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Gene Therapy Breakthrough for Auditory Neuropathy Offers New Hope

16:02, 18 Desember

Diedit oleh: Надежда Садикова

The discovery that a newborn has hearing loss can be a deeply painful experience for any parent. This emotional burden is often magnified for deaf parents, who face a higher likelihood of having a child with hearing impairment due to genetic factors. The realization that their child may face similar challenges they've encountered can be particularly heart-wrenching, adding layers of complexity to an already sensitive situation.

Recent advancements in science have shed light on the genetic underpinnings of hearing loss, identifying mutations in genes such as OTOF, DFNA5, REST, and EYA4 as potential culprits. These genetic alterations can lead to auditory neuropathy spectrum disorder (ANSD), a form of hearing impairment that primarily affects infants and young children. Gene therapy targeting OTOF mutations offers new possibilities for addressing hearing loss in children.

Dr. Shree Rao, a leading cochlear implant specialist, explains how gene therapy brings hope to families grappling with inherited hearing loss. Auditory neuropathy is a rare genetic condition where the inner ear detects sound, but the transmission of these sound signals to the brain is impaired, resulting in hearing difficulties.

Cochlear implants can offer a viable solution for individuals with auditory neuropathy, particularly beneficial for children with an intact auditory nerve. These devices provide opportunities for developing hearing and speech skills. The versatility of cochlear implants highlights their importance in managing this complex condition.

Gene therapy for auditory neuropathy represents an exciting frontier in treatment, with groundbreaking research currently underway in the United Kingdom. The world's first application of gene therapy for auditory neuropathy was recently witnessed in the case of Opal Sandy from Oxfordshire. Born deaf due to auditory neuropathy, Opal underwent a gene therapy where a genetic infusion was given directly into her right ear.

Within just four weeks of the infusion, Opal demonstrated a response to sound. By the 24-week mark post-treatment, her treated ear showed hearing levels close to normal for soft sounds. Now at 18 months old, Opal can respond to her parents' voices and has begun to communicate verbally, marking significant improvements in her auditory perception.

While gene therapy shows promise, it faces challenges. The treatment is still experimental, undergoing clinical trials to establish safety and efficacy. Moreover, its effectiveness is specifically targeted at hearing impairment caused by OTOF gene mutations and may not address other genetic causes or environmental factors.

Cochlear implants remain a widely available solution for many individuals with severe to profound hearing loss. For those considering treatment options, consulting with an experienced specialist is crucial. Dr. Shree Rao offers expert guidance on the most suitable interventions for unique cases.

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