About the Lab
Our research is focused on the regulation of lipid and lipoprotein metabolism. The laboratory is a hybrid of basic science and translational projects, aimed at understanding and treating cardiometabolic disease.
Basic science projects focus on genes that are associated with lipid levels and cardiovascular risk in humans. These studies rely heavily upon genetically engineered animal models, and seek to provide more in depth information about the molecular pathways involved, and the physiology of lipid metabolism in the context of the whole organism. A key area of interest is genes that regulate cholesterol synthesis and transport. Results from these studies will improve our understanding of rare and common genetic variants contributing to cardiovascular disease risk in humans, and help identify new therapeutic targets.
Translational projects in the lab involve the development of novel gene delivery vectors and applications. We use Adeno-Associated Viral (AAV) vectors to manipulate gene expression in animal models, and to perform preclinical gene therapy for lipid and metabolic disorders. AAV vectors are the premier vehicle for tissue-directed gene therapy in humans, with over 100 successful clinical trials completed to date. Gene therapy is coming of age, and major clinical advances are expected in the next few years. Our goal is to develop novel therapeutic approaches to treat rare metabolic diseases through overexpression, gene knockdown, and precise genome editing.
Key Areas of Interest
- Genetic factors influencing de novo cholesterol synthesis and trafficking
- Arv1, a putative ER-Golgi lipid transporter and its role in mammalian energy homeostasis
- Methods for somatic overexpression, knockdown and genome editing with viral vectors
- Novel gene therapy vectors for the treatment of rare lipid disorders
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