More and more children are hearing thanks to advances in gene therapy for profound deafness

Enlarge / Opal Sandy (center), WHO was born completely deaf because of A rare genetic condition, can NOW hear without help For THE First of all time After receive embarrassed therapy has 11 months. She East watch with her mother, father, And sister has their House In Eynsham, Oxfordshire, on Can 7, 2024. Getty | Andrew Matthew

There are little things more comforting that videos of children with deafness earn THE ability has hear, showing them Fortunately turning their heads has THE her of their parents' voice And happily to dance has newly discovered music. THANKS has recent advances In embarrassed therapy, more children are get those soft And triumphant moments - with No hearing AIDS Or cochlear implants necessary.

HAS THE annual conference of THE American Company For Embarrassed & Cell Therapy detained In Baltimore This week, researchers watch a lot of those videos has their public of experts. On Wednesday, Larry Lustig, A otolaryngologist has Colombia University, present clinical trial data of two children with deep deafness - most severe type of deafness - who are NOW able has hear has normal levels After receive A experimental embarrassed therapy. A of THE children was 11 month old has THE time of THE treatment, marking her as THE the youngest child In THE world has date has receive embarrassed therapy For genetic deafness.

On THURSDAY, Yilai Shu, A otolaryngologist has Fudan University In Shanghai, provided A one year progress report on six children WHO were treaty In THE First of all inhuman trial of embarrassed therapy For genetic therapy. Five of THE six had their hearing restored.

That trial, as THE A Lustig present, implied to treat just A ear In all of THE children - one security precaution For such early tests. But Shu And colleagues to have Already moved on has both ears, Or bilateral treatment. After presenting A progress report on THE First of all trial, Shu present unpublished early data on five additional the patients WHO participated In THE First of all inhuman trial of bilateral treatment. All had bilateral hearing restoration And speech perception improvement.

"THE opportunity of provide THE complete complexity And spectrum of her In children born with deep genetic deafness East A phenomenon I did not to wait for has see In My lifetime," Lustig said In A declaration.

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Shu And Lustig tests are separated but THE treatments are, In wide hits, similar. Both are aiming has restoration hearing loss cause by mutation In THE OTOF embarrassed THE codes For THE protein otoferlin. Normally, otoferlin East A critical protein For to transmit her signals has THE brain, specifically playing A key role In synaptic transmission between THE ears interior hair cells And THE auditory nerve. Using empty adeno-associated virus as vectors For embarrassed delivery, THE therapies provide THE interior ear with A functional version of THE OTOF embarrassed. Once In THE ear, THE embarrassed can be translated In functional otoferlin, restoration auditory signage.

In THE trial Lustig present, THE two the patients saw A gradual improvement of hearing as otoferlin protein built up After treatment. For THE 11 months, normal levels of hearing were restored In 24 weeks of treatment. For THE second patient, A 4 years, improvements were detected has A six weeks assessment. In THE trial Shu present, children began seeing hearing improvements has three- And four weeks evaluations. THE children will continue has be follow up In THE future, which is holding a few uncertainties. It is not clear if they will, has a few indicate In their lives, need additional treatments has sustain their audience. In mouse, has less, THE treatment hard For THE duration of THE animals' lives - but they only live For A little years.

"We to wait for This has last A long time," Lustig said Wednesday. But "We don't do it know what is this going has arrive And We don't do it know if We can TO DO A second dose. But, probably, I would be guess, has a few indicate that would be to have has be done."

For NOW, THE treatment East considered at hand fruit For THE booming field of embarrassed therapy Since he targets A severe condition cause by receive...