Keck stem cell scientists awarded grant


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Three scientists from the Keck School of Medicine of USC have won grants from the California Institute for Regenerative Medicine for research that includes creating a temporary liver for patients and developing new ways to treat immune disorders.

The grants are being awarded to USC Stem Cell principal investigators Paula Cannon, Qi-Long Ying and Toshio Miki as part of the CIRM Tools and Technologies Initiative, which supports projects that address the challenges of translating stem cell discoveries into cures.

The winners were chosen from a group of more than 200 proposals to design and create the key technologies needed for progress in the era of regenerative medicine.

“These awards are designed to help find ways […] 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,” said Jonathan Thomas, chair of CIRM’s governing board in a statement to USC News.

Miki’s team at USC’s Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research plans to develop what he calls an “extracorporeal liver support system” for patients suffering from liver failure.

The project overseen by Miki is an international collaboration with Katrin Zeilinger at Berlin’s Charité University, Frank Shubert at Stem Cell Systems GmbH, and Germany’s Federal Ministry for Education and Research.

Cannon and her colleagues are working to develop the next generation of targeted nucleases to treat blood diseases and severe immune deficiencies, such as sickle cell disease.

Her team has already developed a targeted nuclease that could potentially cure HIV/AIDS by introducing a gene mutation called CCR5 that confers natural immunity to HIV. Inspiration stemmed from the “Berlin patient,” a patient cured of both HIV and leukemia through a bone marrow transplant from a donor with a CCR5 mutation.

Ying, also at the Broad Center for Regenerative Medicine and Stem Cell Research, plans to use stem cell technology to create some of the world’s most sophisticated lab rats for use in research of new therapies for diabetes, heart disease and neurodegenerative diseases.

Ying is also collaborating with USC colleagues Justin Ichida, Bangyan Stiles and Ching-Ling “Ellen” Lien in his research efforts.