Edoardo Nusco

AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease

Francesca Iacobellis, Andrea Ballabio, A. Ballabio, 2017, Scientific Reports.

A chaperone enhances blood α-glucosidase activity in Pompe disease patients treated with enzyme replacement therapy.

Carmine Mollica, Anna Pichiecchio, Simona Fecarotta, 2014, Molecular therapy : the journal of the American Society of Gene Therapy.