Sverdrup Fran 2018

Fran Sverdrup, Ph.D.
Associate Professor

Epigenetic control of gene expression in facioscapulohumeral muscular dystrophy (FSHD) and the discovery of drugs to modulate this process.

Office: DRC 417
Voice: (314) 977-5189

Research Interests

My lab is focused on drug discovery in human genetic diseases as well as infectious diseases. We perform target identification and validation, drug screening, and preclinical evaluation of drug candidates. Our major current project targets facioscapulohumeral muscular dystrophy (FSHD), one of the most common forms of muscular dystrophy for which there is no treatment. Our goals are to identify druggable pathways that modulate expression of the toxic DUX4 gene that is responsible for FSHD and translate those finding into potential therapies.

Our approach is to perform high throughput screening of chemical libraries to identify compounds intended to epigenetically suppress the DUX4 gene. We evaluate new compounds by using biochemical and cell based assays to explore mechanisms and map pathways that lead to DUX4 expression. We are currently advancing three exciting classes of drugs that turn off DUX4 expression, including a robust lead optimization program that involves close collaboration with our medicinal chemistry colleagues. We are also utilizing a mouse xenograft model of human FSHD gene regulation to establish pharmacokinetic/pharmacodynamic (PK/PD) relationships of drug candidates. We have established a key collaboration with a pharmaceutical partner to advance one of these into human clinical trials.

A second interest is in anti-infectives research with recent programs targeting malaria, lymphatic filariasis, and African sleeping sickness. To accomplish these activities we maintain a network of collaborations with disease experts, medicinal chemists, pharmaceutical/biotech companies, and foundations.

Recent Publications

Department of Biochemistry and Molecular Biology