Taking the next step in a series of breakthrough stem-cell experiments, scientists have cured sickle- cell anemia in mice by rewinding their skin cells to an embryonic state and manipulating them to create healthy, genetically matched replacement tissue.
After the repaired cells were transfused into the animals, they soon began producing healthy blood cells free of the crippling deformities that deprive organs of oxygen, scientists from the Whitehead Institute for Biomedical Research in Cambridge, Mass., and the University of Alabama at Birmingham reported yesterday.
The experiments, published online by the journal Science, confirmed the therapeutic potential of a new class of reprogrammed stem cells, which can be custom-made for patients without creating and destroying human embryos.
The strategy should work to treat hemophilia, thalassemia and severe combined immunodeficiency disease, the "bubble boy" disease, according to researchers, and might also apply to disorders linked to mutations in a single gene, such as muscular dystrophy and cystic fibrosis.
Scientists hope to use a similar approach to create cardiac cells to treat heart attack patients or nerve cells that could cure spinal cord injuries. Finding an abundant source of stem cells that could be used as a personalized biological repair kit is the goal of regenerative medicine.
The technique is a few years away from being used to treat humans, scientists said. Before it could be tried, several rounds of animal experiments would need to be done. Researchers also will need to overcome key technical hurdles, including finding a way to reprogram adult cells without using genes and viruses that could cause cancer.
But as a proof of principle, the study is certain to attract more researchers into studying the new class of induced pluripotent stem cells, or iPS cells.
"There's going to be this tsunami," said Paul Simmons, director of the Center for Stem Cell Biology at the University of Texas Health Science Center in Houston. "One would have to predict that the pace of observations made using iPS cells is going to rise exponentially."
The study is the latest in a string of significant experiments published in the past five months involving a new approach of reprogramming adult cells so they are capable of growing into any type of tissue in the body. They have captivated researchers, ethicists and politicians looking for an alternative to embryonic stem cells, which can be difficult to work with and are fraught with moral problems.
Japanese researchers pioneered the new method, which involves turning on four genes that are dormant in adult cells but active in days-old embryos. The cells essentially "forget" they have become skin cells and behave like embryonic stem cells. Because they are derived from a patient's own cells, there is no risk of tissue rejection.
In June, three research teams showed that the technique worked reliably in mice. In November, two groups demonstrated that it also worked with human cells. But it remained to be seen whether the cells could serve as the raw material to grow replacement parts for patients.
Karen Kaplan writes for the Los Angeles Times.