Basic science is the indispensable foundation of medical advances

Areas of Research



The heart cannot adequately regenerate damaged tissue after a heart attack. Could stem cells help it along that path? Over the last decade, UCSF researchers have discovered that stem cell implantation after a heart attack does indeed improve cardiac function, but the retention rate of these cells in the heart is limited. The main mechanism of cardiac functional improvement appears to be paracrine in nature, meaning the stem cells release specific proteins that are cardio-protective. UCSF scientists are currently identifying and isolating these proteins in order to develop them into novel therapies. Efforts are also underway to create bioresorbable scaffolds to increase the retention rate of stem cells after implantation in the heart. Lastly, researchers are working to convert cardiac fibroblast cells in vivo into beating heart muscle cells to improve heart function after an injury.



Epithelial cells constitute at least 60% of all differentiated cells in the body. Epithelial tissues, such as the skin and the linings of the gastro-intestinal tract, contain stem cells that are capable of self-renewal and differentiation. The Epithelial Disease Pipeline focuses on therapeutic applications of stem cells for either common chronic diseases or rare but severe diseases of epithelial origin. Knowledge of the properties and localization of epithelial stem cells gained through research into the skin or intestine will help facilitate these studies. Purifying these stem cells and studying their conversion into different lineages could make it feasible to repair genetic or functional defects in the stem cells themselves, or to transplant the cells into severely affected patients. This program also investigates the risks of tumor development from stem cell therapy. At least 85% of all human cancers arise in epithelial tissues like the skin, colon, breast and prostate, and there is increasing evidence that the most malignant tumors develop from epithelial stem cell populations.



Research into the hematopoietic system has provided fundamental insights into how different cell types derive from stem cells and progenitor cells, and this knowledge has served as the basis for therapeutic approaches. Indeed, therapeutic use of hematopoietic stem cells is now common practice, and hematopoietic stem cell transplantation has cured thousands of people with bone marrow failure, leukemia, and hereditary hematologic and immune disorders. Ongoing goals of this burgeoning stem cell field are to extend the success of past studies in identifying cell surface markers present on stem/progenitor populations, developing functional in vitro and in vivo assays that accurately measure stem cell activity, and harnessing these insights to treat human diseases. Much has been accomplished in the areas of hematopoiesis and hematopoietic stem cell biology, but a great deal of work remains to fully realize the promise of hematopoietic cell-based therapies.



The entire musculoskeletal system – bone, cartilage, muscle, fat and connective tissue – develops from mesenchymal stem cells. Studies aimed at understanding the differentiation processes and regenerative capacity of these tissue types have uncovered key signaling molecules that control mesenchymal stem cell function. This knowledge has provided insight into how deficiencies in these molecules give rise to developmental and degenerative diseases of muscle, bone and cartilage. What’s more, it allows researchers to regulate and maybe even redirect mesenchymal stem cell differentiation, opening the door to new therapeutic approaches for repairing and regenerating these tissues. 



The Neural Stem Cell Center focuses on human brain development, evolution and disease. It is a unique subset of the neuroscience community at UCSF, consisting of world-renowned scientists spanning basic and clinical departments who are applying cutting-edge technologies to study the human brain. Their work centers on developmental and stem cell biology, with particular attention paid to brain maturation spanning from early development in utero through adolescence and into adulthood. Findings have included the discovery of new neural stem and progenitor cell types in the embryonic human brain, streams of young migrating neurons and neural progenitor cells in the neonatal brain, and regions of neuron production in the young adolescent human brain that change dynamically with age. Additionally, the Center has hosted UCSF’s first clinical trial for a stem cell therapy to treat neurological disease, as well as developed and patented a novel clinical device for stem cell delivery to the brain.



The eyes are our window to the world. However, a number of injuries and diseases, such as age-related macular degeneration, glaucoma and retinitis pigmentosa, can lead to irreversible vision loss. This is because the light-sensing regions of our eyes, like the retina, lack stem cells with regenerative potential. So, scientists are harnessing the power of newer stem cell technologies to identify novel therapies for the eye. Major focuses include generating replacement cells for the retina from stem cells in a dish, understanding connectivity issues when reconstructing neuronal circuits following damage, and surpassing micro-environmental barriers to regenerative medicine. Another promising area is the use of patient-derived stem cells to build better disease models, which will further scientists’ understanding of the human disease process and help them develop more effective drug or gene-editing strategies using personalized medicine approaches.

Pancreas, Diabetes, & Liver Disease


Diabetes mellitus is one of the most common diseases in the United States, affecting over 16 million people and consuming one out of every eight health care dollars. Chronic liver disease affects as many as 5 million Americans and results in cirrhosis, liver failure and death in approximately 30,000 people each year. Stem cell therapy could potentially provide a source of liver and pancreatic cells for these patients and thus a viable cure for liver failure and diabetes. The Pancreas/Diabetes and Liver Disease Pipeline at UCSF is a comprehensive program devoted to bringing together basic and clinical research to advance the understanding and treatment of liver failure and diabetes through stem cell therapy. It is the only program in California and one of the few programs in the country that combines superb developmental and stem cell research with a dedicated clinical program of liver and islet transplantation, as well as the immunology efforts needed to move aggressive basic research towards potential cures for these diseases.



Abnormal development of gametes (sperm and eggs) and embryos is a leading cause of birth defects and poor pregnancy outcomes. Examples include common errors in chromosomal number (such as Down syndrome), infertility, recurrent miscarriage and serious pregnancy complications, including preeclampsia, intrauterine fetal growth restriction and pre-term delivery. The Center for Reproductive Sciences has built a premier program to investigate fundamental aspects of human reproduction and development. Their goal is to help couples have healthier pregnancies and healthier babies, either by natural conception or by assisted reproductive technologies. To do so, UCSF scientists are using human embryonic stem cells to gain insights into reproductive processes and explore the early stages of human development. They are employing a combination of genetic, proteomic, cell biological, molecular and biochemical methods to address fundamental questions that must be answered in order for scientific discoveries to be translated into advances in clinical care.