Silencing Huntington's chorea: Is RNA Interference a Potential Cure?

Huntington's chorea is a genetic disorder characterized by cognitive, motor and psychiatric impairments. It is caused by a dominant mutation on chromosome 4 featuring the multiplication of a portion of the gene in which the codon CAG occurs. CAG codes for the amino acid, glutamine and so the Huntingtin protein (htt) features an excessive expression of glutamine. The abnormal protein htt is prone to accumulate in neurons eventually resulting in brain cell death and the consequent progressive symptoms of the disease. Therapies for Huntington's chorea include targeting the symptoms, the progression, and the cause of the disease, but no treatment has as yet been found to be completely effective. After describing the symptoms, genetic and proteomic basis of Huntington's chorea, this paper discusses a new approach to treatment of Huntington's chorea, RNA interference. The problems, limitations, and the benefits of RNA interference will be examined, along with avenues for overcoming the limitations of gene therapy with a transposition system, such as Sleeping Beauty. This review suggests that although substantial amount of research is still necessary before RNA interference treatment is successful, success will open new avenues for treating other degenerative nervous system diseases.

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