#shorts #sickle-cell disease #beta-thalassemia #hemoglobin #CRISPR
Sickle-cell disease and beta-thalassemia patients in the UK rejoice!** The world’s first CRISPR-based therapy has been approved by the UK medicines regulator, meaning that these two blood conditions could soon be a thing of the past. The treatment, called CASGEVY, uses CRISPR to edit a gene that normally prevents production of a type of fetal hemoglobin. This allows the body to produce hemoglobin that is free of the abnormalities associated with sickle-cell disease and beta-thalassemia, which in turn boosts oxygen supply to the tissues and alleviates patientsβ symptoms. Of course, this is not a cure-all β the treatment is expensive and only available to patients aged 12 and up. But itβs a huge step forward in the fight against these devastating diseases, and we canβt wait to see what CRISPR can do next.
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