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. CRISPR/CAS9 system is a gene editing tool aimed at repairing a defective gene in functionality and aims to improve the picture of mainly monogenic diseases, having three methodologies, deletion, insertion and knockout, its use can be performed in Huntington's disease, with the deletion of the extra repeats of CAG trinucleotides. The aim of this study was to review the use of CRISPR/CAS9 therapy in the treatment of Huntington's disease. For development of this study, methodology was based in literature review were used scientific papers in indexed databases like PubMed and Scielowith the following descriptors according to DeCS: CRISPR, Huntington's disease, monogenic diseases. Studies indicate that the use of CRISPR/Cas9 technology has the potential to treat Huntington's disease without causing off-targets, although few clinical studies have been conducted.