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.
The red blood cells, which transport oxygen from the lungs to the rest of the body, contain a protein called haemoglobin (Hb) that consists of two alpha globin chain and two beta globin chains.
Vertex and CRISPR Therapeutics have won the backing of NICE for their gene-editing therapy Casgevy as a treatment for the blood disorder beta thalassaemia, a few months after turning it down for ...
Thalassemia can be of different types, but majorly can be alpha or beta. World Thalassaemia Day is marked every year on May 8 to raise awareness around this genetic disorder, and support those who ...
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 ...