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

Sandra O Braz,Marlene M Morgado,Marta I Pereira,Ana C Monteiro,Olga Golonzhka,Matthew Jarpe,Pedro Brites,Monica M Sousa,Joana Nogueira-Rodrigues

Frontiers in molecular neuroscience（2023）

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摘要

Overall, our results support that ACY-738 has a neuroprotective effect in KD and should be considered as an add-on therapy combined with strategies targeting metabolic correction.

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关键词

axonal transport, HDAC6, Krabbe disease, leukodystrophy, microtubule stability, tubulin acetylation, Twitcher mice

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