Pharmacological Premature Termination Codon Readthrough of ABCB11 in Bile Salt Export Pump Deficiency: An In Vitro Study.

Rachida AmzalAlice ThébautMartine LapalusMarion AlmesBrigitte GrosseElodie MareuxMauricette Collado-HillyAnne Davit-SpraulLaure BidouOlivier NamyEmmanuel JacqueminEmmanuel M Gonzales

Published in: Hepatology (Baltimore, Md.) (2021)

This study constitutes a proof of concept for readthrough therapy in selected patients with PFIC2 with nonsense mutations.

Keyphrases

stem cells
replacement therapy
mesenchymal stem cells
bone marrow
chemotherapy induced