Keck Stem Cell researchers, garner $4.3 million in funding

Three scientists from Keck Medicine of USC have won grants exceeding $4.3 million from the California Institute for Regenerative Medicine (CIRM) for research that includes creating a temporary liver for transplant patients, finding novel ways to treat immune disorders and blood diseases, and developing a new animal model for exploring diseases such as heart failure, diabetes and neurodegenerative diseases.
The grants were received by USC Stem Cell Principal Investigators Paula Cannon, PhD; Toshio Miki, MD, PhD; and Qi-Long Ying, PhD. The funds are part of the CIRM Tools and Technologies initiative, which supports projects addressing the challenges of translating stem cell discoveries into cures. The winners were chosen from 212 proposals to create, design and test the key technologies needed to usher in the era of regenerative medicine.
“Sometimes even the most promising therapy can be derailed by a tiny problem,” said Jonathan Thomas, JD, PhD, chair of CIRM’s governing board. “These awards are designed to help find ways to overcome those problems, to bridge the gaps in our knowledge and to ensure that the best research is able to keep progressing and move out of the lab and into clinical trials in patients.”
Miki’s team at the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC plans to develop what he calls an “extracorporeal liver support system (ELS)” for patients with liver failure. The ELS will house a collection of human liver cells, produced from stem cells, in a device outside of the patient’s body but connected to the patient’s circulation. The ELS will therefore be able to function as a temporary liver: removing toxins, preventing irreversible brain damage, and giving the patient’s own liver a chance to recover and regenerate.
If successful, the device will allow patients to recuperate without undergoing liver transplantation.

Cannon plans to improve the precision and safety of “targeted nucleases,” which she describes as “scissors” used to edit specific genes in hematopoietic or blood-forming stem cells. Cannon hopes to develop the next generation of targeted nucleases to treat severe immune deficiencies and blood diseases, such as sickle cell disease.
Cannon and her colleagues have already developed a targeted nuclease that could potentially cure HIV/AIDS by introducing a mutation in a gene called CCR5 that confers natural immunity to HIV.

Ying, who is also at the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC, plans to use stem cell-based technology to create genetically modified laboratory rats for research into new therapies for heart failure, diabetes and neurodegenerative diseases.
Transgenic rats will provide an even more powerful tool than transgenic mice, which for nearly 25 years have allowed scientists to study and model a wide range of diseases that also occur in humans. The rat is widely accepted as more similar to the human in its physiology, which is essential for cardiac, metabolic and neurological studies.