Translating Discovery into Treatment

Upon discovery of novel gene targets for therapeutic intervention in degenerative disease or brain injury, NTI members are uniquely positioned to rapidly translate new knowledge into treatment. Two of the most promising approaches for intervention to date are RNAi-based therapeutics and gene therapy—areas in which the University of Massachusetts Medical School (UMMS) is world leader. The UMMS RNA Therapeutics Institute ( houses faculty who are experts in RNA biology and whose primary goals are to develop novel approaches to RNA delivery and targeting of disease genes for the treatment of a broad range of human diseases. The UMMS Gene Therapy Center (GTC) is harnessing the power of human viral vectors for gene transfer into patients, with the hope of replacing or modifying disease-related gene function. NTI members can therefore partner directly with RTI and GTC scientists working on the delivery of new therapies to patients, and move as quickly as possible from discoveries at the bench to novel therapies for patients.