The long-term safety of gene transfer to treat neovascular age-related macular degeneration (NVAMD), and the production of two therapeutic proteins encoded by those genes for at least 2.5 years in the eyes of patients with ...
Oct 20, 2016
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Extensive biosafety studies of hematopoietic stem cell (HSC) gene therapy, intended to replace a protein that patients with the inherited disease mucopolysaccaridosis I (MPS I) cannot produce, support clinical testing of ...
Oct 6, 2016
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Recent advances in gene editing technology, which allows for targeted repair of disease-causing mutations, can be applied to hematopoietic stem cells with the potential to cure a variety of hereditary and congenital diseases. ...
Sep 23, 2016
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Transplantation of therapeutic stem cells directly into the central nervous system (CNS) is a promising new approach to treating the neurological effects of lysosomal storage diseases (LSD), a group of at least 50 different ...
Sep 21, 2016
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Robert MacLaren, Professor of Ophthalmology. assisted by Dr Thomas Edwards, Nuffield Medical Fellow, used the remotely controlled robot to lift a membrane 100th of a millimetre thick from the retina at the back of the right ...
Sep 13, 2016
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A new, long-term study examined the antibody response to natural infection with adeno-associated virus (AAV) in chimpanzees for the purpose of characterizing the broad-based immune responses that could reduce the effectiveness ...
Aug 11, 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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A new gene therapy approach designed to replace the enzyme that is deficient in patients with the inherited neurodegenerative disorders Tay-Sachs and Sandhoff diseases successfully delivered the therapeutic gene to the brains ...
Jul 28, 2016
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New safety data from a study of patients with advanced Parkinson's disease five years after gene transfer-mediated delivery of the neuroprotective factor neurturin directly to patients' brains reveal no serious adverse events ...
Jul 27, 2016
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The encouraging results of early stage clinical studies and the tremendous amount of preclinical data demonstrating the feasibility and promise of gene therapy to treat disorders of the central nervous system (CNS) are driving ...
Jul 25, 2016
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