CHORI Research on Genome Editing for a Sickle Cell Cure Receives nearly $4.5 million from the California Institute for Regenerative Medicine
Research on Genome Editing to Pave the Way for Sickle Cell Cure
Oakland (December 6, 2016) - UCSF Benioff Children's Hospital Oakland's Dr. Mark Walters has received a nearly $4.5 million grant from the California Institute for Regenerative Medicine (CIRM) for his
research project, "Curing Sickle Cell Disease with CRISPR-Cas9 genome editing." It is one of four research projects, focused on diseases that have an enormous impact on life expectancy and quality of life, that have been awarded a total of $15 million by the governing Board of CIRM.
The funding is part of the Translational Award program, which has a goal of moving the most promising projects out of the laboratory and into clinical trials in people.
Dr. Walters' research uses a genetic editing tool, CRISPR-Cas9, to help find a cure for sickle cell disease. The research involves taking the patient’s own blood stem cells, using CRISPR-Cas9 to correct the genetic mutation causing the disease, and then return those cells to the patient. It’s hoped the “corrected” blood stem cells will then multiply and create a new blood system, one free of sickle cell. The funding from CIRM will enable Dr. Walters' team to do the preliminary testing and research needed to get this project ready for a clinical trial.
“These Translational Awards highlight our goal of creating a pipeline of projects, moving through different stages of research with an ultimate goal of a successful treatment,” says Jonathan Thomas, Ph.D., J.D., Chair of the CIRM Board. “We are hopeful these projects will be able to use our newly created Stem Cell Center to speed up their progress and pave the way for approval by the FDA for a clinical trial in the next few years.”
Dr. Walters also collaborated with CHORI Senior Scientist Dr. David Martin as well as researchers at the University of California, Berkeley and the University of Utah School of Medicine on the research. The team of physicians and laboratory scientists has taken a key step toward a cure for sickle cell disease, using CRISPR-Cas9 gene editing to fix the mutated gene responsible for the disease in stem cells from the blood of affected patients.
For the first time, they have corrected the mutation in a proportion of stem cells that is high enough to produce a substantial benefit in sickle cell patients. The researchers hope to re-infuse patients with the edited stem cells and alleviate symptoms of the disease, which primarily afflicts those of African descent and leads to anemia, painful blood blockages and early death.
In tests in mice, the genetically engineered stem cells stuck around for at least four months after transplantation, an important benchmark to ensure that any potential therapy would be lasting.
“This is an important advance because for the first time we show a level of correction in stem cells that should be sufficient for a clinical benefit in persons with sickle cell anemia,” said co-author Mark Walters, a pediatric hematologist and oncologist and director of UCSF Benioff Children's Hospital Oakland’s Blood and Marrow Transplantation Program.
The results will be reported in the Oct. 12 issue of the online journal Science Translational Medicine
Sickle cell disease is a recessive genetic disorder caused by a single mutation in both copies of a gene coding for beta-globin, a protein that forms part of the oxygen-carrying molecule hemoglobin. This homozygous defect causes hemoglobin molecules to stick together, deforming red blood cells into a characteristic “sickle” shape. These misshapen cells get stuck in blood vessels, causing blockages, anemia, pain, organ failure and significantly shortened lifespan. Sickle cell disease is particularly prevalent in African Americans and the sub-Saharan African population, affecting hundreds of thousands of people worldwide.
About UCSF Benioff Children’s Hospital Oakland
UCSF Benioff Children’s Hospital Oakland (formerly Children’s Hospital & Research Center Oakland) is a premier, not-for-profit medical center for children in Northern California, and is the only hospital in the East Bay 100% devoted to pediatrics. UCSF Benioff Children’s Hospital Oakland affiliated with UCSF Benioff Children’s Hospital San Francisco on January 1, 2014. UCSF Benioff Children’s Hospital Oakland is a national leader in many pediatric specialties including cardiology, hematology/oncology, neonatology, neurosurgery, endocrinology, urology, orthopedics, and sports medicine. The hospital is one of only five ACS Pediatric Level I Trauma Centers in the state, and has one of largest pediatric intensive care units in Northern California. UCSF Benioff Children’s Hospital Oakland is also a leading teaching hospital with an outstanding pediatric residency program and a number of unique pediatric subspecialty fellowship programs.
UCSF Benioff Children’s Hospital Oakland’s research arm, Children’s Hospital Oakland Research Institute (CHORI), is internationally known for its basic and clinical research. CHORI is at the forefront of translating research into interventions for treating and preventing human diseases. CHORI has 250 members of its investigative staff, a budget of about $50 million, and is ranked among the nation’s top ten research centers for National Institutes of Health funding to children’s hospitals. For more information, go to www.chori.org.
Contact: Melinda Krigel
UCSF Benioff Children’s Hospital Oakland
510-428-3069, 510-388-5927 (cell)