Patrice Vidal

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

Francesco Puzzo, Pasqualina Colella, Maria Grazia Biferi et al. · Science Translational Medicine · 2017 · 133 citations

Pharmacy, Biochemistry, Biotechnology +11

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

Giuseppe Ronzitti, Giulia Bortolussi, Remco van Dijk et al. · Molecular Therapy — Methods & Clinical Development · 2016 · 62 citations · Full text

Life Sciences, Biotechnology, Molecular Biology +16

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

Fanny Collaud, Giulia Bortolussi, Laurence Guianvarc’h et al. · Molecular Therapy — Methods & Clinical Development · 2018 · 61 citations · Full text

Medicine, Molecular Genetics, Molecular Medicine +4

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

Patrice Vidal, Serena Pagliarani, Pasqualina Colella et al. · Molecular Therapy · 2017 · 37 citations · Full text

Life Sciences, Genetics, Molecular Biology +15

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

Giulia De Sabbata, Florence Boisgérault, Corrado Guarnaccia et al. · Molecular Therapy — Methods & Clinical Development · 2020 · 23 citations · Full text

Medicine, Biotechnology, Molecular Biology +14