Adeno-associated viral vector for exon skipping in a gene...

Details Adeno-associated viral vector for exon skipping in a gene... Adeno-associated viral vector for exon skipping in a gene...

A - Human Necessities

61

K

A61K 48/00 (2006.01) C12N 15/86 (2006.01)

Patent

CA 2576462

The invention concerns an adeno-associated vector comprising: a modified U7 snRNA sequence; the native U7 promoter; at least one antisense sequence directed against at least one splicing site of at least one exon, said exon encoding a dispensable-domain of dystrophin.

L'invention concerne un vecteur viral adéno-associé comprenant : une séquence snRNA modifiée de type U7 ; le promoteur natif de U7 ; au moins une séquence antisens dirigée contre au moins un site d'épissage d'au moins un exon, ledit exon codant un domaine dispensable de la dystrophine.

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