http://www.bbc.co.uk...health-15272081
Two of the holy grails of medicine - stem cell technology and precision gene therapy - have been united for the first time in humans, say scientists.
It means patients with a genetic disease could, one day, be treated with their own cells.
[...]
Prof Lomas said if this could be developed into a therapy it would be preferable to liver transplant as the patient would not need to take immunosuppressant drugs.
He told the BBC that the technique was "ridiculously hard," yet "the potential is enormous, but only time will tell".
Further animal studies and human clinical trials would be needed before any treatment as "the key thing is safety".
For example, concerns have been raised about "induced" stem cells being prone to expressing cancer causing genes.
Prof Robin Ali, from University College London and the Medical Research Council's stem cell translational research committee, said: "It's very interesting.
"Most gene therapy is not correcting the gene, it's introducing a new copy of the gene, what's exciting is that this corrects.
Looks like we might have some gene-therapy medicines available within our lifetimes. That's a plus at least.
