Development of therapeutic strategies and biomarkers for metabolic diseases  

Most metabolic and genetic disorders cause patients with these disorders to have unmet medical needs. Therefore, it is crucial to make progress in developing novel therapies and biomarkers that can be used to monitor the clinical course of patients with these disorders over time. Our lab, in collaboration with others, is interested in the following: 1) developing small molecule drugs (agonist or antagonist drugs) that target a specific gene and reverse the phenotype and 2) using an ASO-mediated exon-skipping approach that targets storage disorders at the mRNA level and edits out-of-frame transcripts to produce in-frame ones. This approach is an emerging approach to drug development for treating incurable monogenic diseases.