This is a landmark approval which opens the door for further applications of CRISPR therapies in the future for the potential cure of many genetic diseases, said Kay Davies, a geneticist at the University of Oxford, UK.
The trial for sickle-cell disease has followed 29 out of 45 participants long enough to draw interim results. Casgevy completely relieved 28 of those people of debilitating episodes of pain for at least one year after treatment. Researchers also tested the treatment for a severe form of -thalassaemia, which is conventionally treated with blood transfusions roughly once a month. In this trial, 54 participants received Casgevy and 42 patients have participated for long enough to provide interim results. For at least one year after treatment, 39 participants, or 93% of those treated, did not need a red-blood-cell transfusion.
The therapy involves extracting stem cells from the patient's bone marrow, editing the genes using CRISPR, and then infusing the modified cells back into the patient's bloodstream. This allows the corrected cells to replicate and produce healthy blood cells, potentially providing a long-term cure for the diseases.
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