Antisense Oligonucleotide Reverses Leukodystrophy in Canavan Disease Mice.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 31925837.
- Also identified by DOI 10.1002/ana.25674 and PMC identifier 8523037.
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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.
Medical subject headings
- Aspartic Acid
- Canavan Disease
- Oligonucleotides, Antisense