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.
Bao, in collaboration with 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 at the annual American Association for the Advancement of Science meeting in Austin Feb. 16, 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.”
“Sickle cell disease is caused by a single mutation in the beta-globin gene (in the stem cell’s DNA),” he 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.”
The findings, part of an upcoming paper, are a step toward treating sickle cell disease. Obstacles in the way of a cure include optimizing the CRISPR/Cas9 system to eliminate off-target effects, as well as finding a way to further increase the amount of gene-corrected stem cells.
Bao pointed out that researchers still don’t know whether repairing as much as 40 percent of the cells is enough to cure a patient. “We’d like to say, ‘Yes,’” he said, “but we don’t really know yet. That’s something we hope to learn from an eventual clinical trial.”
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