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Prospects For Combined Gene and Stem Cell Therapy

Here is news of a technology demonstration that hints the future of medicine, "proving in principle that a human genetic disease can be cured using a combination of gene therapy and induced pluripotent stem (iPS) cell technology ... 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 ... After taking hair or skin cells from patients with Fanconi anemia, the investigators corrected the defective gene in the patients' cells using gene therapy techniques ... They then successfully reprogrammed the repaired cells into induced pluripotent stem (iPS) cells ... Since bone marrow failure as a result of the progressive decline in the numbers of functional hematopoietic stem cells is the most prominent feature of Fanconi anemia, the researchers then tested whether patient-specific iPS cells could be used as a source for transplantable hematopoietic stem cells. They found that [the] cells readily differentiated into hematopoietic progenitor cells primed to differentiate into healthy blood cells. ... 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."

Link: http://www.sciencedaily.com/releases/2009/05/090531141321.htm

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