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Redirecting N-acetylaspartate metabolism in the central nervous system normalizes myelination and rescues Canavan disease

8 Feb 2017

Abstract excerpt

-acetylaspartate (NAA) metabolism in the CNS and peripheral tissues. Recombinant adeno-associated virus (rAAV) has the ability to cross the blood-brain barrier and widely transduce the CNS. We developed a rAAV-based and optimized gene replacement therapy, which achieves early, complete, and sustained rescue of the lethal disease phenotype in CD mice. Our treatment results in a super-mouse phenotype, increasing...

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PMID: 28194442
DOI: 10.1172/jci.insight.90807

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