Hearing loss affects over 1.5 billion people worldwide, and more than 60% of cases among children are genetically linked. Current treatments for hereditary deafness are limited, making gene therapy a promising solution.
Researchers from Mass Eye and Ear, in collaboration with Mass General Brigham, have conducted a groundbreaking study demonstrating the effectiveness of gene therapy as a treatment for hereditary deafness in children.
In the trial, which took place at the Eye & ENT Hospital of Fudan University in Shanghai, China, six children with a specific form of deafness caused by mutations of the OTOF gene were treated with the gene therapy. The results were remarkable, showing significant improvement in hearing and speech ability week by week.
This research represents the first human clinical trial of its kind, with the highest number of patients and the longest follow-up period. The findings were published in The Lancet on January 24.
The study involved careful introduction of a modified version of the OTOF gene into the inner ears of the patients using an adeno-associated virus (AAV). After 26 weeks, five out of six children showed significant hearing recovery, improved speech perception, and the ability to have normal conversations. No severe side effects were observed, with the majority of adverse events being mild and temporary.
The success of this study highlights the safety and effectiveness of gene therapies for treating hereditary deafness and suggests potential applications for other forms of genetic hearing loss. Furthermore, the use of a dual-AAV vector carrying two pieces of the OTOF gene is a notable breakthrough that opens up possibilities for treating other large genes.
“These findings mark a major milestone in the treatment of deafness. We are entering a new era in the battle against all types of hearing loss,” said Zheng-Yi Chen, DPhil, a lead researcher on the study.
The researchers plan to expand the trial to include a larger sample size and longer follow-up periods to further evaluate the therapy’s effectiveness and safety.
The study author, Yilai Shu, MD, expressed excitement about future research on other forms of genetic hearing loss, with the aim of bringing effective treatments to more patients.
The potential of gene therapy to restore hearing function brings hope to millions of individuals worldwide and offers a promising solution for addressing the significant impact of hereditary deafness.
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