Virology
Gene therapy corrects Fabry disease in neonatal mice
Published in Gene Therapy Weekly, February 24th, 2005
"Successful therapy for many inherited disorders could be improved if the intervention were initiated early. This is especially true for lysosomal storage disorders. Earlier intervention may allow metabolic correction to occur before lipid buildup has irreversible consequences and/or before the immune system mounts limiting responses," investigators in Canada report.
"We have been developing gene therapy to treat lysosomal storage disorders, especially Fabry disease. We describe studies directed toward metabolic correction in neonatal animals mediated by recombinant lentiviral vectors," wrote M....
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Source: Gene Therapy Weekly (2005-02-24)
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