Uncoupling N-acetylaspartate from brain pathology: implications for Canavan disease gene therapy

N-Acetylaspartate (NAA) is the second most abundant organic metabolite in the brain, but its physiological significance remains enigmatic. Toxic NAA accumulation appears to be the key factor for neurological decline in Canavan disease—a fatal neurometabolic disorder caused by deficiency in the NAA-d...

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Detalles Bibliográficos
Autores principales: von Jonquieres, Georg, Spencer, Ziggy H. T., Rowlands, Benjamin D., Klugmann, Claudia B., Bongers, Andre, Harasta, Anne E., Parley, Kristina E., Cederholm, Jennie, Teahan, Orla, Pickford, Russell, Delerue, Fabien, Ittner, Lars M., Fröhlich, Dominik, McLean, Catriona A., Don, Anthony S., Schneider, Miriam, Housley, Gary D., Rae, Caroline D., Klugmann, Matthias
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2017
Materias:
Original Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5756261/
https://www.ncbi.nlm.nih.gov/pubmed/29116375
http://dx.doi.org/10.1007/s00401-017-1784-9

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5756261/
https://www.ncbi.nlm.nih.gov/pubmed/29116375
http://dx.doi.org/10.1007/s00401-017-1784-9

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