Medical regulators in the UK have approved a gene therapy that aims to cure two blood disorders, sickle cell disease and beta thalassemia, using the gene-editing tool Crispr.
In a world first, medical regulators in the UK have approved a gene therapy that aims to cure two blood disorders . The treatment for sickle cell disease and beta thalassemia is the first to be licensed using the gene-editing tool known as Crispr . This is a revolutionary advance for two inherited blood conditions, both triggered by errors in the gene for haemoglobin.
People with sickle cell disease produce unusually shaped red blood cells that can cause problems because they do not live as long as healthy blood cells and can block blood vessels, causing pain and life-threatening infections. People with beta thalassaemia do not produce enough haemoglobin, which is used by red blood cells to carry oxygen around the body. Patients with beta thalassemia often need a blood transfusion every few weeks of their lives. DNA is the blueprint of life - and genes contain the instructions for how every cell in our body works. Gene-editing allows the precise manipulation of DNA. The treatment involves removing bone marrow stem cells from a patient's blood
UK Gene Therapy Blood Disorders Sickle Cell Disease Beta Thalassemia Crispr Gene-Editing
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