The Effect of Donepezil Hydrochloride in the Twitcher Mouse Model of Krabbe Disease.

ABSTRACT: Krabbe disease (KD) is a rare demyelinating disorder characterized by demyelination caused by mutations in the GALC gene, resulting in toxic accumulation of psychosine. Psychosine has been identified as detrimental to oligodendrocytes, leading to demyelination through diverse hypothesized pathways. Reducing demyelination is essential to maintain neurological function in KD; however, therapeutic interventions are currently limited. Acetylcholinesterase inhibitors (AChEi) are commonly used for symptomatic management of Alzheimer's Disease and are suggested to have potential disease-modifying effects, including regulating myelin state. In particular, donepezil, an AChEi, has demonstrated promising effects in cellular and animal models, including promotion of the expression of myelin-related g

SUBMITTER: Papakyriakopoulou P 

PROVIDER: S-EPMC11496341 | biostudies-literature | 2024 Nov

REPOSITORIES: biostudies-literature