UK approves world’s 1st gene therapy to treat sickle-cell, thalassemia ...Middle East

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London, Nov 18 (IANS) The UK has approved the world’s first gene therapy to treat blood disorders sickle-cell disease and thalassemia using the gene-editing tool CRISPR, which won its inventors the Nobel Prize in 2020. So far, a bone marrow transplant — which must come from a closely matched donor and carries a risk of rejection — has been the only permanent treatment option. The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) authorised the new treatment called Casgevy for patients with sickle-cell disease and transfusion-dependent beta-thalassemia aged 12 and over. Both sickle cell disease and beta-thalassemia are genetic conditions caused by errors in the genes for haemogl

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