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Gene Therapy
Study results from Washington University, Medical Department broaden understanding of gene therapy
February 10th, 2009
"Neonatal intravenous injection of gammaretroviral vectors (gamma-RVs) with an intact long terminal repeat (LTR) and an internal liver promoter can result in long-term expression in liver cells and correction of mucopolysaccharidosis. Some expression also occurs in blood cells and brain, which likely derives from the LTR, and may contribute to clinical efficacy," investigators in the United States report. "The goal of this project was to determine whether neonatal gene therapy with an LTR-intact gamma-RV would induce tumors in mice. Fifty-one normal newborn C57BL/6 mice were injected intravenously at 10(10) transducing units/kg with a gamma-RV expressing canine...
Source: Cancer Weekly (2009-02-10)
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