Abstract
Marked elevation in the brain concentration of N-acetyl-L-aspartate (NAA) is a characteristic feature of Canavan disease, a vacuolar leukodystrophy resulting from deficiency of the oligodendroglial NAA-cleaving enzyme aspartoacylase. We now demonstrate that inhibiting NAA synthesis by intracisternal administration of a locked nucleic acid antisense oligonucleotide to young-adult aspartoacylase-deficient mice reverses their pre-existing ataxia and diminishes cerebellar and thalamic vacuolation and Purkinje cell dendritic atrophy. Ann Neurol 2020;87:480–485.
Original language | English (US) |
---|---|
Pages (from-to) | 480-485 |
Number of pages | 6 |
Journal | Annals of Neurology |
Volume | 87 |
Issue number | 3 |
DOIs | |
State | Published - Mar 1 2020 |
ASJC Scopus subject areas
- Neurology
- Clinical Neurology