Researchers from Case Western Reserve University School of Medicine and The Cleveland Clinic designed a way to screen brain tumor cells and identify potential drug targets missed by other methods. The team successfully used ...
Jul 5, 2017
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Researchers have uncovered 30 genes that could, one day, serve as therapeutic targets to reverse Rett syndrome, a rare neurological disorder that affects only girls and is a severe form of an autism spectrum disorder.
Jan 30, 2017
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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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Researchers from the Monash Biomedicine Discovery Institute in Melbourne have identified two new genes linked to a major cause of mitochondrial disease. Their research opens the way for better genetic diagnosis of the disease ...
Sep 15, 2016
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We often think of the body as a machine, with every part—right down to each single gene—working with optimal efficiency to keep us healthy and disease-free. Take a single bolt out of that machine and something could go ...
Aug 10, 2016
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Diseases of the eye that cause vision loss and blindness, especially neurodegenerative disorders affecting the retina, are ideal targets for gene therapy, including gene replacement and promising corrective gene editing strategies. ...
Jun 22, 2016
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Combining the therapeutic potential and advantages of existing oligonucleotide-based approaches to turn off disease-related genes, a type of single-stranded silencing RNAs (ss-siRNAs) has shown significantly improved potency ...
May 12, 2016
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The MYC oncogene intervenes in many types of cancer, some of which are very aggressive; researchers suspect that controlling its activity could open avenues to new treatments. However, MYC is an especially complex oncogene ...
Jan 5, 2016
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The gene p53 has been described as the "guardian of the genome" due to its prominent role in preventing genetic mutations. More than half of all cancers are thought to originate from p53 mutations or loss of function, and ...
Dec 14, 2015
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(Medical Xpress)—A number of lung diseases are resistant to, or only marginally handled by, conventional therapies. Thanks to the discovery of numerous genetic targets, gene therapy provides an alternative or complementary ...
