Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases

Federica Esposito, Fabio Dell'Aquila, Manuel Rhiel, Stefano Auricchio, Kay Ole Chmielewski, Geoffroy Andrieux, Rita Ferla, Paula Sureda Horrach, Arjun Padmanabhan, Roberto Di Cunto, Simone Notaro, Manel Llado Santeularia, Melanie Boerries, Margherita Dell'Anno, Edoardo Nusco, Agnese Padula, Sofia Nutarelli, Tatjana I. Cornu, Nicolina Cristina Sorrentino, Pasquale PiccoloIvana Trapani, Toni Cathomen, Alberto Auricchio

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Biochemistry, Genetics and Molecular Biology