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Deafness. four children were treated with gene therapy

Four children deaf from birth have regained almost normal hearing after being treated with gene therapy, scientists from Shanghai’s Fudan University announced at a specialized conference held in Brussels at the end of October. This is the first time that humans have been restored to feeling through genetic modification.

“It’s huge and a world first that gives hope that one day we’ll be able to treat other genetic causes of deafness.”, says Nathalie Laundon, who heads the pediatric audiophonology department at the Necker-Enfants Malades Hospital in Paris. A very rare genetic mutation deprived these children of otoferrin, a protein needed for communication between sensory hair cells and the auditory nerve in the inner ear. Adding the rescue gene otoferlin to these cells using a harmless viral vector restored the children’s hearing within weeks.

This success is explained by several reasons. “First, this…

Source: Le Figaro

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