Antisense Oligonucleotide Reverses Leukodystrophy in Canavan Disease Mice

Vanessa Hull, Yan Wang, Travis Burns, Sheng Zhang, Sarah Sternbach, Jennifer McDonough, Fuzheng Guo, David Pleasure

Research output: Contribution to journalArticlepeer-review

13 Scopus citations


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 languageEnglish (US)
Pages (from-to)480-485
Number of pages6
JournalAnnals of Neurology
Issue number3
StatePublished - Mar 1 2020

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology


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