Scientists identify potential new genetic target for sickle cell disease treatment

Scientists from Johns Hopkins Medicine and eight other institutions in the United States, Africa and Europe say they have identified a potential new gene target that could be edited to treat sickle cell disease, an inherited blood disorder marked by sickle-shaped red blood cells that cause intense pain and shorten lifespans. The potential target, the FLT1 gene, contributes to the production of a protein, fetal hemoglobin, whose presence is already known to improve the lifespan of people with sickle cell disease.

Mar 27, 2025 - 17:08
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Scientists from Johns Hopkins Medicine and eight other institutions in the United States, Africa and Europe say they have identified a potential new gene target that could be edited to treat sickle cell disease, an inherited blood disorder marked by sickle-shaped red blood cells that cause intense pain and shorten lifespans.

The potential target, the FLT1 gene, contributes to the production of a protein, fetal hemoglobin, whose presence is already known to improve the lifespan of people with sickle cell disease.