A gene editing technology called CRISPR/Cas9 repaired 20 to 40 percent of stem and progenitor cells obtained from sickle cell disease patients, scientists report, suggesting such an approach might offer a way of treating this disease.
Sickle cell anemia is the most severe type of sickle cell disease, which is a group of inherited conditions that affect hemoglobin, the molecule in red blood cells that transports oxygen throughout the body. A single mutation in the gene that helps to make hemoglobin alters red blood cells to a sickle, crescent shape. These cells die prematurely, leading to anemia, and may also get stuck in small blood vessels, blocking blood flow.
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Researchers injected these altered cells into the bone marrow of mice with defective immune systems and determined how many retained the edit after 19 weeks. “The rate of repair remained stable, which is great,” [said Gang Bao, PhD, a member of the research team and a bioengineer at Rice.]
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Regarding their clinical significance and whether repairing as much as 40 percent of the cells is enough to cure the disease, Bao noted that more work is needed. “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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