Ex vivo transduced liver progenitor cells as a platform for gene therapy in mice

Hepatology : Official Journal of the American Association for the Study of Liver Diseases
Sihong SongByron E Petersen

Abstract

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 alpha1-antitrypsin in murine liver progenitor cells. We showed that recombinant adenoassociated virus 1 was the most efficient vector for liver progenitor cell transduction among five different serotypes of recombinant adenoassociated virus vectors. Ex vivo infected green fluorescent protein-positive liver progenitor cells from C57BL/6 mice with recombinant adenoassociated virus 1-vector-expressing human alpha1 antitrypsin were transplanted into the liver of monocrotaline-treated and partial-hepatectomized C57BL/6 recipients. Using green fluorescent protein as a donor marker, we were able to determine that at 18 weeks after transplantation, approximately 40% to 50% of the regenerated liver was green fluorescent protein positive. In addition, transgene expressi...Continue Reading

Citations

Oct 5, 2010·Molecular Imaging and Biology : MIB : the Official Publication of the Academy of Molecular Imaging·Randall McClellandEdward Hsu
Nov 27, 2008·Molecular Therapy : the Journal of the American Society of Gene Therapy·Xiaoming LiuJohn F Engelhardt
Jun 17, 2010·Molecular Therapy : the Journal of the American Society of Gene Therapy·Hong LiSihong Song
Jul 27, 2005·Proceedings of the National Academy of Sciences of the United States of America·Leah SantatSaswati Chatterjee
Oct 7, 2009·Proceedings of the National Academy of Sciences of the United States of America·Yuanqing Lu, Sihong Song
May 3, 2013·The Journal of Clinical Investigation·Claus Kordes, Dieter Häussinger
Jul 18, 2009·Expert Opinion on Therapeutic Targets·Michail Papoulas, Stamatios Theocharis
Jun 5, 2007·The Journal of Surgical Research·Montserrat CaballeroJeffrey H Fair
Aug 25, 2005·IUBMB Life·Vance B Matthews, George C Yeoh
Feb 1, 2006·Hepatology : Official Journal of the American Association for the Study of Liver Diseases·David A ShafritzMariana D Dabeva
Sep 29, 2015·Human Gene Therapy·Joanna WozniakJoanna Chorostowska-Wynimko
Jan 30, 2007·Hepatology : Official Journal of the American Association for the Study of Liver Diseases·George C T YeohVance B Matthews
Nov 17, 2009·Journal of Hepatology·Laurent DolléAlbert Geerts
Dec 21, 2010·Journal of Hepatology·Hong LiSihong Song
Jan 12, 2008·Biochimica Et Biophysica Acta·Michael Oertel, David A Shafritz
Sep 25, 2016·Stem Cell Research & Therapy·Panpan CenLanjuan Li

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