Michela Milani, Cesare Canepari, Simone Assanelli, Simone Merlin, Ester Borroni, Francesco Starinieri, Mauro Biffi, Fabio Russo, Anna Fabiano, Desirèe Zambroni, Andrea Annoni, Luigi Naldini, Antonia Follenzi, Alessio Cantore
Lentiviral vectors (LV) are efficient vehicles for in vivo gene delivery to the liver. LV integration into the chromatin of target cells ensures their transmission upon proliferation, thus allowing potentially life-long gene therapy following a single administration, even to young individuals. The glycoprotein of the vesicular stomatitis virus (VSV.G) is widely used to pseudotype LV, as it confers broad tropism and high stability. The baculovirus-derived GP64 envelope protein has been proposed as an alternative for in vivo liver-directed gene therapy...
April 29, 2024: EMBO Molecular Medicine