Gene therapy for Sickle cell Disease

The world’s 1^st gene therapy “Casgevy” for sickle cell disease has been approved in Britain recently.
It is made by Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics.
Gene therapy for sickle cell disease uses a non-infectious lentivirus to silence the BCL11A gene.
It approved the treatment for patients with sickle cell disease and thalassemia who are 12 years old and over.

Both sickle cell disease (common in people with African or Caribbean backgrounds) and thalassemia are caused by mistakes in the genes that carry hemoglobin.

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