Primate researchers at the University of California, Davis, will be testing the safety and efficacy of gene editing tools that they expect will have future applications in humans, work that the university said Monday is being financed by $9 million from the National Institutes of Health.
While discoveries in genome editing, such as the well-known CRISPR/Cas9 system, now make it possible to change DNA code inside living cells, challenges still remain before such techniques can be widely used in patient care to treat genetic diseases, UC Davis researchers said.
“This is an incredibly exciting opportunity to advance translational research that will one day enable the treatment of a range of human diseases,” said UCD professor and scientist Alice Tarantal, who will lead the center doing the research. “UC Davis has all of the relevant research expertise and capabilities, 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. We are pleased to be a member of the nationwide consortium focused on somatic cell genome editing, and to partner with the NIH in this crucial initiative.”
Tarantal works jointly as a core scientist and unit/core leader at UCD’s California National Primate Research Center and a professor of pediatrics and cell biology and human anatomy in the UC Davis School of Medicine.
The new center will be known as the UC Davis Nonhuman Primate Testing Center for Evaluation of Somatic Cell Genome Editing Tools, and as the name implies, testing will be done on nonhuman primates. There, Tarantal and other researchers will be looking for ways to ensure that genome-editing tools target only specific disease genes, avoiding any alterations in other cells and mitigating unintended consequences.
The leaders of the center will be led David Segal, a professor of biochemistry and molecular medicine and a faculty member in the UC Davis Genome Center and Dennis Hartigan-O’Connor, an associate professor of medical microbiology and immunology and core scientist and core leader at the California National Primate Research Center.
Segal said: “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. 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.”
Why did the NIH turn to UC Davis researchers to do this research? It turns out that they had expertise in a number of areas critical to doing the work:
- Expertise in nonhuman primate models for translational research and within the NIH-supported California National Primate Research Center.
- Innovation in gene therapy and genome-editing technologies and tools, including the use of viral and non-viral vectors.
- The UC Davis Genome Center, which has state-of-the-art instrumentation and experienced faculty.
- Leaders in database management, evaluation, and related translational resources through UC Davis’ Clinical and Translation Science Center.
- A renowned group of external advisers who will serve as members of the new testing center’s steering committee.