The first therapy that uses gene-editing is to be offered on the NHS in a “revolutionary breakthrough” for patients. 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. Kirthana Balachandran, 21, was diagnosed when she was only three months old and gets muscle pain and back pain and can have palpitations when walking uphill. “The idea of depending on transfusions for quite literally the rest of my life is daunting, I constantly worry about the future,” she says.
The idea of gene-editing is you only need to do it once. It relies on the a tool called Crispr, which won the Nobel Prize for Chemistry in 2020. It is essentially a satnav connected to a pair of scissors – one part targets the right section of DNA and the other performs the edit.
You might think this technology is being used to repair the genetic defect, but it is actually cuter than that. Instead it relie.