GeneticsMolecular TherapyPeer-reviewed
AAV-mediated gene therapy for Sialidosis
Diantha van de Vlekkert, Huimin Hu, Jason A. Weesner…·Alessandra d’Azzo
This study tests AAV-mediated gene therapy for sialidosis in a mouse model, showing restored NEU1 activity and reduced tissue damage.
11 citations