Gene therapy: mice cured of deafness

Publié le 16 Feb, 2017

A team of scientists at Harvard University, led by Doctor Jeffrey Holt, has succeeded in restoring hearing to mice suffering from Usher’s syndrome, thanks to gene therapy. Their study, which was published in the Nature Biotechnology journal, is deemed to be a “major breakthrough” because “it is the first time that we have managed to restore hearing to this level,” explained Doctor Jefferey Holt. Gene therapy tested here paves the way for similar treatment in humans “in the foreseeable future”.


Usher’s syndrome is linked to a genetic mutation which leads to defective cilia in the inner ear. Scientists used a synthetic virus to target and then correct this mutation. The results have been assessed as “spectacular”. “Profoundly deaf mice have been capable of hearing 25 sounds at 25 decibels, which corresponds to a murmur”. The study must be extended long-term. Currently, the mice have been followed up for six months. Furthermore, scientists want to pinpoint the right time to treat deafness. Attempts have worked in mice treated from birth but have failed in mice treated ten days later.


There are different forms of deafness, half of which can be linked to genetic errors. A different gene therapy will have to be developed for each error.

Radio Canada, Alain Labelle (7/02/2017)

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