Research

The CMM has a broad research portfolio including the following areas:

Human Disease Models

These studies apply a broad range of approaches towards understanding the molecular and cellular basis of human disease. These included the use of animal models, stem cell culture models and cell-based drug discovery through CRISPR screening and high-throughput small molecule screening. The Center has a broad interest in human health and disease including the development of new therapies and diagnostics in areas relating to cancer therapeutics, cancer biomarkers, cardiovascular disease, chronic infections, diabetes, immune disorders, and neurological disease.

Stem Cells and Regenerative Medicine

The goal of these studies is to generate pluripotent stem cell-derived cell types that can be transplanted into patients with conditions for which there are no current cures. This includes the development of cell-based therapeutics and tissue engineering to treat common diseases and repair of damaged or non-functional tissue. Areas of research interest include cardiovascular disease, metabolic diseases including diabetes, and neurological disorders including autism, brain cancers, peripheral nervous system disorders and dysfunction of the adrenal glands. The CMM Stem Cell and Regenerative Medicine Program also has interests in iPSC technology to model human disease—those of current interest include diseases of the peripheral nervous system, the adrenal gland, and congenital disorders of glycosylation.

Molecular and Cellular Mechanisms of Human Muscle Development and Regeneration

This research aims to uncover the therapeutic targets for muscle repair and human developmental disorders with muscle defects, and intersect with broader efforts in cell signaling and tissue regeneration.

Metabolic Control of Transcription in Development and Disease

This research focuses on the nuclear hormone receptor (NHR) family of ligand-regulated transcription factors and seeks to understand biological processes and gene networks regulated by NHRs, to identify cellular metabolites that serve as their natural ligands and to develop synthetic agonists/antagonists to modulate NHR activities in development and disease.

Single Cell Technologies

Recent advances in “omics” technologies make it possible to transcriptionally profile complex tissues containing cells of different lineages and differentiation stages. We are developing novel, broadly applicable computational approaches to construct differentiation trajectories, gene regulatory networks and metabolic states from single cell data.

Fueling Biotechnology

Approaches include computational systems biology tools, artificial intelligence and machine learning models, cell engineering, and deploying and optimizing next-generation biomanufacturing platforms.