Gene therapy and stem cells unite
By James Gallagher,
BBC News
| 10. 13. 2011
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.
A study in Nature corrected a mutation in stem cells made from a patient with a liver disease.
Researchers said this was a "critical step" towards devising treatments, but safety tests were still needed.
At the moment, stem cells created from a patient with a genetic illness cannot be used to cure the disease as those cells would also contain the corrupted genetic code.
Scientists, at the Wellcome Trust Sanger Institute and the University of Cambridge, were working on cirrhotic liver disease.
It is caused by a change to a single pair of letters, out of the six billion which make up the genetic code.
As a result, a protein which protects the body from damage, antitrypsin, cannot escape from the liver where it is made.
The illness is one of the most common genetic diseases, affecting...
Related Articles
By Fyodor D. Urnov and Sadik H. Kassim, Nature | 04.21.2026
In February, the US Food and Drug Administration (FDA) proposed a radical rethink of how scientists, physicians and manufacturers develop personalized genetic therapies. The regulator’s suggested introduction of a ‘plausible mechanism pathway’ should increase incentives for drug companies to develop...
By Miguel Muñoz, Cadena SER | 08.04.2026
"Para ellos, una familia numerosa no solo es una preferencia personal, sino que es una obligación. Creen que tener tantos hijos como sea posible es necesario para evitar un futuro apocalíptico", aseguraba Xavier Orri, periodista y cofundador de Página Internacional...
By Ryan Knutson and Jessica Mendoza, The Journal. | 03.27.2026
Genetically engineered babies are banned in the U.S. But that isn’t stopping Silicon Valley tech titans from trying to make one. In this final installment from The Journal’s investigation into the fringes of the fertility industry, WSJ’s Emily Glazer reports...
By Antonio Regalado, MIT Technology Review | 03.30.2026
After operating in secrecy for years, a startup company called R3 Bio, in Richmond, California, suddenly shared details about its work last week—saying it had raised money to create nonsentient monkey “organ sacks” as an alternative to animal testing.
In...