My research program focuses on modulating gene expression in vivo with the goal of developing a treatment for dominant genetic disease of muscle. My laboratory team seeks to employ an endogenous cell mechanism for fine-tuning gene expression at the post-transcriptional level, referred to as the RNA interference (RNAi). RNAi is triggered by processing of double-stranded RNA for sequence-specific gene silencing through direct base pairing with the target mRNA. Development of RNAi expression cassettes targeting mRNAs that lead to dominant genetic disease in muscle is a main focus of the laboratory. Both high expression level ubiquitous promoters and tissue-specific promoters are incorporated into adeno-associated virus vectors (AAV) to drive double-stranded RNA hairpin expression to carry out targeted RNAi. AAV-mediated tissue tropism combined with variations in muscle promoter-dependent expression are used to fine-tune therapeutic RNAi delivery. The RNAi expression vectors are tested both in cell cultures and in pre-clinical disease models, with the ultimate goal of preventing the effects of unwanted gene expression leading to human disease.