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
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Hull, V., Wang, Y., Burns, T., Zhang, S., Sternbach, S., McDonough, J., Guo, F., & Pleasure, D. (2020). Antisense Oligonucleotide Reverses Leukodystrophy in Canavan Disease Mice. Annals of Neurology, 87(3), 480-485. https://doi.org/10.1002/ana.25674