( 12.27.25 ) Written by Rian Kim (special edition)
SCD mostly affects those of African or Indian descent, as carrying sickle cell genes could protect against malaria. Malaria is noted as a common threat in those regions, thus making sickle cells common.
In the study, researchers discovered that by editing bases, functional fetal hemoglobin levels can increase by over 60%. This led to less red blood cell sickling in 1-6 months, reduced cell adhesion, when cells stick to each other, and improved blood circulation. Furthermore, treated patients saw no painful vasocclusive crises, such as chest pain, like those SCD might cause. The editing technology, known as BEAM-101, used adenine base editors to implement single-base changes to the promoter regions of y-globin genes HBG1 and HBG2.
BEAM-101, and its applications seem to be on par with other gene editing technologies, such as CRISPR, allowing it to bring forward exciting new applications. Primarily, with more advancements, it could finally introduce a universal treatment to sickle cell disease, which plagues thousands today.
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You can read the full paper here:
Caption: Picture of sickle cells and normal red blood cells
https://outliermedia.org/what-detroiters-should-know-about-gene-therapies-for-sickle-cell-disease/