Modeling Congenital Heart Disease with Induced Pluripotent Stem Cells

Funding Type: 
Tissue Collection for Disease Modeling
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 

Induced pluripotent stem cells are stem cells that can be derived from a differentiated cell by introducing genes which, when over-expressed, revert the cell into a less differentiated state. These cells are very valuable because they can be programmed to differentiate once again into a variety of cell types. The purpose of this study is to provide blood cells from patients with congenital heart disease as well as from healthy controls for the generation of lines of induced pluripotent stem cells that can be used to unravel the molecular basis for specific congenital heart diseases. In many cases, the causes of the disease are not known or are multi-faceted and not well understood. The cells will provide a valuable research resource for scientists interested in identifying why specific congenital heart diseases occur. The cells will also be used to develop new ways to repair the heart. There are approximately 36,000 infants born each year with a congenital heart defect and congenital heart disease affects between one and two million adults in the United States. The availability of these cells will not only provide a valuable research resource but can also be used to develop safer drugs for young patients whose heart cells respond differently to drugs when compared with adults. This resource will provide the opportunity for new and better treatments to be made available for all patients with congenital heart disease.

Statement of Benefit to California: 

The American Heart Association has reported that the costs of treating heart disease in the United States are expected to soar from $272.5 billion to $818 billion dollars over the next twenty years. Since heart disease is one of the most common chronic health conditions in California, the associated costs of treating these patients will be a significant public health challenge in this state. The current costs of annual health care for California residents with heart disease exceed $12,900 per capita, over five times the health care costs of the general adult population. It would benefit California, and the general population, if scientists could develop new and cost-efficient treatments for patients with heart disease. Our clinical and biomedical research team proposes to provide samples from patients with congenital heart disease to develop a research resource of induced pluripotent stem cells that can be used to study congenital heart defects that impact children and one to three million adults in this country. The development of stem-based therapies would be facilitated by this resource, thereby improving patient outcome and reducing medical care costs. New methods for the use of these cells, in partnership with industry, can bring new revenue to California.