Ablating N‐acetylaspartate prevents leukodystrophy in a C anavan disease model
F Guo, P Bannerman, E Mills Ko, L Miers… - Annals of …, 2015 - Wiley Online Library
Annals of neurology, 2015•Wiley Online Library
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
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
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
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