Three researchers from the School of Medicine have been awarded grants by the governing board of the California Institute for Regenerative Medicine to promote the discovery of potential stem cell-based therapies.
The awards, which were announced Jan. 19, were made as part of the state stem cell agency's Quest program, which funds the discovery phase of research expected to advance to the next stage of development within two years.
Rosa Bacchetta, MD, associate professor of pediatrics, was awarded $1.1 million to use a gene-editing technique to repair blood stem cells from patients with a rare but fatal genetic autoimmune disease called IPEX.
"Although it is a rare disease, IPEX is a prototype of a series of diseases with autoimmunity of genetic origin that overall severely affect children at a very early age," Bacchetta said in a statement from the agency about the awards, adding that she hopes the work will provide "a unique path forward in developing a definitive cure for this devastating genetic disease."
Roeland Nusse, PhD, professor of developmental biology and the Virginia and Daniel K. Ludwig Professor in Cancer Research, was awarded $1.7 million to investigate ways to grow liver stem cells in the laboratory while also maintaining their regenerative capacity. These cells could potentially be used to treat severe liver disease or to alleviate the shortage of donor organs.
Matthew Porteus, MD, associate professor of pediatrics, was awarded $2.2 million to investigate ways to use gene editing to correct cystic fibrosis mutations in airway stem cells.
"At CIRM we never underestimate the importance of early stage scientific research; it is the birth place of groundbreaking discoveries," said C. Randal Mills, PhD, president and CEO of CIRM, in a statement about the awards. "We hope these Quest awards will not only help these incredibly creative researchers deepen our understanding of several different diseases, but also lead to new approaches on how best to use stem cells to develop treatments."
In total, the agency awarded more than $20.5 million to 11 researchers during this round of funding.
