PhD in Biomedical Sciences and Biotechnology
Home – PhD students – Faculty – Seminars
- Ronzitti G, Bortolussi G, van Dijk R, Collaud F, Charles S, Leborgne C, Vidal P, Martin S, Gjata B, Simon Sola M, van Wittenberghe L, Vignaud A, Veron P, Bosma PJ, Muro AF, Mingozzi F. (2016) A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar. Molecular Therapy Methods Clinical Development
- Barbon E, Ferrarese M, van Wittenberghe L, Sanatine P, Ronzitti G, Collaud F, Colella P, Pinotti M and Mingozzi F (2016) Transposon-mediated generation of cellular and mouse models of splicing mutations to assess the efficacy of snRNA-based therapeutics Molecular Therapy Nucleic Acids
- Meliani A, Boisgerault F, Fitzpatrick Z, Marmier S, Leborgne C, Collaud F, Simon Sola M, Charles S, Ronzitti G, Vignaud A, van Wittenberghe L, Marolleau B, Jouen F, Tan S, Boyer O, Christophe O, Brisson A, Maguire C, Mingozzi F (2017) Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors. Blood Advances
- Puzzo F, Colella P, Biferi MG, Bali D, Paulk NK, Vidal P, Collaud F, Simon-Sola M, Charles S, Hardet R, Leborgne C, Meliani A, Cohen-Tannoudji M, Astord S, Gjata B, Sellier P, van Wittenberghe L, Vignaud A, Boisgerault F, Barkats M, Laforet P, Kay MA, Koeberl DD, Ronzitti G, Mingozzi F (2017) Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes. Science translational medicine
- Vidal P, Pagliarani S, Colella P, Costa Verdera H, Jauze L, Gjorgjieva M, Puzzo F, Marmier S, Collaud F, Simon Sola M, Charles S, Lucchiari S, van Wittenberghe L, Vignaud A, Gjata B, Laforet P, Malfatti E, Mithieux G, Rajas F, P. Comi G, Ronzitti G, Mingozzi F (2017) Rescue of GSDIII phenotype with gene transfer requires liver- and muscle- targeted GDE expression. Molecular Therapy in press
