Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model
oleh: Nicholas D. Weber, Leticia Odriozola, Javier Martínez-García, Veronica Ferrer, Anne Douar, Bernard Bénichou, Gloria González-Aseguinolaza, Cristian Smerdou
| Format: | Article |
|---|---|
| Diterbitkan: | Nature Portfolio 2019-12-01 |
Deskripsi
Progressive familial intrahepatic cholestasis type 3 is a disease caused by mutations in the gene encoding the multidrug resistance protein 3, and has limited treatment options. Here they show that adeno-associated virus mediated gene therapy prevents disease progression in a mouse disease model.