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

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 (<i>GALC</i&g...

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Bibliographic Details
Main Authors: Aimee R. Herdt, Hui Peng, Dennis W. Dickson, Todd E. Golde, Elizabeth A. Eckman, Chris W. Lee
Format: Article
Language:English
Published: MDPI AG 2023-07-01
Series:Genes
Subjects:
Krabbe disease
galactosylceramidase
psychosine
twitcher
adeno-associated virus serotype 1
gene therapy
Online Access:https://www.mdpi.com/2073-4425/14/8/1517

Internet

https://www.mdpi.com/2073-4425/14/8/1517

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