(Medical Xpress)—Gene therapy developed at St. Jude Children's Research Hospital, University College London (UCL) and the Royal Free Hospital has transformed life for men with a severe form of hemophilia B by providing ...
Nov 20, 2014
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REGENXBIO Inc. today announced that gene transfer mediated by REGENXBIO's NAV AAV8 vectors resulted in sustained serum α-L-iduronidase (IDUA) expression, as well as correction of systemic features of MPS I, or Hurler syndrome, ...
Sep 30, 2014
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Treating the rare disease MPS I is a challenge. MPS I, caused by the deficiency of a key enzyme called IDUA, eventually leads to the abnormal accumulation of certain molecules and cell death.
Aug 26, 2014
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On 15th April is the 1st International Pompe Disease Day, a campaign to raise awareness of this rare but severe gene defect. Pompe Disease is only one of more than 40 metabolic disorders that mainly affect children under ...
Apr 15, 2014
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Enzyme therapy proves effective in treating LSDs, whilst gene therapy is an upcoming contender.
Apr 15, 2014
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Preclinical studies show that gene therapy can improve muscle strength in small- and large-animal models of a fatal congenital pediatric disease known as X-linked myotubular myopathy. The results, appearing in the Jan. 22 ...
Jan 22, 2014
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(Medical Xpress)—A gene that controls how quickly smokers process nicotine also predicts whether people who try to kick the habit are likely to respond to nicotine replacement therapy, a new study shows.
Oct 18, 2013
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New research on two promising gene therapies suggests that combining them into one treatment not only repairs muscle damage caused by Duchenne muscular dystrophy, but also prevents future injury from the muscle-wasting disease. ...
Aug 9, 2013
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The scientists who led the team that developed Glybera, the first gene therapy drug approved for use in the Western world, provide a fascinating first-person account of their pioneering work in Human Gene Therapy, a peer-reviewed ...
May 30, 2013
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An innovative anti-bacterial spray that will kill MRSA is being developed by Norwich Research Park scientists thanks to funding from the University of East Anglia.
Mar 21, 2013
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