The research of Timothy J. Nelson, M.D., Ph.D., focuses on cardiovascular regeneration using bioengineered stem cells. His work is directed at improving scientists' ability to discover, diagnose and ultimately treat mechanisms of degenerative diseases such as cardiomyopathy that weaken the heart muscle and lead to progressive heart failure.

Specifically, Dr. Nelson's research team starts with skin samples from patients with severe forms of heart disease and generates induced pluripotent stem (iPS) cells to create a model system for that patient in the laboratory. They examine the differences between samples obtained from patients with heart disease and family members without heart disease to determine the molecular underpinning of a particular disease. The goal of this research is to identify better ways to predict the onset and progression of that disease in the laboratory without the need for probing the patient beyond the initial tissue collection.

Additionally, Dr. Nelson works to expand understanding of diseases in bioengineered cell culture systems with next-generation sequencing platforms that enable innovative collaborations to better define associations between certain genotypes and phenotypes.

Focus areas

Current projects include:

• Dilated cardiomyopathy and the application of iPS cell technology to determine molecular defects in cardiogenesis and cardiac self-renewal pathways as potential determinants of degenerative heart disease.
• Hypoplastic left heart syndrome and the application of iPS cell technology to determine molecular defects in developmental pathways that could explain the lack of left ventricular muscle formation as possible etiologies for congenital heart disease.
• Mouse model of natural cardiogenesis to produce a "roadmap" for systems developmental biology and engineer stem cell progeny for improved therapeutic applications.
• Mechanisms for improved nuclear reprogramming to produce pluripotent stem cells with cardiac differentiation potential devoid of the risk of tumor formation.

Significance to patient care

Ultimately, the research of Dr. Nelson's team will advance stem cells beyond scientific discovery to help translate the technology into clinical applications and alleviate the ever-growing burden of degenerative diseases that currently require organ transplantation.