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Gene therapy for Sickle cell Disease
November 22 , 2023
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- The United Kingdom has approved the world's first gene therapy treatment for sickle cell disease and thalassemia.
- It approved Casgevy for patients with sickle cell disease and thalassemia who are 12 years old and over.
- Casgevy is made by Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics.
- Both sickle cell disease and thalassemia are caused by errors in the genes that carry haemoglobin, the protein in red blood cells that carry oxygen.
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