Paper of the field n°4

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Abstract for General public

Sickle cell disease (SCD) is a genetic disorder caused by a mutation in the HBB gene. This mutation can cause normal red blood cells to change into a ‘sickle’ shape, leading to painful episodes and other health problems. Gene editing can be used to correct blood stem cells and enable SCD patients to live a normal life. With prime editing technique, the genome of human cells can be modified precisely without the need for a repair template, thereby lowering the risk of immune rejection. In this study, the researchers used prime editing to correct the disease-causing mutation in blood stem cells from SCD patients. They were able to correct 15% to 41% of mutated alleles. When these prime-edited cells were injected into mice, they displayed similar characteristics to those of unedited healthy donors in terms of engraftment, differentiation, and maturation. According to this data, prime editing can hence make lasting changes to transplanted blood stem cells, resulting in the development of red blood cells that resist sickling.

Abstract written by Parinaz Zarghamian from Universitaetsklinikum Freiburg (Germany)

Everette, Kelcee A., et al. « Ex Vivo Prime Editing of Patient Haematopoietic Stem Cells Rescues Sickle-Cell Disease Phenotypes after Engraftment in Mice ». Nature Biomedical Engineering, vol. 7, n^o 5, mai 2023, p. 616‑28. https://doi.org/10.1038/s41551-023-01026-0.