Krabbe Disease Successfully Treated With Gene Therapy in Preclinical Animal Model
Krabbe Disease Successfully Treated With Gene Therapy in Preclinical Animal Model https://pediatricsnationwide.org/wp-content/uploads/2021/02/AdobeStock_134232290-DNA-header-1024x575.gif 1024 575 Lauren Dembeck https://pediatricsnationwide.org/wp-content/uploads/2021/03/Dembeck_headshot.gifGene therapy shows promise for clinical benefit in demyelinating, neurodegenerative disease. Krabbe disease is an aggressive, incurable pediatric neurodegenerative disease caused by mutations in the galactosylceramidase (GALC) gene. Deficiency of the GALC protein activity leads to cytotoxic accumulation of a cellular metabolite called psychosine, which compromises normal turnover of myelin in the central and peripheral…