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Blood sample of patient positive tested for sickle cell.Kitsawet Saethao | Istock | Getty ImagesThe approval of two gene therapies to treat sickle cell disease has given hope to patients who suffer from the debilitating disease, which overwhelmingly affects Black people and people of color.Health officials now face a challenge in finding a way to provide equitable access to the costly treatments.The crippling episodes of pain from the genetic blood disorder make life unpredictable for patients like Michael Goodwin.…

It really shouldn’t have come as a surprise. Perhaps no technology has more power to transform medicine, and its vast potential is just beginning to be realized. Gene editing can be used to delete, insert, or alter portions of our genetic code. We’ve been able to modify DNA for years, but newer technologies like CRISPR mean that we can do it faster, more accurately, and more efficiently than ever before. In 2023, we saw the first approval of a CRISPR-based gene-editing therapy. And many more are to come. So let’s take a…

Such drawbacks are why a pill to alleviate sickle-cell, if developed, could sweep CRISPR from the playing field. A pill version could also resolve a brewing moral dilemma: Vertex so far has no plans to offer its gene-editing treatment in those countries where sickle-cell is most common. A wide ribbon of lower-income nations across the middle of Africa, including Nigeria and Ghana, account for 80% of sickle-cell cases but, according to US researchers, lack the hospitals, medical expertise, and money to implement this…

The association technique hasn’t always paid off—but starting in 2007 the gene searches hit pay dirt for sickle-cell. In one study, for instance, a team in Italy studied DNA from thousands of Sardinians (some of whom had beta-thalassemia, another hemoglobin disorder, which is shockingly common on the island) as well from Americans with sickle-cell. When they compared each person’s DNA with the amount of fetal hemoglobin each had, variations kept popping up in one gene: BCL11A. This gene was far from the hemoglobin…

The United Kingdom’s…