UCLA researchers led by Dr. Donald Kohn have created a method for modifying blood stem cells to reverse the genetic mutation that causes a life-threatening autoimmune syndrome called IPEX. The gene therapy, which was tested ...
Jan 11, 2019
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Viral vectors used to deliver gene therapies undergo spontaneous changes during manufacturing which affects their structure and function, found researchers from the Perelman School of Medicine at the University of Pennsylvania ...
Oct 18, 2018
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A new gene therapy for treating an inherited form of Amyotrophic Lateral Sclerosis has been given an Orphan Drug designation by the European Medicines Agency. The therapy is already in preclinical development at EPFL.
May 23, 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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(Medical Xpress)—A large team of researchers from the U.S. and Canada has conducted a randomized double-blind study of the effectiveness of an HIV vaccine and has found it to be ineffective in suppressing the virus. In ...
Researchers from Genethon, the AFM-Telethon laboratory, Inserm (UMR) and the Royal Holloway University of London demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne muscular dystrophy. Indeed, ...
Jul 25, 2017
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Viruses have evolved to be highly effective vehicles for delivering genes into cells. Seeking to take advantage of these traits, scientists can reprogram viruses to function as vectors, capable of carrying their genetic cargo ...
Jun 26, 2017
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Researchers at UC Berkeley have found unexpected effects of viral infections, a discovery that may explain why viruses can make people feel so lousy.
Jun 9, 2017
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Overwhelming evidence from the biomedical literature shows that adeno-associated virus 2 (AAV2), a viral vector often used to deliver therapeutic genes, is not associated with cancer and, in fact, may protect against cancer. ...
Feb 23, 2017
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For the first time ever, a single flexible fiber no bigger than a human hair has successfully delivered a combination of optical, electrical, and chemical signals back and forth into the brain, putting into practice an idea ...
Feb 22, 2017
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