| First Author | Hull V | Year | 2020 |
| Journal | Ann Neurol | Volume | 87 |
| Issue | 3 | Pages | 480-485 |
| PubMed ID | 31925837 | Mgi Jnum | J:351543 |
| Mgi Id | MGI:7627271 | Doi | 10.1002/ana.25674 |
| Citation | Hull V, et al. (2020) Antisense Oligonucleotide Reverses Leukodystrophy in Canavan Disease Mice. Ann Neurol 87(3):480-485 |
| abstractText | 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. |
