John Landers, PhD
University of Massachusetts Chan Medical School
Development of Mutant Specific ASOs for TDP-43 and KIF5A
Gene-based therapeutics, including gene delivery and RNA interference (RNAi)-based therapies, have made tremendous advances over the past several years towards the development of therapeutics for ALS/FTD and numerous other diseases. The objective of this application is to develop potential therapeutics for ALS/FTD resulting from mutations in the TARDBP and KIF5A genes.