Since 2007, clinical trials using gene therapy have resulted in often-dramatic sight restoration for dozens of children and adults who were otherwise doomed to blindness. Now, researchers from the Perelman School of Medicine ...
Jul 15, 2015
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238
Researchers from Macquarie University have discovered a world-first new treatment that reverses the effects of memory loss associated with Alzheimer's disease in a study of mice with advanced dementia.
Jul 30, 2020
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375
Therapeutic use of gene editing with the CRISPR-Cas9 technique may inadvertently increase the risk of cancer, according to a new study from Karolinska Institutet, Sweden, and the University of Helsinki, Finland, published ...
Jun 11, 2018
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111
A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.
Mar 1, 2017
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219
A novel therapy developed at the University of Alabama at Birmingham ameliorates obesity and Type 2 diabetes in mice fed a high-fat diet. The therapy acts through sustained release of nitric oxide, a gaseous signaling chemical ...
Apr 29, 2022
1
132
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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86
A recently developed system for switching on the activity of genes could improve treatments for a broad range of neurological diseases. Esteban Engel, a researcher in viral neuroengineering in the Princeton Neuroscience Institute, ...
Apr 17, 2020
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344
Gene therapy has partly restored the function of the retina's cone receptors in two children who were born completely color blind, reports a new study led by University College London (UCL) researchers.
Aug 23, 2022
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2081
A contagious facial cancer that has ravaged Tasmanian devils in southern Australia isn't the first place one would look to find the key to advancing cell therapies in humans.
Nov 15, 2018
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123
OHSU scientists have discovered a naturally occurring disease in monkeys that mimics a deadly childhood neurodegenerative disorder in people—a finding that holds promise for developing new gene therapies to treat Batten ...
Jul 27, 2018
1
150
