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Researchers get closer to gene therapy that would restore hearing for congenitally deaf
CORVALLIS, Ore. – Researchers at Oregon State University have found a key new piece of the puzzle in the quest to use gene therapy to enable people born deaf to hear.
The work centers around a large gene responsible for an inner-ear protein, otoferlin. Mutations in otoferlin are linked to severe congenital hearing loss, a common type of deafness in which patients can hear almost nothing.
“For a long time otoferlin seemed to be a one-trick pony of a protein,” said Colin Johnson, associate professor of biochemistry and biophysics in the OSU College of Science. “A lot of genes will find various things to do, but the otoferlin gene had appeared only to have one purpose and that was to encode sound in the sensory hair cells in the inner ear. Small mutations in otoferlin render people profoundly deaf.”
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Anti-hyperlipidemia drug improves brain connectivity in mouse models of schizophrenia
New therapies that improve connectivity and circuitry in the brain of people with schizophrenia could result from the discovery by a RIKEN-led team of a potential new target for drugs to treat the psychiatric disorder
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Treatment for schizophrenia has not changed much in 60 years. Doctors still generally prescribe medicines designed to tame psychosis by blocking neurotransmitter signaling in the brain. While the drugs ease symptoms such as delusions and hallucinations, they leave many other aspects of the illness untreated and their side effects can be off-putting to many patients. Researchers are thus keen to find new targets for drugs.
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A study of spatial learning in mice shows that exposure to new experiences dampens established representations in the brain’s hippocampus and prefrontal cortex, allowing the mice to learn new navigation strategies. The study, published in Nature, was supported by the National Institutes of Health.
“The ability to flexibly learn in new situations makes it possible to adapt to an ever-changing world,” noted Joshua A. Gordon, M.D., Ph.D., a senior author on the study and director of the National Institute of Mental Health, part of NIH. “Understanding the neural basis of this flexible learning in animals gives us insight into how this type of learning may become disrupted in humans.”