Redirecting N-acetylaspartate metabolism in the central nervous system normalizes myelination and rescues Canavan disease.
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DOI: 10.1172/jci.insight.90807
Abstract: Canavan disease (CD) is a debilitating and lethal leukodystrophy caused by mutations in the aspartoacylase (ASPA) gene and the resulting defect in N-acetylaspartate (NAA) metabolism in the CNS and peripheral tissues. Recombinant adeno-associated virus (rAAV)… read more here.
Keywords: disease; metabolism central; redirecting acetylaspartate; canavan disease ... See more keywords