AAV-mediated gene therapy for Sialidosis
Diantha van de Vlekkert, Huimin Hu, Jason A. Weesner, Leigh E. Fremuth, Scott A. Brown, Meifen Lu, Elida M. Gomero, Yvan Campos, Heather Sheppard, Alessandra d’Azzo
This study tests AAV-mediated gene therapy for sialidosis in a mouse model, showing restored NEU1 activity and reduced tissue damage.
Molecular Therapy · May 25, 2024Peer-reviewedGenetics