Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapy.
Keeler AM, Conlon T, Walter G, Zeng H, Shaffer SA, Dungtao F, Erger K, Cossette T, Tang Q, Mueller C, Flotte TR. Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapy. Mol Ther. 2012 Jun; 20(6):1131-8.