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Metabolic acetate therapy improves phenotype in the tremor rat model of Canavan disease

Genetic mutations that severely diminish the activity of aspartoacylase (ASPA) result in the fatal brain dysmyelinating disorder, Canavan disease. There is no effective treatment. ASPA produces free acetate from the concentrated brain metabolite, N-acetylaspartate (NAA). Because acetyl coenzyme A is...

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Detalhes bibliográficos
Main Authors: Arun, Peethambaran, Madhavarao, Chikkathur N., Moffett, John R., Hamilton, Kristen, Grunberg, Neil E., Ariyannur, Prasanth S., Gahl, William A., Anikster, Yair, Mog, Steven, Hallows, William C., Denu, John M., Namboodiri, Aryan M. A.
Formato: Artigo
Idioma:Inglês
Publicado em: Springer Netherlands 2010
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2877317/
https://ncbi.nlm.nih.gov/pubmed/20464498
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-010-9100-z
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