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To Turn on the Other Hemoglobin

Doctors in the US have used the CRISPR gene-editing tool to try to treat a woman with sickle cell disease as part of a clinical trial, Popular Science reports.

Victoria Gray is taking part in a study being conducted by Vertex Pharmaceuticals and CRISPR Therapeutics, NPR adds. For it, doctors are removing cells from patients' bone marrow and editing those cells to produce fetal hemoglobin, before then re-introducing the cells back in to the patients. People with sickle cell disease have faulty hemoglobin, but by editing the repressor of fetal hemoglobin — which is typically only expressed very early in life — the researchers hope to spur patients to produce that version of the protein to then ease their disease, Popular Science adds.

In all, the companies plan to enroll about 45 patients in their study, NPR says.

While NPR notes that it will be some time before doctors can tell if the treatment is working, Gray tells it she is envisioning a life without sickle cell disease. "Being able to wake up and not be in pain," she says. "And to just be tired because I've done something — and not just tired for no reason. And just to be outside, and jump on the trampoline with my kids."

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