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(Reference retrieved automatically from SciELO through information on FAPESP grant and its corresponding number as mentioned in the publication by the authors.)

Combating oncogene activation associated with retrovirus-mediated gene therapy of X-linked severe combined immunodeficiency

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B.E. Strauss ; E. Costanzi-Strauss
Total Authors: 2
Document type: Journal article
Source: Brazilian Journal of Medical and Biological Research; v. 40, n. 5, p. 601-613, 2007-03-14.

A successful gene therapy clinical trial that also encountered serious adverse effects has sparked extensive study and debate about the future directions for retrovirus-mediated interventions. Treatment of X-linked severe combined immunodeficiency with an oncoretrovirus harboring a normal copy of the gc gene was applied in two clinical trials, essentially curing 13 of 16 infants, restoring a normal immune system without the need for additional immune-related therapies. Approximately 3 years after their gene therapy, tragically, 3 of these children, all from the same trial, developed leukemia as a result of this experimental treatment. The current understanding of the mechanism behind this leukemogenesis involves three critical and cooperating factors, i.e., viral integration, oncogene activation, and the function of the therapeutic gene. In this review, we will explore the causes of this unwanted event and some of the possibilities for reducing the risk of its reoccurrence. (AU)

FAPESP's process: 00/12156-5 - Strategies of transcriptional regulation in retrovirus production and expression
Grantee:Bryan Eric Strauss
Support Opportunities: Research Grants - Young Investigators Grants