"CRISPR Breakthrough: Gene Editing Offers Hope for Sickle Cell Patients and Infertility Concerns"
The world's first commercial gene-editing treatment, Casgevy, which employs CRISPR technology, has been approved in the UK and is pending approval in the US. Developed by Vertex Pharmaceuticals, the treatment aims to cure sickle-cell disease by turning on fetal hemoglobin, a second form of the molecule that is lost in adults. By inhibiting the gene responsible for suppressing fetal hemoglobin production, the treatment allows for increased production of the protective molecule. This breakthrough is a result of decades of research on sickle-cell disease and the discovery of the BCL11A gene's role in controlling fetal hemoglobin. The treatment involves a bone marrow transplant and is expected to be used by patients with severe symptoms. Two other gene-editing treatments targeting fetal hemoglobin production are also in trials, highlighting the potential of CRISPR technology in treating genetic diseases.
