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JCI table of contents: March 23, 2009
Date:3/23/2009

UTHOR CONTACT:
David M. Thomas
Peter MacCallum Cancer Centre, Melbourne, Victoria, Australia.
Phone: 613-9656-1111; Fax: 613-9656-1411; E-mail: david.thomas@petermac.org.

View the PDF of this article at: https://www.the-jci.org/article.php?id=37175

ACCOMPANYING COMMENTARY
TITLE: Wnt therapy for bone loss: golden goose or Trojan horse?

AUTHOR CONTACT:
Greg H. Enders
Fox Chase Cancer Center, Philadelphia, Pennsylvania, USA.
Phone: (215) 214-3956; Fax: (215) 728-4333; E-mail: Greg.Enders@fccc.edu.

View the PDF of this article at: https://www.the-jci.org/article.php?id=38973


GENE THERAPY: Making gene therapy safer using self-inactivating LTRs

Several patients in gene therapy clinical trials have developed leukemia as a result of their treatment. The underlying cause of leukemia is thought to be that the viral vectors used to carry the therapeutic gene into cells (gamma-RVs) integrate into the genome of the cells disrupting the natural control of cancer-associated genes (a process known as insertional mutagenesis). By analyzing specific elements of gamma-RVs and another type of viral vector, LVs, in a tumor-prone mouse model, a team of researchers, at San Raffaele-Telethon Institute for Gene Therapy, Italy, has now provided evidence that LVs are substantially less likely to cause insertional mutagenesis and tumors than gamma-RVs. Further, they found that modifying an element (known as the LTR) of both LVs and gamma-RVs such that it is self-inactivating (SIN) further improved safety. The authors therefore conclude that SIN viral vectors should be the preferred choice in future gene therapy trials.

In an accompanying commentary, Ute Modlich and Christo
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Contact: Karen Honey
press_releases@the-jci.org
215-573-1850
Journal of Clinical Investigation
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