Programmed Autoregulation of Stem Cell Differentiation into Pancreatic Beta cell: Potential Treatment for Diabetes

Web Published:

Princeton Docket # 07-2347


Researchers in Electrical Engineering and Molecular Biology, Princeton University have developed new levels of controlled differentiation in embryonic stem cells that are well-beyond existing technologies. The proposed approach will lead to new opportunities and strategies in many different biomedical therapies, such as Diabetes Mellitus.  Princeton is currently seeking commercial partners for the further development and commercialization of this opportunity.


The current standard treatment for diabetes is to maintain insulin levels by monitoring blood glucose and diet, to provide exogenous doses of insulin when necessary, and to treat the consequences of diabetes such as loss of circulation to the extremities, glaucoma, and sepsis, as the disease progresses. More radical treatments include full organ transplants, islet cell transplants or beta cell transplants. Pancreatic transplantation candidates are put on a long waiting list for a suitable organ, and after transplantation, patients must take immunosuppressants in order to battle graft vs. host disease. A recent attempt to use islet cell transplant therapy provided short-lived relief but the transplanted beta cells subsequently died or ceased to produce insulin in the majority of the patients.


Researchers at Princeton are the first to instruct stem cells to differentiate into beta cells, in vitro, using only a genetic program, a feat accomplished by mimicking the natural biological differentiation process that takes place during embryonic development. By incorporating the differentiation autoregulation pathway into embryonic stem cells, a constant and steady supply of precursor cells and beta cells is ensured. Also, this approach has the potential to bypass graft vs. host disease by using naïve embryonic stem cells or the patient¿s own adult stem cells. Finally, this approach is modular, controllable and flexible, allowing the pathways to be genetically engineered to best address each patient¿s disease state. Although use of the complete system to treat diabetes is many years off, advances made along the way may also assist in the development of approaches to treat other conditions as well.


In the case of diabetes research this technology may also provide an important platform for studying issues such as how pancreatic beta cells develop, what is necessary to create and maintain pancreatic beta cell function in vitro and in vivo, what type of signaling promotes the maintenance of beta cells, and what role other islet cells play, if any, in insulin homeostasis.



·        Treatment for diabetes

·        Stem cell therapy for other tissues

·        Research platform



·        Constant and steady supply of precursor cells and beta cells

·        No graft vs. host disease

·        Modular, controllable and flexible






The Inventors

Ron Weiss is an associate professor of electrical engineering and computer science in Massachusetts Institute of Technology.  His research focuses on programming biological organisms by embedding synthetic biochemical logic circuits into cells, as well as embedding sensors, actuators, and intercellular communication mechanisms. 

Ihor Lemischka is an internationally recognized stem cell biologist and stem cell research advocate and currently both the Lillian and Henry M. Stratton Professor of Gene and Cell Medicine and Director of the Black Family Stem Cell Institute at Mount Sinai Medical Center.

Christoph Schaniel is an assistant professor of developmental and regenerative biology in the Mount Sinai Medical Center.

Priscilla Purnick is currently an instructor of developmental and regenerative biology in the Mount Sinai Medical Center.

Patrick Guye is a postdoctoral research fellow in Massachusetts Institute of Technology in the fields of synthetic biology and stem cell research.

Miles Miller is a Ph.D. student in the department of bioengineering in Massachusetts Institute of Technology.


Intellectual Property status

U.S. patent application has been filed.

The researchers have continued to improve the DNA circuit assembly/engineering techniques, and have developed applications based on similar technology in the cancer field.

Patent Information:
For Information, Contact:
Cortney Cavanaugh
New Ventures and licensing associate
Princeton University
Ronald Weiss
Ihor Lemischka
Christoph Schaniel
Priscilla Purnick
Miles Miller
Patrick Guye
stem cell