- Medical regulators in the UK has approved a gene therapy that aims to cure two blood disorders.
- Gene editing tool for treatment of sickle cell disease and beta thalassemia is known as Crispr.
- Gene-editing allows the precise manipulation of DNA.
- The treatment involves removing bone marrow stem cells from a patient’s blood
Medical regulators in the UK has 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.
How it works
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.
In a laboratory, the gene-editing tool Crispr uses molecular scissors to make precise cuts in the DNA of the cells, thus disabling the faulty gene.The modified cells are infused back,allowing the body to start producing functioning haemoglobin.
In trials, 28 out of 29 sickle cell patients were free of severe pain and 39 of 42 beta thalassemia patients no longer needed blood transfusions for at least a year. It’s hoped it could be a permanent fix.
Trials are continuing in the UK, US, France, Germany and Italy.
Around 15,000 people in the UK have sickle cell disease,most with an African or Caribbean family background. Almost 300 babies are born in the UK with sickle cell disease each year.
More than 1,000 people in the UK are affected by thalassemia, mainly those of Mediterranean, southeast Asian and Middle Eastern origin.