Scientific Advisory Board, Member

Neil Aronin  M.D.
Professor of Medicine and Cell Biology
Chief, Endocrinology and Metabolism
Co-Director, Neuro-Therapeutics Institute
University of Massachusetts Medical School

My research focuses on developing gene silencing for therapy in Huntington’s disease, an autosomal dominant neurodegenerative disease. The laboratory uses allele selective RNAi to silence the mutant huntingtin and preserve wild type huntingtin.  We explore potential therapies and neuronal pathophysiology attributable to the mutant huntingtin. We also study viral delivery of allele selective shRNAmir, with single stranded and self-complementary adeno-associated virus.  Thus, my laboratory studies next-generation therapeutics that will be applicable to a spectrum of autosomal dominant diseases.  Furthermore, we study mechanisms of pathogenesis in Huntington’s disease: mutant huntingtin mRNA kinetics, vesicle recycling and roles of proteins that interact with mutant huntingtin.