Testing the efficacy of mRNA displacement technique in huntingtin cell lines to treat Huntington's Disease
Huntington's Disease (HD) is a genetic disorder that results in the breakdown of nerve cells in the brain. Although the disease affects the whole brain, the basal ganglia, which plays a critical role in motor functions and behavior, is most severely affected. Thus, symptoms of HD include involuntary movements, personality changes, and cognitive problems. Over time, the effects of HD is extremely debilitating, eventually rendering a patient unable to speak, walk, or even reason.
HD is a result of a specific mutation in the huntingtin (HTT) gene, which results in a faulty production of the HTT protein, mHTT. The mutation is a trinucleotide repeat of the codon CAG, where 40 or more will lead to the expression of HD.
The most common form of HD is adult-onset, affecting people between their 30s and 40s. Many people form families and have children by the time they are diagnosed with HD, which automatically gives their children a 50% chance of inheriting the disease. HD affects about 1 in every 10,000 people worldwide.
Our goal is to develop a cure to Huntington's Disease by employing RNA strand displacement technology to target erroneous mRNA within the affected cells and replace it with the corrected RNA strand for proper protein synthesis.