Gene Therapy
Reports summarize gene therapy study results from Washington University, Department of Internal Medicine
Published in Gene Therapy Weekly, June 21st, 2007
New investigation results, "Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation," are detailed in a study published in Molecular Therapy. "Mucopolysaccharidosis I (MPS I) is caused by deficient alpha-L-iduronidase (IDUA) activity and results in the accumulation of glycosaminoglycans and multisystemic disease. Gene therapy could program cells to secrete mannose 6-phosphate-modified IDUA, and enzyme in blood could be taken up by other cells," investigators in the United States report.
"Neonatal retroviral vector (RV)-mediated gene therapy has been shown to reduce the manifestations of murine MPS I;...
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Source: Gene Therapy Weekly (2007-06-21)
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