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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...
Tallennettuna:
| Julkaisussa: | JCI Insight |
|---|---|
| Päätekijät: | , , , , , , , , , |
| Aineistotyyppi: | Artigo |
| Kieli: | Inglês |
| Julkaistu: |
American Society for Clinical Investigation
2017
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| Aiheet: | |
| 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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