Gene therapy to the inner retina prevented blindness in a mouse model of the neuro-degenerative disorder CLN3 Batten disease. Adeno-associated viral (AAV)-mediated expression of the human CLN3 gene led to significant survival ...
Aug 11, 2020
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The Editor-in-Chief of Human Gene Therapy, the first journal devoted to the field of gene therapy, and one of the world's leading experts on gene therapy have co-authored a new editorial, Moving Forward After Two Deaths in ...
Jul 2, 2020
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An initial trial in patients indicates that a new genetic treatment for complete color blindness, developed by research groups based in Tübingen and Munich is safe. Preliminary evidence for its efficacy has also been obtained.
May 1, 2020
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In an unprecedented effort, hundreds of thousands of researchers and clinicians worldwide are locked in a race against time to develop cures, vaccines, and better diagnostic tests for COVID-19, the illness caused by the virus ...
Apr 24, 2020
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Gene therapy investigators can greatly benefit from the resources and services provided by the National Gene Vector Biorepository (NGVB), housed at the Indiana University School of Medicine. These include 93 unique reagents, ...
Mar 27, 2020
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Children (and adults) with hemophilia are slow to form blood clots, so are at constant risk for uncontrolled bleeding. Even when the skin isn't broken, a fall or a simple toe stub can become a serious medical issue: internal ...
Mar 10, 2020
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A new study has shown that gene therapy to treat one of the most common hereditary disorders, aldehyde dehydrogenase type 2 (ALDH2) deficiency, may prevent increased risk for esophageal cancer and osteoporosis associated ...
Feb 12, 2020
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Mice born blind have shown significant improvement in vision after undergoing a new gene therapy developed by a team of Japanese scientists.
Feb 7, 2020
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One challenge facing researchers who study brain diseases is that for understandable reasons it is difficult to obtain human brain tissue for experiments. For that reason, experimental models are used, such as rodent studies, ...
Jan 9, 2020
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In a radical new approach to treat cocaine addition, researchers at the Mayo Clinic are seeking approval for first-in-human studies of a single-dose gene therapy. To support the safety and efficacy of this approach they have ...
Jan 7, 2020
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