Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type iv a
AssigneesEsteve Pharmaceuticals, S.A., Universitat AutonóNoma De Barcelona
InventorsMaria-Fàtima 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.
Claims13
A recombinant adeno-associated viral (AAV) vector comprising a polynucleotide sequence encoding a functional human galactosamine (N-acetyl)-6-sulfatase (GALNS), wherein the polynucleotide sequence exhibits between 75% to 90% identity with the polynucleotide sequence as set forth in SEQ ID NO:
- 1, and wherein AAV vector expression of the polynucleotide sequence provides a GALNS enzyme having enzymatic activity levels which are higher than wild-type GALNS enzymatic activity levels in a subject.
Dependencies are inferred from the claim text.