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 Ryan Cross, Endpoints News | 09.02.2026
The deaths of two children who received CRISPR therapies in China have rattled scientists who work on similar genetic medicines in the US, adding new fuel to a yearslong debate about the danger of the gene therapy field’s most-used delivery...
By Emily Baumgaertner Nunn, The New York Times | 09.08.2026
It is one of biology’s most ingenious rhythms: Every baby is born young. No matter how much wear and tear their parents’ DNA accumulated over their lifetimes, reproduction erases the molecular scars, handing a clean slate to the next generation...
What We Inherit by Sam Trejo and Daphne O. Martschenko; and Original Sin by Kathryn Paige Harden have a great deal in common, with substantial differences in style and emphasis. Both books attempt, in different ways, to show the reader just where current research into human genomics stands, where that might be going, and why the authors are interested. In short, the influence of genomic research on our societies is already large and seems likely to change them in ways...
By Dan Falk, Nautilus | 08.20.2026
The pseudoscience of eugenics, founded on the mistaken notion that nature envisions some “right” combination of genes, and that selective breeding can improve the human race, is making a comeback. While the heyday of eugenics was a century ago, the...