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Gene Therapy

Study Results from University of Florida Broaden Understanding of Gene Therapy

Published in Gene Therapy Weekly, September 22nd, 2011

2011 SEP 22 -- A new study, "Ex vivo transduction and transplantation of bone marrow cells for liver gene delivery of alpha1-antitrypsin," is now available. According to the authors of recent research from Gainesville, Florida, "Adult stem cell-based gene therapy holds several unique advantages including avoidance of germline or other undesirable cell transductions. We have previously shown that liver progenitor (oval) cells can be used as a platform for liver gene delivery of human alpha1-antitrypsin (hAAT)."

"However, this cell source cannot be used in humans for autologous transplantation. In the present study, we tested the feasibility of bone marrow...

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