Gene therapy: A ray of hope for people born with hearing loss.

VTV.vn - The results of the largest and longest-running clinical trial ever for gene therapy to treat hereditary hearing loss show the potential for sustainable hearing restoration.

(Illustrative image: Audiology)

A breakthrough in the treatment of hereditary deafness.

On April 22nd, the scientific journal Nature published the results of an international study on gene therapy for hereditary hearing loss, co-conducted by scientists from Harvard Medical School, the Massachusetts Eye and Ear Hospital in the US, and the Eye, Ear, Nose, and Throat Hospital of Fudan University (China). According to the study, the experimental gene therapy successfully restored hearing in the majority of patients with congenital hearing loss related to mutations in the OTOF gene – one of approximately 200 genes identified as being associated with hereditary hearing loss.

The study involved 42 patients at eight medical facilities in China, ranging in age from 9 months to over 32 years. Patients received a single injection of gene therapy directly into the inner ear to deliver a copy of the normal OTOF gene to damaged auditory cells. Results showed that approximately 90% of participants experienced significant improvement in hearing, with about half achieving near-normal hearing after 2.5 years of follow-up. Many patients began hearing sounds within just a few weeks of treatment.

(Photo: Shutterstock)

In particular, researchers noted a significant improvement in language and speech recognition abilities in young children. Many children who were completely unresponsive to sounds were able to imitate speech, speak short sentences, recite poetry, or sing after treatment.

Professor Zheng-Yi Chen, co-lead author of the study and an ENT surgeon at Mass Eye and Ear, said the results were truly remarkable. He emphasized that after 2.5 years, more than half of the patients achieved normal hearing, even being able to hear whispers.

According to the World Health Organization (WHO), approximately 430 million people globally suffer from hearing loss requiring rehabilitation, including about 34 million children. Around 60% of congenital deafness cases are genetic. Of these, OTOF gene mutations account for about 2-8% of inherited congenital deafness cases. Children carrying this mutation often experience complete hearing loss from birth, severely impacting their language and cognitive development if not treated early with cochlear implant surgery.

Scientists believe that DFNB9 – a form of deafness caused by the OTOF mutation – is a suitable target for gene therapy because the disease is associated with only a single gene and the auditory cells in the inner ear have not been completely destroyed. A noteworthy aspect of this study is that the therapy not only proved effective in young children but also showed some efficacy in adults – a group previously excluded from similar trials.

Opening a new era for the treatment of genetic diseases.

Experts believe that the success of OTOF gene therapy is significant not only for treating congenital deafness but also opens up new prospects for precision medicine and the treatment of other genetic diseases.

The research team stated that the current technological platform can be further adapted to address other gene mutations associated with hearing loss. Scientists have begun researching treatments for the GJB2 gene mutation – the most common cause of inherited deafness globally, but more complex than the OTOF gene mutation.

Furthermore, the US Food and Drug Administration's (FDA) approval of another OTOF gene therapy developed by Regeneron on April 23, 2026, is considered a significant milestone for this treatment field.

(Photo: AOO)

Experts believe that the लगातार positive results indicate that gene therapy is gradually moving from the research phase to practical clinical application. However, many challenges remain before the therapy can be widely deployed. Approximately 10% of patients in the study did not respond to treatment, and the scientific community has not yet clearly identified the reasons. Researchers are also investigating why young children respond better than adults. Furthermore, the cost of gene therapy treatment remains very high and requires specialized healthcare infrastructure. Expanding access to treatment to low- and middle-income countries is considered a major challenge for the future.

Another issue is the need for long-term follow-up to fully assess the safety and durability of the treatment's efficacy. In the current study, patients will continue to be followed for a total of 5 years. However, the absence of serious side effects or dose-related toxicity throughout the trial is considered a significant positive sign.

According to scientists, the future of gene therapy is entering a period of rapid development. Current advances offer hope for people with hearing loss and pave the way for the treatment of a wide range of other genetic disorders in the coming years.

Source: https://vtv.vn/lieu-phap-gene-hy-vong-cho-nguoi-khiem-thinh-bam-sinh-100260531183407766.htm