Treatment effective in improving hearing in five deaf children

gene therapy can solve the Hereditary deafnessAt least this is what the preliminary data from a trial that is still ongoing and that has already been done suggests Hearing restored To five children.

More than 150 genes have been linked to inherited deafness; one of them is, OTOFis the gene that encodes otoferlin, a protein essential for the transmission of sound signals to the inner ear. People with mutations in this gene suffer Congenital hearing lossSomething that can be cured thanks to gene therapy.

In January this year, a team from Fudan UniversityDirected in Shanghai by Yilai Shu, published in Knife An interesting discovery about gene therapyGiven to children aged 1 to 8 years, there was a significant improvement Auditory function In one ear.

The treatment introduces the OTOF transgene into the DNA using adeno-associated virus (AAV) as a vector to produce normal Ottoferlin And, therefore, hearing is possible.

Preliminary data on five children

From this experience, these researchers have improved the original protocol and, as they explain In an article published yesterday Naturopathyhas given gene therapy to five children aged 1 to 11 years, with a single injection in both ears in a minimally invasive procedure. Hereditary deafness. none of them received cochlear implant,

Preliminary data indicate that the simultaneous administration of the treatments not only restored the hearing ability of the children (two girls and three boys), but also Improved speech and made them worthy Detecting and determining the position of soundand even to differentiate React to music by dancing,

Treatment and follow-up for 26 weeks was done in a specialized center Otorhinolaryngology And Ophthalmology from Fudan University.

It was decided to administer the therapy bilaterally Reduce the risk of participants developing neutralizing antibodies against the virus which carries the gene (AAV) after initial exposure to the vector.

Studies have not shown an increase Adverse effect one of two Toxicity Despite the dual treatment being dose-limiting, it shows similar efficacy to unilateral administration which has already been published.

Promising results

Preliminary results from this trial add to other early experimental and clinical work the promise ofAs they indicate substantial improvement in hearing thresholds and speech perception in treated children, details Nature In a report on the study.

Too Assurance of the absence of adverse effects paves the way for wider application of gene therapy in the treatment of severe diseases and various forms of Genetic deafness,