Gene therapies that let deaf children hear bring hope —and many questions

The past few months have brought electrifying news that, for the first time, a gene therapy has provided some hearing to children born with mutations that left them deaf. Eli Lilly announced this week, for example, that a profoundly deaf boy from Morocco given its treatment as part of a clinical trial in Philadelphia can now hear. And five children in China treated similarly at younger ages gained hearing with some able to verbally communicate without their cochlear implants. Their hearing recovery, first covered by the press in October 2023, is described in detail this week in The Lancet . “It’s an enormous breakthrough,” says geneticist Karen Avraham of Tel Aviv University. Otolaryngologist and gene therapist Lawrence Lustig of Columbia University, whose lab was first to test the same approach in mice, agrees. “Other than cochlear implants, we haven’t really had any successful therapies to treat deafness,” he notes. More good news is anticipated next week when results from two other gene therapy teams, along with more data from the Lilly and China trials, will be presented on 3 February at the annual meeting of the Association for Research in Otolaryngology. The various efforts from companies and academic centers each use a virus to insert the same gene, OTOF , into the children’s inner ear so that the so-called hair cells there can sense sound and transmit it to the brain. The new deafness t...
Source: ScienceNOW - Category: Science Source Type: news