CRISPR Gene-Editing Being Offered To British Blood Disorder Patients

The first therapy that uses gene-editing is to be offered on the NHS in a "revolutionary breakthrough" for patients. From a report: It will be used as a potential cure for the blood disorder beta thalassaemia. Stem cells which make blood will be extracted, reprogrammed to correct the condition and returned to the patient's body. It could spare them needing a blood transfusion, every three to five weeks, for life. People with beta thalassaemia struggle to produce enough haemoglobin, which is the protein in red blood cells that carries oxygen around the body. It is a genetic disease that is passed down through families and caused by defects in the body's instructions for manufacturing haemoglobin. It can leave people severely tired, weak, and short of breath and also cuts life expectancy.

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