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Sickle Change

Researchers from Stanford University have used the CRISPR gene-editing tool to fix sickle cell anemia-causing mutations within the β-globin (HBB) gene in hematopoietic stem cells, Reuters reports.

"What we've finally shown is that we can do it. It's not just on the chalkboard," senior author Matthew Porteus from Stanford tells Reuters.

As they report in Nature, Porteus and his colleagues developed a system that relies on CRISPR/Cas9 gene editing to edit HBB as well as an adeno-associated viral vector delivery system to introduce that mechanism into cells. They tested this approach in healthy hematopoietic stem cells as well as in hematopoietic stem cells from people with sickle cell disease. Reuters reports that the researchers were able to fix the gene mutation in some 30 percent to 50 percent of cells. The researchers further introduced edited cells into immuno-compromised mice, which 16 weeks later were still present in the bone marrow.

"We think we have a complete data set to present to the [Food and Drug Administration] to say we've done all pre-clinical experiments to show this is ready for a clinical trial," Porteus tells Reuters.