Injections for gene illnesses

GENE-EDITING injections could one day offer hope to people suffering from devastating inherited diseases, according to research.

US scientists used the process on mice with the wasting disease Duchenne muscular dystrophy (DMD) by cutting out a tiny piece of flawed DNA.

It does not require controversial tampering with genes in eggs and sperm that are passed on to future generations.

The disease is one of the most common and severe of a group of inherited muscle-wasting conditions that affect around 70,000 people in the UK. DMD is incurable, but the teams looked at inserting functional DNA into cells.

Deleting a small piece of scrambled DNA whose presence prevents the gene working normally saw the dystrophin gene activity restored in the mice. It would enable them to achieve adequate muscle function.

Professor Peter Braude, at King’s College London, said: “The fact that it theoretically has the potential to be used to treat such a large proportion of DMD patients (83 per cent) makes it exciting.”