The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) awarded a $195,625 Project Grant under the Diabetes, Digestive, and Kidney Diseases Extramural Research program (CFDA 93.847) to Emory University to conduct preclinical studies on the use of gene therapy to treat classic galactosemia, a rare and potentially lethal genetic disease. The 2-year study will test whether a single dose of an adeno-associated viral vector delivering the human GALT gene can prevent long-term complications in a GALT-null rat model of classic galactosemia. The results of this work will help inform the development of future clinical trials for this disorder, which currently lacks an effective treatment beyond dietary restriction.
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