For the first time, researchers have combined gene therapy and cellular reprogramming technologies in human cells to correct a genetic defect. After taking skin and hair cells from patients with a rare genetic disorder and fixing the aberrant mutation, the investigators successfully reprogrammed the cells to an embryonic-like state and then turned them into the very cell types that usually go awry, according to a linkurl:study; published online today (May 31) in __Nature__.
Stained FA-specific iPS cells
Image: Juan Carlos Izpisúa Belmonte
The approach should be applicable to any disorder with simple, Mendelian inheritance, noted Juan Carlos Izpisúa Belmonte, a developmental biologist at the Salk Institute in La Jolla, Calif., and the Center for Regenerative Medicine in Barcelona, who led the study. "In principle," he wrote in an email, "our approach could be used with any disease that could be corrected by gene therapy and where there is loss...
Genetically-corrected fibroblasts (above)
are reprogrammed to give rise to
disease-free blood cell precursors (below)

Image: Juan Carlos Izpisúa Belmonte
earlier this week

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