Scientists have created stem cells from patients suffering from 10 incurable diseases, from Down syndrome to diabetes and Parkinson’s – immortal cells that might one day be turned into repair material for wasting muscles or damaged brains.
The Harvard University-led team has taken skin and bone marrow cells from diseased patients and re-programmed those cells to behave like cells from days-old embryos.
The feat allows scientists for the first time to watch muscular dystrophy and other diseases unfold in a petri dish, “that is, to watch what goes right or wrong,” said Doug Melton, co-director of the Harvard Stem Cell Institute. The cells will also allow researchers to screen new drugs to treat the diseases.
“In these complex genetic diseases, we’re so ignorant at the moment we don’t even know when a patient gets diabetes if they all get it the same way,” Melton said. “There could be 50 different ways to get Type 1 diabetes.” The stem cell lines could help researchers hone in on exactly which mutations are responsible and find “the weak point where you could try to prevent, or treat it.”
“We have good reason to believe that this will make it possible to find new treatments, and eventually drugs, to slow or even stop the course of a number of diseases,” Melton said.
The new cells are “pluripotent” cells that can be coaxed into making any tissue in the human body, and can grow forever.
It may one day be possible to start with a single stem cell, fix the genetic defect in the cell, and then use it to make healthy muscle, brain or other tissue that could be transplanted back into the patient. “We’re many years from that… but that in itself is exciting,” said Dr. Duncan Stewart, CEO of the Ottawa Health Research Institute and professor of medicine at the University of Ottawa.
But, “it’s early days. These cells aren’t perfect,” Stewart said. As well, no one is recommending “that anyone start sticking these cells into patients,” he said.
The new stem cell lines will be distributed virtually free to any scientist who wants them, in the hope it will speed research. Researchers are trying to make insulin-producing pancreatic cells and immune cells from the new stem cells.
The new research is published in the journal Cell.
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