Fixing or replacing faulty genes has emerged as a key to unlocking cures for numerous devastating diseases. But if the new, engineered genes can't find their way into the patient's genomic sequence, they won't help.
Oct 2, 2018
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A team at the University of Manchester have developed a novel stem cell gene therapy approach to treat children with a devastating genetic disease. The approach is currently being developed for clinical trial in patients ...
Jul 6, 2018
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Toby Willis gave up on medical miracles long ago.
Apr 10, 2018
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Zebrafish, roundworms, fruit flies, mice, rats, rabbits, dogs, cats, pigs, and monkeys provide steppingstones to clinical trials to evaluate new treatments for people. The value of animal studies continues, even after a new ...
Mar 30, 2018
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Localized gene delivery to diseased joints to achieve sustained drug production at the site of osteoarthritis or rheumatoid arthritis is gaining momentum, with clinical trials underway in the U.S. and the first arthritis ...
Jan 22, 2018
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Research carried out by academics at Northumbria University, Newcastle could lead to improvements in treating patients with diseases caused by mutations in genes, such as cancer, cystic fibrosis and potentially up to 6,000 ...
Sep 19, 2017
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Based on the encouraging safety data that has emerged from multiple clinical trials that used different gene transfer approaches to improve heart function in patients suffering from heart failure with reduced ejection fraction, ...
Jun 6, 2017
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Two weeks and several political disasters ago, the House of Representatives passed the American Health Care Act of 2017, and soon lists of "pre-existing conditions" festooned news feeds. We all ticked off a few. But the lists, ...
May 22, 2017
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Researchers are reporting the highest and most sustained levels to date of the essential blood-clotting factor IX in patients with the inherited bleeding disorder hemophilia B. After receiving a single dose of an experimental ...
Dec 3, 2016
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A Washington State University researcher has developed a way to reduce the development of cancer cells that are an infrequent but dangerous byproduct of gene therapy.
Nov 4, 2016
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