Article

HDAC-6 inhibition ameliorates the early neuropathology in a mouse model of Krabbe disease

2023-05-01

Abstract excerpt

In Krabbe disease (KD), mutations in β-galactosylceramidase (GALC), a lysosomal enzyme responsible for the catabolism of galactolipids, lead to the accumulation of its substrates galactocerebroside and psychosine. This neurologic condition is characterized by a severe and progressive demyelination together with neuron-autonomous defects and degeneration. Twitcher mice mimic the infantile form of KD, which is the m...

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Literature Corpus work: 5eba046d-90c2-5d32-bd56-decdbae9d6b4
DOI: 10.1101/2023.05.01.538924

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