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US 12,146,169 B2Granted
Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type IV A
AssigneesEsteve Pharmaceuticals, S.A., Universitat Autonoma De Barcelona
InventorsMaria Fátima Bosch Tubert, Victor Sanchez Clares, Albert Ribera Sanchez, Virginia A. Haurigot
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Abstract
The present invention provides new polynucleotide sequences, adeno-associated virus-derived vectors and pharmaceutical compositions containing the same for the treatment of lysosomal storage disorders and specially, for the treatment of mucopolysaccharidosis type IVA or Morquio A syndrome.
Claims12
An isolated polynucleotide having a nucleotide sequence selected from the group consisting of SEQ ID NO:
- 3, SEQ ID NO:
- 5, and SEQ ID NO:
- 7, wherein the polynucleotide encodes a functional human galactosamine (N-acetyl)-6-sulfatase.
Dependencies are inferred from the claim text.