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

Canavan disease (CD) is a debilitating and lethal leukodystrophy caused by mutations in the aspartoacylase (ASPA) gene and the resulting defect in N-acetylaspartate (NAA) metabolism in the CNS and peripheral tissues. Recombinant adeno-associated virus (rAAV) has the ability to cross the blood-brain...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:JCI Insight
Päätekijät: Gessler, Dominic J., Li, Danning, Xu, Hongxia, Su, Qin, Sanmiguel, Julio, Tuncer, Serafettin, Moore, Constance, King, Jean, Matalon, Reuben, Gao, Guangping
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: American Society for Clinical Investigation 2017
Aiheet:
Research Article
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC5291725/
https://ncbi.nlm.nih.gov/pubmed/28194442
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.90807
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