A new federally funded research center at UC Davis is designed to help the nation develop safe, effective genome editing tools to treat patients with both common and rare diseases.
Funded by an approximately $9 million grant from the National Institutes of Health, the UC Davis Nonhuman Primate Testing Center for Evaluation of Somatic Cell Genome Editing Tools will support funded projects for research institutions that are part of the NIH’s Somatic Cell Genome Editing (SCGE) Program.
This is an incredibly exciting opportunity to advance translational research that will one day enable the treatment of a range of human diseases,” said Alice Tarantal, Ph.D., a UC Davis professor of pediatrics, cell biology and human anatomy who will direct the new center. Tarantal also serves as core scientist and unit/core leader at the California National Primate Research Center.
“UC Davis has all of the relevant research expertise and capabilities,” she said, “which made it a logical choice to be selected as the nation’s testing site for evaluating the safety and efficacy of new genome editing tools in nonhuman primates.”
While gene-editing technologies hold great promise in correcting disease-causing DNA within the human body’s somatic (non-reproductive) cells, the NIH says many challenges remain before they can be widely used in patient care to treat genetic diseases.
Advances in genome editing made over the past decade – such as the well-known CRISPR/Cas9 system – now make it possible to change DNA code inside living cells. However, to avoid or minimize unintended consequences, genome-editing tools must be able to target very specific disease genes. The tools also must work selectively on only those cells affected by a disease, while not altering other cells.
“The modern tools of gene editing have given scientists the possibility to treat genetic diseases in ways that did not exist just a few years earlier,” said David Segal, Ph.D., a professor of biochemistry and molecular medicine and faculty member in the UC Davis Genome Center. “However, before we can realize the dream of helping people, we must ensure these treatments are safe and effective. That is the work we will be doing now.
Segal will help Tarantal lead the center along with Dennis Hartigan-O’Connor, M.D., Ph.D., associate professor of medical microbiology and immunology and core scientist and core leader at the primate research center. Other key faculty members include UC Davis researchers Megan Dennis, Ph.D., Nicholas Anderson, M.S., Ph.D., Daniel Tancredi, Ph.D., and Stanford University’s Matthew Porteus.