Treatment discovered for deadly childhood disease Researchers have discovered that a treatment involving enzyme replacement therapy dramatically reduces the risk of death in children with Pompe disease, a rare genetic disorder in which most children die before their first birthday. view more (2006-12-07)
One shot of gene therapy spreads through brain in animal study By targeting a site in a mouse brain well connected to other areas, researchers successfully delivered a beneficial gene to the entire brain-after one injection of gene therapy. If these results in animals can be realized in people, researchers may have a potential method for gene therapy to treat a host of rare but devastating congenital human... view more... (2007-10-09)
