P. Vidal

Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector

L. D’Antiga, G. Ronzitti, T. Kishimoto, 2020, Molecular therapy. Methods & clinical development.

Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.

G. Comi, E. Malfatti, I. Richard, 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome

P. Bosma, G. Ronzitti, A. Muro, 2018, Molecular therapy. Methods & clinical development.

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase

M. Kay, P. Laforêt, G. Ronzitti, 2017, Science Translational Medicine.

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

P. Bosma, G. Ronzitti, Laetitia van Wittenberghe, 2016, Molecular therapy. Methods & clinical development.

Muscle‐specific, liver‐detargeted adeno‐associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa−/− mice

G. Ronzitti, F. Mingozzi, F. Collaud, 2023, Journal of inherited metabolic disease.