Team:New York City/Integrated Practices

Integrated Practices

Last year, our team focused on developing a modified RNA protein replacement technology for treating Huntington’s Disease. We were motivated to delve further into research at the RNA level after collaborating with the Huntington’s Disease Society of America. Through our collaboration, we realized that current research in Huntington’s Disease was not selective in specifically targeting mutated Huntingtin RNA strands. Instead of targeting mutated strands only, research would try to target both endogenous and mutated strands, thereby stopping all production of the Huntingtin protein and resulting in undesired side effects. To resolve this, our team began developing modified RNA technology that would specifically target mutated Huntingtin mRNA strands through a specific toehold recognition and exchange process, allowing the mutated strands to be replaced by a corrected synthetic mRNA strand. This year our team is taking this research further by testing the efficacy of the modified RNA replacement technology in Huntingtin cell lines to determine whether our treatment decreases the levels of mutated Huntingtin protein.