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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UT Southwestern Medical Center researchers have deciphered how a small protein made by the human immunodeficiency virus (HIV) that causes AIDS manipulates human genes to further its deadly agenda.
Jan 27, 2016
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The conclusion drawn from a recent study that insertion of adeno-associated virus 2 (AAV2) into human DNA causes mutations leading to the development of hepatocellular carcinoma (HCC) was resoundingly rejected by leading ...
Jan 6, 2016
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Mitchell Finer, PhD, a scientist with expertise in biochemistry and molecular biology, who has had an integral role in the success of numerous biotechnology companies, including most recently as chief scientific officer of ...
Oct 22, 2015
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Highly anticipated phase III clinical trial results of Spark Therapeutics's gene therapy to treat visual impairment are due by year-end and could have profound implications for the broader gene therapy field, leading to the ...
Oct 15, 2015
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A gene therapy approach to treating the progressive muscle wasting disorder Duchenne muscular dystrophy (DMD) that does not replace the mutated DMD gene but instead delivers the gene for ITGA7, a protein in skeletal muscle, ...
Sep 29, 2015
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Mice lacking the protein retGC1, which is deficient in humans suffering Leber congenital amaurosis-1 (LCA1), a disorder that causes severe visual impairment beginning in infancy, received gene therapy to replace retGC1 and ...
Aug 31, 2015
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Chimeric antigen receptor (CAR) T cells, which can specifically recognize and target tumor cells, have resulted in complete responses in patients with leukemia, and although equally promising for treating lymphoma, obstacles ...
Aug 24, 2015
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Canada's first human gene therapy trial for eyes—the replacement of a faulty gene with a healthy one—is now underway at the Royal Alexandra Hospital to preserve and potentially restore vision for people with a genetic ...
Jun 29, 2015
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A team from China grabbed the headlines last month when it announced it had edited DNA in the nucleus of human embryos. Whatever the ethics of such research, the breakthrough raises the question of just how far we will take ...
May 25, 2015
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