Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model

por Nicholas D. Weber, Leticia Odriozola, Javier Martínez-García, Veronica Ferrer, Anne Douar, Bernard Bénichou, Gloria González-Aseguinolaza, Cristian Smerdou
Publicado 2019

Lentiviral vectors can be used for full-length dystrophin gene therapy

por John R. Counsell, Zeinab Asgarian, Jinhong Meng, Veronica Ferrer, Conrad A. Vink, Steven J. Howe, Simon N. Waddington, Adrian J. Thrasher, Francesco Muntoni, Jennifer E. Morgan, Olivier Danos
Publicado 2017