The first person in the US given a gene editing-based therapy for a genetic disorder, Victoria Gray, is heading home, NPR reports.
Gray, who has sickle cell disease, was treated with her own bone marrow cells, which had been collected from her and altered with the CRISPR genome-editing tool to produce fetal hemoglobin. The trial she is part of is being conducted by Vertex Pharmaceuticals and CRISPR Therapeutics, which plan to eventually enroll 45 patients, as NPR previously reported. The two companies also used CRISPR as part of a treatment for a patient in Germany with beta thalassemia, NPR adds, noting that there are initial signs that that treatment might be working.
As NPR now notes, Gray had to spend months away from her home in Mississippi to get the treatment at Sarah Cannon Research Institute and TriStar Centennial Medical Center, but now she can go home, only returning to Nashville about once a month for four months for tests. NPR adds that it'll take time before researchers will know whether the treatment has worked for Gray.
"I try not to focus on dates and when I'm going to find this out, because I don't want to stress," Gray tells NPR. "So I think about just looking forward to the future and just see what comes of it. I've done it. So the hardest part is over. So now it's just waiting."