A new study demonstrates how patient-derived stem cells might one day be used to treat genetic diseases. Scientists from Cambridge, England, corrected a genetic error in stem cells derived from patients with a liver disease, and then differentiated them into liver cells. When injected into the livers of mice, the cells integrated into the organ and started functioning normally.
"They showed that the cells are able to function; that's a very big achievement and a great opportunity," says Anil Dhawan, a researcher at King's College London who was not involved in the study. The study was published today in Nature.
The research combines several techniques, including cell reprogramming and gene editing, that scientists hope will make gene therapy and cell replacement therapies a reality. While safety testing is needed before the treatment can be tested in people, researchers used technologies that left the cells "pristine," with no signs of the genetic manipulation that took place. This makes them more likely to be suitable for patients.