ADENOASSOCIATED VIRUS VECTORS FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSES

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 II.

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Bibliographische Detailangaben
Hauptverfasser: AREBA HAURIGOT, VIRGINIA, MOTAS MALLOL, SANDRA, BOSCH TUBERT, MARIA FATIMA
Format: Patent
Sprache:eng
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Beschreibung
Zusammenfassung: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 II.