Researchers have generated stem cells from patients with a rare genetic disease, corrected the cells' defect and reprogrammed to pluripotency, a paper published online in Nature reports. The teams have effectively put the cells through gene therapy and, although they did not use them to treat patients, the results have potential value for cell therapy applications.
Fanconi anaemia (FA) is a genetic disorder, manifesting in skeletal anomalies, increased incidence of solid tumours and leukaemias, and bone marrow failure. Juan Carlos Izpis?lmonte and colleagues use somatic cells from patients with the disease and corrected the genetic defect by gene therapy. They go on to reprogram them to pluripotency and generate patient-specific induced pluripotent stem (iPS) cells.
The group generated 19 iPS cell lines that appear indistinguishable from human embryonic and iPS cells from healthy individuals. The corrected FA-specific iPS cells can give rise to disease-free haematopoietic progenitors.
Juan Carlos Izpisua Belmonte (The Salk Institute for Biological Studies, La Jolla, CA)
Abstract available online.
(C) Nature press release.
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