Children find hearing through gene therapy

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Children find hearing through gene:

Each year, nearly 800 infants are born with bilateral deafness in France, according to Inserm figures. Consequently, These hearing disorders from birth are generally of genetic origin. Consequently, “”Nearly 130 genes involved have already been identified: their mutation causes anomalies of the hearing system that cause the. For example, deficit “specifies the organization. Consequently, In Sweden. Moreover, researchers have developed gene therapy capable of restoring one of these changes: tests have shown that this allows children to find hearing. Therefore, Their results are presented in Nature Medicine.

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Congenital deafness: the role of the OTOF gene – Children find hearing through gene

For their work, these scientists from the Karolinska Institute, located in Sweden, recruited ten patients aged 1 to 24, distributed in five Chinese hospitals, who collaborated in this study. However, All participants suffered from a genetic form of deafness or severe hearing children find hearing through gene impairment caused by mutations in the OTOF gene. Consequently, “”These mutations lead to an otoferline deficit. For example, a protein essential to the transmission of hearing signals from ear to the brain “specify the authors. In their test, they tested the effects of gene therapy. It consisted in using an adeno-associated virus (VAA) synthetic to administer a functional version of the OTOF gene in. the inner ear. The process was carried out by injection. through a membrane located at the base of the cochlea, part of the inner ear.

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A significant improvement in hearing through gene therapy – Children find hearing through gene

The effect of gene therapy was rapid and the majority of patients recovered part of the hearing after only a month “they observe. During the six -month follow -up. the researchers noted a considerable hearing improvement in all participants: the average volume of perceptible sounds going from 106 decibels to children find hearing through gene 52. “The youngest patients, especially those aged five to eight, responded better to the treatmentthey specify. One of the participants. a seven-year-old girl, quickly recovered almost all of her hearing and was able to converse daily with her mother four months later. “ No serious side effects have been reported.

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Other genes could become a therapeutic target

This is a major advance in the genetic treatment of deafness, which can change the lives of children and adults “considers Maoli Duann, co-author of the study and teacher at the Karolinska Institute. For her, this targeting gene therapy OTOF is only the beginning. “”With other researchers. we extend our work to other more common genes responsible for deafness, such as GJB2 and TMC1, she develops. These genes are more complex to treat, but animal studies have so far given promising results. We are convinced that patients with different children find hearing through gene forms of genetic deafness will one day benefit from treatment.”

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Hearing loss: a French test confirms the interest of gene therapy

They are not the only scientists to be interested in gene therapy to treat deafness. In France, the sensorion biotechnology company published a press release on gene therapy targeting hearing on July 1. Called Sens-501, the treatment also acts on the OTOF gene. “”The three-month results of a young child treated with Sens-501 in the first cohort attest to an early. promising improvement in his hearingsaid the press release. The second cohort, evaluating a higher dose of meaning-501, is underway and recruitment is about to be finalized. “ These works are carried out with the Pasteur Institute and the Necker Hospital Malades.

children find hearing through gene

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