Article
Adeno-associated virus-mediated aspartoacylase gene transfer to the brain of knockout mouse for canavan disease.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 May 2003
Matalon Reuben, Surendran Sankar, Rady Peter L, Quast Michael J, Campbell Gerald A, Matalon Kimberlee M, Tyring Stephen K, Wei Jingna, Peden Carmen S, Ezell E L, Muzyczka Nicholas, Mandel Ronald J
Abstract excerpt
Canavan disease (CD) is an autosomal recessive leukodystrophy caused by deficiency of aspartoacylase (ASPA). Deficiency of ASPA leads to elevation of N-acetyl-L-aspartic acid (NAA) in the brain and urine. To explore the feasibility of gene transfer to replace ASPA in CD, we generated a knockout mouse and constructed an AAV vector that encodes human ASPA cDNA (hASPA) followed by green fluorescent protein (GFP)...
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