Princeton Docket #
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
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.
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
Constant and steady supply of precursor cells and beta
No graft vs. host disease
Modular, controllable and
WO2008/066658, ENGINEERED CELLULAR PATHWAYS FOR PROGRAMMED
AUTOREGULATION OF DIFFERENTIATION
2010/0285584 A1, ENGINEERED
CELLULAR PATHWAYS FOR PROGRAMMED AUTOREGULATION OF
is an associate professor of electrical engineering
computer science in Massachusetts Institute of Technology. His
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
Christoph Schaniel is an assistant
professor of developmental and regenerative biology in the Mount Sinai Medical
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
Miles Miller is a Ph.D. student in the
department of bioengineering in Massachusetts
Institute of Technology.
application has been filed.
researchers have continued to improve the DNA circuit assembly/engineering
techniques, and have developed applications based on similar technology in the