Astroglial conditional Slc13a3 knockout is therapeutic in murine Canavan leukodystrophy

Autor:	Vanessa L. Hull, Yan Wang, Jennifer McDonough, Meina Zhu, Travis Burns, Najmah Al Ramel, Ali Dehghani, Fuzheng Guo, David Pleasure
Jazyk:	angličtina
Rok vydání:	2024
Předmět:	Neurosciences. Biological psychiatry. Neuropsychiatry RC321-571 Neurology. Diseases of the nervous system RC346-429
Zdroj:	Annals of Clinical and Translational Neurology, Vol 11, Iss 4, Pp 1059-1062 (2024)
Druh dokumentu:	article
ISSN:	2328-9503
DOI:	10.1002/acn3.52010
Popis:	Abstract Canavan disease is a leukodystrophy caused by ASPA mutations that diminish oligodendroglial aspartoacylase activity, and is characterized by markedly elevated brain concentrations of the aspartoacylase substrate N‐acetyl‐l‐aspartate (NAA) and by astroglial and intramyelinic vacuolation. Astroglia express NaDC3 (encoded by SLC13A3), a sodium‐coupled transporter for NAA and other dicarboxylates. Astroglial conditional Slc13a3 deletion in aspartoacylase‐deficient Canavan disease model mice (“CD mice”) reversed brain NAA elevation and improved motor function. These results demonstrate that astroglial NaDC3 contributes to brain NAA elevation in CD mice, and suggest that suppressing astroglial NaDC3 activity would ameliorate human Canavan disease.
Databáze:	Directory of Open Access Journals
Externí odkaz:	https://doaj.org/article/eeb55501af2040ab83c03837d9466fbf Zobrazit plný text záznamu View record in DOAJ Plný text ve formátu PDF Plný text ve formátu HTML
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