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Stem Cells International
Volume 2011, Article ID 393698, 8 pages
Review Article

Phase I/II Clinical Trials Using Gene-Modified Adult Hematopoietic Stem Cells for HIV: Lessons Learnt

1Center for Clinical AIDS Research and Education (CARE Center), University of California-Los Angeles, Los Angeles, CA 90035, USA
2Departments of Medicine and of Microbiology, Immunology and Molecular Genetics, University of California-Los Angeles, Los Angeles, CA 90095, USA
3SpanCyte Pty Ltd., B.O. Box 960, Sydney, Leichhardt, NSW 2040, Australia
4Faculty of Medicine and Centre for Applied Medical Research, St. Vincent's Hospital, The University of New South Wales, Kensington, Sydney, NSW 2052, Australia

Received 10 January 2011; Accepted 15 March 2011

Academic Editor: Dominique Bonnet

Copyright © 2011 Ronald T. Mitsuyasu et al. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.


Gene therapy for individuals infected with HIV has the potential to provide a once-only treatment that will act to reduce viral load, preserve the immune system, and mitigate cumulative toxicities associated with highly active antiretroviral therapy (HAART). The authors have been involved in two clinical trials (phase I and phase II) using gene-modified adult hematopoietic stem cells (HSCs), and these are discussed as prototypic trials within the general field of HSC gene therapy trials for HIV. Taken as a group these trials have shown (i) the safety of both the procedure and the anti-HIV agents themselves and (ii) the feasibility of the approach. They point to the requirement for (i) the ability to transduce and infuse as many as possible gene-containing HSC and/or (ii) high engraftment and in vivo expansion of these cells, (iii) potentially increased efficacy of the anti-HIV agent(s) and (iv) automation of the cell processing procedure.