Identification of small molecular inhibitors targeting ATXN1-CIC interaction for alleviating neurological pathogenesis in SCA1 cell model
Jeewoo Kim, Minsung Kang, Doyoun Kim · FENS Forum 2024
The spinocerebellar ataxia type1 (SCA1) is a progressive rare neurodegenerative disease commonly associated with impairment of motor coordination and movement control. The cerebellar Purkinje neuron, spinal cord, and its connective tissues are the predominant affecting brain regions. The molecular pathology underlying SCA1 is known as accumulation of poly-glutamine (polyQ) expansion in ATXN1 gene. The interaction between ATXN1 and CIC plays a pivotal role in the pathogenesis of SCA1, particularly in cerebellar Purkinje cells. Recent findings have demonstrated phenotypic rescue through a mutant
ePoster · Neuroscience