- Bannerman, Peter;
- Mills Ko, Emily;
- Miers, Laird;
- Xu, Jie;
- Burns, Travis;
- Li, Shuo;
- Freeman, Ernest;
- McDonough, Jennifer;
- Pleasure, David;
- Guo, Fuzheng
Canavan disease is caused by inactivating ASPA (aspartoacylase) mutations that prevent cleavage of N-acetyl-L-aspartate (NAA), resulting in marked elevations in central nervous system (CNS) NAA and progressively worsening leukodystrophy. We now report that ablating NAA synthesis by constitutive genetic disruption of Nat8l (N-acetyltransferase-8 like) permits normal CNS myelination and prevents leukodystrophy in a murine Canavan disease model.