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| Dario Fauza, MD |
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| Induced pluripotent stem cells derived from skin cells taken from a patient with Parkinson's disease. (Image: Courtesy Hyun Park, PhD.) |
Last December, researchers led by George Daley, MD, PhD, and In-Hyun Park, PhD, of Children's Stem Cell Program, reported converting skin cells from an adult volunteer into cells that look and act like embryonic stem cells. These cells, called induced pluripotent stem cells (iPS), are created through genetic reprogramming and can potentially form any cell type in the body. Now, in collaboration with the Harvard Stem Cell Institute (HSCI), Daley and Park have created iPS cell lines modeling 10 different diseases, allowing researchers to watch the diseases as they unfold in developing tissue and perhaps discover new treatment possibilities.
Daley had long sought cell-based models to better understand human diseases, with his particular interest being blood disorders. The new iPS cells, to be shared freely through a repository housed at the HSCI, include lines with Parkinson's disease, Type I diabetes, Huntington's disease, Down syndrome, a form of combined immunodeficiency ("bubble boy disease"), Lesch-Nyhan syndrome, Gaucher's disease and two forms of muscular dystrophy. "The original embryonic stem cell lines are generic and allow you to ask only basic questions,"Daley told reporters at a press conference in August. "These new lines are just the beginning for studying thousands of diseases in a petri dish."
Described online on August 6 in the journal Cell, the iPS lines were developed from the cells of patients ranging in age from one month to 57 years old. But Daley emphasizes that he's far from ready to abandon experiments with embryonic stem cells. Such research needs to continue in tandem with approaches that reprogram adult cells, he says, to bring cell-based therapies to the clinic as quickly as possible.
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