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

Internet

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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