OAKLAND, Calif. - UCSF Benioff Children’s Hospitals has received $17 million in federal funding to launch clinical trial in Oakland aimed to cure sickle cell disease using CRISPR gene-editing technology, the hospital president announced on Wednesday.
Matt Cook wrote a letter to colleagues saying that this money is the "largest research trial grant ever awarded to UCSF Benioff Children’s Hospital Oakland and continues a decades-long commitment to advancing new treatments and eliminating health disparities tied to this debilitating genetic blood disorder, which disproportionately impacts Black and Latino communities."
The four-year clinical trial will be led by Dr. Mark Walters, director of Oakland’s Pediatric Blood and Marrow Transplant Program. And scientists will use the CRISPR-Cas9 gene-editing technology on patients’ own blood stem cells to correct the mutated gene that causes sickle cell disease.
A CRISPR is a tool used for editing genomes, allowing researchers to easily alter DNA sequences and modify gene function. It has the potential to correct genetic defects, treat and stop the spread of diseases, among other benefits.
Researchers are planning to enroll the first patient by mid-2022. Recruitment has started in Oakland and Los Angeles.
First, up to six adults with the disease will be treated and if the treatment is safe and effective, three children 12 to 17 years old will be enrolled.
Researchers expect to treat seven patients in Oakland and two at the University of California at Los Angeles.
The grant comes from the National Institutes of Health.
Meanwhile, the hospital also raised $175,000 in donations this week while partnering with KTVU on Giving Tuesday.
Bay City News contributed to this report.