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Brain Targeted AAV1-GALC Gene Therapy Reduces Psychosine and Extends Lifespan in a Mouse Model of Krabbe Disease

Krabbe disease (KD) is a progressive and devasting neurological disorder that leads to the toxic accumulation of psychosine in the white matter of the central nervous system (CNS). The condition is inherited via biallelic, loss-of-function mutations in the galactosylceramidase (GALC) gene. To rescue...

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Detalles Bibliográficos
Autores principales:	Herdt, Aimee R., Peng, Hui, Dickson, Dennis W., Golde, Todd E., Eckman, Elizabeth A., Lee, Chris W.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2023
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10454254/ https://www.ncbi.nlm.nih.gov/pubmed/37628569 http://dx.doi.org/10.3390/genes14081517

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