World first… as Victoria freed of sickle cell disease
SCIENTISTS in the US discovered a cure for one of the most common blood diseases, which affects 14,000 Britons.
Thanks to a pioneering gene-editing technique, 34-year-old mother-of-three Victoria Gray from Mississippi became the first in the world to be free from sickle cell disease – a deadly genetic condition.
Sickle cell disease alters the shape of red blood cells, causing them to get stuck inside blood vessels.
The lack of oxygen-rich red cells flowing around the body results in agonising pain and irreversible damage to organs.
Treatments involve regular blood transfusions and drugs, but sufferers with some types of the disease often do not make it through their 40s.
The breakthrough came thanks to Nobel Prizewinning CRISPR technology, which copies a technique used by bacteria to spot and destroy specific areas of
DNA, like scissors that snip out sections of genetic code. Scientists from Nashville removed cells from bone marrow – the spongy tissue inside bones – and used CRISPR to edit a gene to encourage them to produce a protein called haemoglobin.
Haemoglobin affects the shape of red blood cells – and in sickle cell disease, defective haemoglobin is what forces them into a ‘sickle’ shape.
The healthy, haemoglobinrich cells were then transferred into Victoria’s body, replacing nearly half of the defective ones.
Her symptoms soon vanished and scientists believe she is effectively cured. She said: ‘It’s wonderful. It’s the change I’ve been waiting on my whole life.’
Dame Sally Davies, the former UK chief medical officer and an expert in sickle cell disease, said she was thrilled that the technique could soon offer hope to many sufferers.