Ex Vivotransduced liver progenitor cells as a platform for gene therapy in mice
S Song, RP Witek, Y Lu, YK Choi, D Zheng… - Hepatology, 2004 - journals.lww.com
Allogeneic stem cell-based transplants may be limited by allograft rejection, as is seen with
conventional organ transplantation. One way to avert such a response is to use autologous
stem cells, but that may carry the risk of recurrence of the original disease, particularly in the
context of a genetic defect. We investigated the potential for gene modification of autologous
stem cells to avoid both problems, using recombinant adenoassociated virus vector
expressing human α1-antitrypsin in murine liver progenitor cells. We showed that …
conventional organ transplantation. One way to avert such a response is to use autologous
stem cells, but that may carry the risk of recurrence of the original disease, particularly in the
context of a genetic defect. We investigated the potential for gene modification of autologous
stem cells to avoid both problems, using recombinant adenoassociated virus vector
expressing human α1-antitrypsin in murine liver progenitor cells. We showed that …