Nature Communications (Dec 2019)

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

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

DOI
https://doi.org/10.1038/s41467-019-13614-3
Journal volume & issue
Vol. 10, no. 1
pp. 1 – 13

Abstract

Read online

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.