Article
Antisense Oligonucleotide Reverses Leukodystrophy in Canavan Disease Mice.
Annals of neurology - 1 Mar 2020
Hull Vanessa, Wang Yan, Burns Travis, Zhang Sheng, Sternbach Sarah, McDonough Jennifer, Guo Fuzheng, Pleasure David
Abstract excerpt
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...
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