11 July 2013 Last updated at 14:04 ET By James Gallagher Health and science reporter, BBC News

A disease which robs children of the ability to walk and talk has been cured by pioneering gene therapy to correct errors in their DNA, say doctors.

The study, in the journal Science, showed the three patients were now going to school.

A second study published at the same time has shown a similar therapy reversing a severe genetic disease affecting the immune system.

Gene therapy researchers said it was a "really exciting" development.

Both diseases are caused by errors in the patient's genetic code - the manual for building and running their bodies.

Babies born with metachromatic leukodystrophy appear healthy, but their development starts to reverse between the ages of one and two as part of their brain is destroyed.

Wiskott-Aldrich syndrome leads to a defective immune system. It makes patients more susceptible to infections, cancers and the immune system can also attack other parts of the body.

The technique, developed by a team of researchers at the San Raffaele Scientific Institute in Milan, Italy, used a genetically modified virus to correct the damaging mutations in a patient's genes.

The hype around gene therapy was huge - nipping into the genome and tweaking a bit of DNA was supposed to change medicine.

Originally posted here:
Gene therapy trial 'cures children'

Comments are closed.