon skipping in genes encoding unwanted domain proteins

摘要

The adeno-associated viral (AAV) vector consists of a modified snRNA sequence, a promoter and at least one reverse sequence directed against at least one exon splicing site, with the exon carrying the code for a dispensable domain of a protein, i.e. dystrophine. The AAV vector comprises aa serotype 1 capside or a 2/1 pseudotype, the modified snRNA sequence is type U7, the promoter is the native promoter of the snRNA sequence, and the splicing site is selected from the group comprising the 5' donor site, the 3' recipient site, the BP (Branch Point) sequence and the ESE sequences.