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To Get More Hemoglobin

New gene therapies in trials to treat sickle-cell disease are in the early stages, but seem promising, the New York Times reports.

As sickle-cell disease can be traced to a single mutation in one gene, it has long been viewed as an attractive candidate for gene therapy, the Times says. It adds, though, that characteristics of the disease — hemoglobin genes that need to be targeted are only active for a brief time in red blood cell precursors — have held the field back. It has also been overlooked, experts tell the Times, since the condition mostly affects people from less-affluent, minority communities.

But now, the Times says there are three main approaches companies are taking to try to treat the disease: genetically modifying immature blood cells isolated from patients' bone marrow, activating fetal hemoglobin to take over the job of adult hemoglobin, and using CRISPR for gene editing. It says that Bluebird Bio recently reported some success with its gene therapy approach to spur fetal hemoglobin production. The company has treated nine patients with its approach and has reported results on four, saying that, after six months, all four produce enough non-sickled hemoglobin that they no longer have disease symptoms.

This, one patient's mother tells the Times, that when she learned her son has producing enough hemoglobin, her response was: "I was like, yes, yes, thank you Lord."