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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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Detalles Bibliográficos
Autores principales: 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: Texto
Lenguaje:English
Publicado: Springer Netherlands 2010
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2877317/
https://www.ncbi.nlm.nih.gov/pubmed/20464498
http://dx.doi.org/10.1007/s10545-010-9100-z