OHSU scientists have discovered a naturally occurring disease in monkeys that mimics a deadly childhood neurodegenerative disorder in people—a finding that holds promise for developing new gene therapies to treat Batten ...
Jul 27, 2018
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After three decades of hopes tempered by setbacks, gene therapy—the process of treating a disease by modifying a person's DNA—is no longer the future of medicine, but is part of the present-day clinical treatment toolkit. ...
Jan 11, 2018
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Researchers at Fred Hutchinson Cancer Research Center have identified a specific subset of adult blood stem cells that is exclusively responsible for repopulating the entire blood and immune system after a transplant.
Nov 1, 2017
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For many patients, participating in gene therapy clinical trials isn't an option because their immune system recognizes and fights the helpful virus used for treatment. Now, University of Florida Health and University of ...
Jun 13, 2017
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Investigators from Whitehead Institute, the Ragon Institute of MGH, MIT and Harvard and the Broad Institute of MIT and Harvard have used CRISPR-Cas9 gene-editing technology to identify three promising new targets for treatment ...
Dec 19, 2016
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Researchers are closer to finding a better way to treat children with a rare metabolic disorder called MPS I. It is caused by a deficiency of the key enzyme IDUA needed to break down complex sugars in cells. MPS I eventually ...
Jul 28, 2016
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Gene therapy, whereby a patient's disorder is treated by inserting a new gene, replacing a defective one, or disabling a harmful one, suffered a setback in 1999, when Jesse Gelsinger, an 18-year-old with a genetic liver disease, ...
Jan 28, 2016
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An Australian scientist said Wednesday he had discovered a way to turn the HIV virus against itself in human cells in the laboratory, in an important advance in the quest for an AIDS cure.
Jan 16, 2013
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Cold viruses generally get a bad rap—which they've certainly earned—but new findings by a team of scientists at the Salk Institute for Biological Studies suggest that these viruses might also be a valuable ally in the ...
Oct 16, 2012
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Researchers have demonstrated that a refined gene therapy approach safely restores the immune systems of some children with severe combined immunodeficiency (SCID). The rare condition blocks the normal development of a newborn's ...
Sep 11, 2012
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