Creating Replacement Body Parts for Transplant 

A range of visionary biological and engineering techniques holds the promise of constructing organs from scratch.

Nearly 120,000 people in the US are on waiting lists for organ transplants, and that number grows every day. But a band of revolutionary thinkers at UCSF is attacking the problem from every direction. 

Zev Gartner, PhD, is engineering living materials, but not in the way we engineer cars and bridges. Rather, he is learning to harness and control the unique ability of living materials to self-organize. Constructing entire cell ecosystems would allow scientists to better understand and treat disease – and maybe even build organs from scratch. 

Gartner, professor and vice chair of the Department of Pharmaceutical Chemistry, and his colleagues have already developed a technique to build tiny models of human tissues, called organoids. The technique allows them to work with biological specimens as if they were LEGO® toy bricks. With samples from a cancer patient’s mammary gland, for example, they can construct a model to use as a personalized drug-screening platform, potentially allowing them to identify the best treatments for that particular patient’s cancer. In the process, they are also learning lessons about how these tissues form and break down in the body. These lessons may one day enable scientists to grow entire organs or stop the breakdown of tissues that occurs during disease progression and aging. 

Ophir Klein, MD, PhD, resident alumnus, is revealing how organs and tissues form in the embryo and how molecular processes within adult stem cells enable them to renew and differentiate into a range of body tissues throughout life. His research team hopes to create a paradigm for preventing and treating birth defects that arise as a result of abnormalities in these processes. Beyond that lies the potential for eventually developing replacement organs. 

A major focus for Klein’s team is craniofacial and dental development since such malformations are both common and devastating for patients and their families. But Klein, chief of the Division of Medical Genetics and chair of the Division of Craniofacial Anomalies, is also studying these processes in animal models of long bones, external genitalia, skin, intestines, and other organs. 

Holger Willenbring, MD, PhD, professor in the Department of Surgery, is working on re-engineering organs within the body, specifically the liver. His lab has shown that in mice, the cells responsible for liver cirrhosis can be converted into functional liver cells, reducing further damage to the organ and improving its function in one stroke. 

This technique is a source of hope for the 600,000 Americans suffering from liver cirrhosis, particularly the 14,000 currently on the waiting list for liver transplantation. 

Shuvo Roy, PhD (above, right), professor in the Department of Bioengineering and Therapeutic Sciences, and colleagues from pharmacy, engineering, and medicine, will soon begin human trials of an implantable artificial kidney. Their goal is to free the half a million Americans who spend several days each week tethered to dialysis machines, enabling them to once again lead normal lives. 

UCSF supports revolutionary thinkers like these, who continually push scientific boundaries to yield life-changing results. 

Ophir Klein is the Larry L. Hillblom Distinguished Professor of Craniofacial Anomalies and the Charles J. Epstein Professor of Human Genetics.

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