Team:EpiphanyNYC/Description

Tackling Huntington’s Disease with RNA Strand Displacement

Huntington's disease (HD) is an autosomal dominant genetic disorder that causes the breakdown of nerve cells in the brain and currently has no cure, which is why we’re aiming to find one! HD, which is usually adult-onset, is particularly devastating because it includes symptoms such as amnesia, involuntary movements, and physical incoordination, usually giving a patient a lifespan of only ten years after onset. The primary cause of this disease is a trinucleotide repeat of CAG in the huntingtin gene, where a repeat of 40 or more can cause the manifestation of the disease. Our goal is to create a synthetic RNA strand displacement technology that consists of the corrected RNA strand and a “guide” strand. The guide strand will bond with endogenous, faulty messenger RNA and release the corrected RNA strand for proper protein synthesis of the huntingtin protein, while disposing of the repeating CAG sequence. Ultimately, once such technologies are developed to overcome endocytosis and blood brain barrier traversal, an injectable cure can be produced. Wish us luck!

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