E. Zaccariotto

In vivo delivery of naked antisense oligos in aged mdx mice: Analysis of dystrophin restoration in skeletal and cardiac muscle

C. Reggiani, G. Occhi, T. Zaglia, 2008, Neuromuscular Disorders.

A novel brain-penetrant oral UGT8 inhibitor decreases in vivo galactosphingolipid biosynthesis in murine Krabbe disease.

R. Barker, S. Sardi, Hyejung Park, 2022, Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie.

Gene therapy approaches for lysosomal storage disorders, a good model for the treatment of mendelian diseases

R. Tomanin, M. Scarpa, C. Bellettato, 2012, Acta paediatrica.

Genetics and Therapies for GM2 Gangliosidosis

T. Cox, M. Cachón-González, E. Zaccariotto, 2018, Current gene therapy.