Integrated Practices
Our team originally focused on developing a modified RNA protein replacement technology for Huntington’s under the guise that the Huntington’s phenotype was caused by the loss of function of the Huntingtin protein due to polyglutame repeats. During our continued research thought the project, we discovered that there are 2 copies of HTT and simply up regulating the good copy as long as disease state HTT protein is present. Thus we decided it may be better to change the focus of our project from validating toe-hold displacement to expression of human endogenous HTT mRNA so that others could use it do develop RNAi style therapies. However, after we collaborated with the Huntington’s Disease Society of America, we discovered that the current research in treatment both targeted the endogenous and normal RNA strands, essentially stopping the production of all Huntington protein. Thus, our team is once again focused on validating toehold exchange to affect HTT to replace the mRNA targeted by RNAi with a corrected synthetic mRNA.
