Medical News Article on Gene Therapy

Studies from University of Pennsylvania Reveal New Findings on Gene Therapy

2012 MAY 8 -- Current study results from the report, "Sustained correction of OTC deficiency in spf('ash) mice using optimized self-complementary AAV2/8 vectors," have been published. According to the authors of recent research from Philadelphia, Pennsylvania, "Ornithine transcarbamylase deficiency (OTCD) is the most common inborn error of urea synthesis. Complete OTCD can result in hyperammonemic coma in the neonatal period, which can rapidly become fatal."

"Current acute therapy involves dialysis; chronic therapy involves the stimulation of alternate nitrogen clearance pathways; and the only curative approach is liver transplantation. Adeno-associated...

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