Posted on 8 February 2017
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An improved gene therapy construct has restored hearing in genetically deaf mice to impressive levels of sensitivity
Usher syndrome causes deafness in many children and progressive vision loss too. Mice with a similar mutation in the gene Ush1c are also born with a defective protein called harmonin – leading to a lack of organisation in sensory hair cell bundles that leads to profound deafness and a deterioration of light sensing cells in the retina. A number of promising gene therapy approaches are now being tested for blindness, and previous tests have enabled transfection of inner hair cells in the ear, but not the outer hair cells which are trickier to reach. Now, scientists from Boston Children’s Hospital and Harvard Medical School have designed a new and optimised vector called Anc80 which is able to reach outer hair cells in the cochlear.“We have shown that Anc80 works remarkably well in terms of infecting cells of interest in the inner ear. Outer hair cells amplify sound, allowing inner hair cells to send a stronger signal to the brain. We now have a system that works well and rescues auditory and vestibular function to a level that’s never been achieved before. With more than 100 genes already known to cause deafness in humans, there are many patients who may eventually benefit from this technology”
Normal mice sensory hair bundles (bottom left) and mutant mice (middle column). After treatment structure is restored (bottom right). Credit: Gwenaelle Géléoc and Artur Indzkykulian
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