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The drug called “Casgevy” has been approved for use in the treatment of two genetic blood diseases, sickle cell disease and beta thalassemia, in patients twelve years of age and older. Both diseases are caused by errors in the hemoglobin gene.
This method, called gene scissors, can specifically target individual genes. The MHRA said in a statement that “Casgevy” was developed to enable patients to produce functioning hemoglobin by replacing faulty genes in their bone marrow stem cells.
For this, stem cells are taken from the bone marrow, processed in the laboratory and then given back to the patient. It was stated that the result has the potential to last a lifetime. (SDA)
Source : Blick
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