Progress Reported in Using CRISPR/Cas9 Editing to Correct Sickle Cell Mutation

Scientists have successfully used gene editing to repair 20 to 40 percent of stem and progenitor cells taken from the peripheral blood of patients with sickle cell disease, according to Rice University bioengineer Gang Bao., PhD. Dr. Bao, in collaboration with colleagues at the Baylor College of Medicine, Texas Children's Hospital, and Stanford University, is working to find a cure for the hereditary disease. A single DNA mutation causes the body to make sticky, crescent-shaped red blood cells that contain abnormal hemoglobin and can block blood flow in limbs and organs. In his talk on February 16, 2018 at the annual American Association for the Advancement of Science (AAAS) meeting (http://meetings.aaas.org/) in Austin, Texas, Dr. Bao revealed results from a series of tests to see whether CRISPR/Cas9-based editing can fix the mutation. His presentation was part of a scientific session titled "Gene Editing and Human Identity: Promising Advances and Ethical Challenges." (https://aaas.confex.com/aaas/2018/meetingapp.cgi/Session/17907) "Sickle cell disease is caused by a single mutation in the beta-globin gene (in the stem cell's DNA)," Dr. Bao said. "The idea is to correct that particular mutation, and then stem cells that have the correction would differentiate into normal blood cells, including red blood cells. Those will then be healthy blood cells." Dr. Bao's lab collaborated with Dr. Vivien Sheehan, an Assistant Professor of Pediatrics and Hematology at Baylor and a member of the Sickle Cell Program at Texas Children's, to collect stem and progenitor cells (CD34-positive cells) from patients with the disease. These cells were then edited in the Bao lab with CRISPR/Cas9 together with a custom template, a piece of DNA designed to correct the mutation.
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