Can you imagine going from not being able to hear anything at all to hearing whispers? This is what a team of researchers at Children’s Hospital in Boston (USA) has achieved using a gene therapy vector developed by Massachusetts Eye and Ear in an experiment with mice.
The animal model used in the experiment were mice that reproduced Usher syndrome, an inherited disease that causes deafness or hearing loss from birth, and impaired or loss of vision.
Scientists showed several years ago that gene therapy – in which faulty genes are replaced by healthy genes – would be able to restore hearing slightly. Now, they have managed to improve the therapy until the mice could hear below 25 decibels , which we could extrapolate to almost a whisper.
After once again improving gene therapy with a new vector to transport genes, they have managed to penetrate to the inner ear ; that is, the genes have been directed to the most distant sensory cells of the inner ear and have managed to restore the hearing of the mice (treated as soon as they are born), as well as improve other symptoms of Usher syndrome such as vestibular and balance problems.
“ This strategy is the most effective we have tried. We now have a system that works well and rescues auditory and vestibular function to a level that has never been achieved before,” explains Gwenaëlle Géléoc, leader of the work.
The treatment has worked only in newborn rodents, not in mice that are several days old.
“Anything that could stabilize or improve native hearing at a young age would give a huge boost to a child’s ability to learn and use spoken language ,” says Margaret Kenna, a specialist in genetic hearing loss at Children’s Hospital Boston and also study coatura.
“This is a landmark study demonstrating for the first time that by delivering the correct genetic sequence to large numbers of sensory cells in the ear, we can restore hearing and balance to near normal levels ,” concludes Jeffrey R. Holt, co-author of the job.
The study has been published in the journal Nature Biotechnology.
