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Genome Engineering of a Safety Switch and Immunoprotection in Stem Cells

Andras Nagy, Canada

Senior Investigator and Professor
Lunenfeld-Tanenbaum Research Institute, Sinai Health System
Sinai Health and University of Toronto


The solutions for cell therapy safety and allogeneic cell acceptance are critical for getting therapies, including those that targeting T1D, into the clinic. Our work in these areas has led to the development of two platform technologies; FailSafeTM cells and induced Allogeneic Cell Tolerance (iACT). We feel strongly that both could become the standard of care in many cell therapy applications and allow us to envision a single, pluripotent cell line that could be used as a source of safe and off-the-shelf therapeutic cells to serve all humankind. 

We foresee several complementary approaches for our technologies towards the treatment of T1D.  First, we showed that we could generate a subcutaneous immune-privileged hosting tissue site for transplanted islets, which protects from allogeneic or even xenogeneic rejection and autoimmune attack. In addition to creating a subcutaneous tissue as a protective site for grafts, we also envision that our FailSafeTM and cloaked pluripotent cells could be used to make therapeutic cells via directed differentiation, such as insulin-producing, beta cells.


Dr. Nagy is currently a Shawn Kimel Senior Scientist at the Lunenfeld-Tanenbaum Research Institute, Sinai Health System, Professor in the Department of Obstetrics & Gynaecology and Institute of Medical Science at the University of Toronto, Investigator at the McEwen Centre for Regenerative Medicine and Professor at the Australian Regenerative Medicine Institute in Monash University, Melbourne.  He holds a Tier I Canada Research Chair in Stem Cells and Regeneration.  He also has a Fellowship of the Royal Society of Canada in the Life Sciences Division of the Academy of Science and recently became a Foreign Member of the Hungarian Academy of Sciences.   Dr. Nagy has made significant breakthroughs in the development of mouse and human pluripotent stem cells (both embryonic and induced) that could accelerate research in regenerative medicine and lead to future therapies for currently incurable diseases, such as blindness, diabetes, arthritis, spinal cord injury and many others. His team created the first two Canadian human embryonic stem cell lines and developed a novel method for generating non-viral induced pluripotent stem cells. His current research focuses on understanding the process of reprogramming to stem cells at the molecular level and using sophisticated genome editing methodology to pave the way leading to safe and effective cell based therapies of diseases.

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