Gene editing by crispr/cas9 for treatment of huntington disease
Huntington's disease (HD) is an autosomal dominant full-penetrating neurodegenerative disorder and mutant gene is located in the short arm of chromosome 4, which encodes huntingtin protein and leads to degeneration of the basal ganglia in the brain, causing motor disorders and eventually communication. Patient`s DNA whit HD shows different number of cytosine-adenine-guanine trinucleotide (CAG) repeats, which indicates whether or not the person has the genetic defect of the disease.