SCIENCE:A LANDMARK study has married gene therapy with stem cell technology to "cure" a genetic disorder, in the Petri dish at least.
A research team from Spain and the United States removed skin and hair cells from six patients with Fanconi anemia, a genetic condition that can lead to leukaemia and other cancers.
Using gene therapy the scientists corrected the genetic abnormalities in the harvested cells and then reprogrammed them to become "induced pluripotent" stem (iPS) cells. In the lab, the engineered stem cells could be grown into blood precursor cells that were disease-free.
The study, published online in Nature at the end of May, offers proof of concept that a patient's own cells can be genetically treated and rebooted as stem cells. And because the cells came from the patient themselves, they would not be rejected when introduced back into the body.
"The hope in the field has always been that we'll be able to correct a disease genetically and then make iPS cells that differentiate into the type of tissue where the disease is manifested and bring it to clinic," said researcher Dr Juan-Carlos Izpisúa Belmonte. "We haven't cured a human being, but we have cured a cell. In theory we could transplant it into a human and cure the disease."
However hurdles still remain before such an approach could be used for human therapies. Currently the most efficient way to make iPS cells involves using viruses that in theory could induce tumours, but iPS techniques were improving.
"If we can demonstrate that a combined iPS-gene therapy approach works in humans, then there is no limit to what we can do," said fellow researcher Prof Inder Verma.
