Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
AssigneesEsteve Pharmaceuticals, S.A., Universitat Autonoma De Barcelona
InventorsCarles Roca Lecha, Virginia A. Haurigot-Mendonça, Fàtima Bosch Tubert
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Abstract
The present invention provides new adeno-associated virus-derived vectors and pharmaceutical compositions containing the same for the treatment of lysosomal storage disorders and specially, for the treatment of mucopolysaccharidoses Type IIID.
Claims14
A polynucleotide comprising an expression cassette, wherein the expression cassette comprises a transcriptional regulatory region comprising a promoter operatively linked to a nucleotide sequence as set forth in SEQ ID NO:
- 4 encoding the N-acetylglucosamine 6-sulfatase (GNS) protein.
A plasmid pAAV-CAG-ohGNS-version3 having accession number DSM 32345, as set forth in SEQ ID NO:
- 8.
Dependencies are inferred from the claim text.