Amelioration of Huntington's disease phenotype in astrocytes derived from iPSC-derived neural progenitor cells of Huntington's disease monkeys.

ABSTRACT: Huntington's disease (HD) is a devastating monogenic, dominant, hereditary, neurodegenerative disease. HD is caused by the expansion of CAG repeats in exon 1 of the huntingtin (HTT) gene, IT15, resulting in an expanded polyglutamine (polyQ) residue in the N-terminus of the HTT protein. HD is characterized by the accumulation of mutant HTT (mHTT) in neural and somatic cells. Progressive brain atrophy occurs initially in the striatum and extends to different brain regions with progressive decline in cognitive, behavioral and motor functions. Astrocytes are the most abundant cell type in the brain and play an essential role in neural development and maintaining homeostasis in the central nervous system (CNS). There is increasing evidence supporting the involvement of astrocytes in the develop

SUBMITTER: Cho IK 

PROVIDER: S-EPMC6428250 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature