Utility of Engineered Human Neural Stem Cells (NSCs) Expressing Varying Exon 1 HTT Fragments to Study Huntington's Disease


Huntington's disease (HD) is a rare, progressive neurodegenerative disorder that usually presents itself with symptoms of motor and cognitive decline between the ages of 30 and 50. It is most commonly caused by an expansion mutation in the CAG trinucleotide repeat within exon 1 of the HTT gene which codes for the protein huntingtin and is inherited in an autosomal dominant manner.