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Stem cell-gene therapy technique cures genetic disease in human cell line
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Stem cell colony
Scientists are one step closer to creating a gene therapy/stem cell combination to combat genetic diseases. With work, this research may lead to not only curing the disease, but also repairing the damage left behind.
While gene therapy is a burgeoning field that has shown great results in treating genetic disorders, many of those diseases leave behind heavily damaged tissue that the body is unable to repair. So even if the disease is completely eradicated, quality of life may not necessarily improve, and without help, health can still continue to deteriorate.
Since stem cell research began, there has been a hope that use of those cells may help alleviate some of the trauma left behind by genetic diseases. While the theory has been shown to work in mice, this is the first time a human cell line has confirmed that it is possible the therapy will work for humans as well.
Researchers at the Salk Institute for Biological Studies in La Jolla, California, chose to focus on Fanconi anemia (FA), a genetic disorder that is characterized by short stature, bone marrow failure, irregularities in blood cells that can lead to clotting, and an increased risk of leukemia. Even after gene therapy and “bone marrow transplants to correct the hematological [blood] problems, patients remain at high risk of developing cancer and other serious health conditions.”
The Nature-published technique employed by Juan-Carlos Izpisúa Belmonte’s team was two-fold. First, they collected hair and skin cells from those suffering from FA. These types of cells are known as somatic cells, and can be used to create induced pluripotent stem (iPS) cells. Once a hair cell has been transformed into an iPS cell, it can be coaxed to differentiate into virtually any cell in the human body.
With the collected cell samples, Belmonte’s team applied gene therapy to fix the mutated gene that causes FA. Those repaired cells were then programmed into iPS cells that were “indistinguishable from human embryonic stem cells and iPS cells generated from healthy donors.” [EurekAlert]
Once they had successfully created iPS cells, researchers investigated if they could use the cells to repair damaged tissue. Because blood cells are some of the worst affected by FA, they “tested whether patient-specific iPS cells could be used as a source for transplantable hematopoietic stem cells. They found that FA-iPS cells readily differentiated into hematopoietic progenitor cells primed to differentiate into healthy blood cells.” [EurekAlert]
Though this is an important step forward, the research is still some time away from clinical trials in humans. iPS cells, like embryonic stem cells, are difficult to work with inside the body, for they can grow out of control and form tumors—which is, of course, counterproductive to a therapeutic technique. However, at least the theory has held. “We haven't cured a human being, but we have cured a cell," Belmonte explains. "In theory we could transplant it into a human and cure the disease." [EurekAlert]
And the group has ample funding to prove their work. In April, they were awarded $6.6 million from the California Institute Regenerative Medicine. It is a hope that the gene therapy/stem cell combination will one day lead to true cures for genetic diseases.
"If we can demonstrate that a combined iPS–gene therapy approach works in humans, then there is no limit to what we can do," says Belmonte team member Inder Verma. [EurekAlert]
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