Ablating N ‐acetylaspartate prevents leukodystrophy in a C anavan disease model

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. Ann Neurol 2015;77:884–888