Gene Therapy
Findings from University Medical Center, Division of Medical Genetics Provide New Insights into Gene Therapy
Published in Gene Therapy Weekly, February 17th, 2011
2011 FEB 17 -- New research, 'Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease,' is the subject of a report. According to recent research from the United States, "Infantile Pompe disease progresses to a lethal cardiomyopathy in absence of effective treatment. Enzyme-replacement therapy (ERT) with recombinant human acid alpha-glucosidase (rhGAA) has been effective in most patients with Pompe disease, but efficacy was reduced by high-titer antibody responses."
"Immunomodulatory gene therapy with a low dose adeno-associated virus (AAV) vector (2 x 10(10) particles) containing a...
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Source: Gene Therapy Weekly (2011-02-17)
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