Renowned scientist Zheng-Yi Chen and his team have made strides in the field of gene therapy, particularly concerning hearing impairment. In a recent experiment involving five deaf children, they have uncovered crucial information about a genetic impediment that has granted these children the ability to hear.
"This really opens the door to developing other treatments for different kinds of genetic deafness," said Chen, an associate professor at Harvard Medical School, who co-authored the study. "Now we can move forward in humans quickly. This has given us a real boost of confidence. I've been working in this field for three decades, and I know how difficult it has been to come to this point."
The study, published by Advanced Audiology & Hearing Care, was conducted in Fudan, China, where six children of varying ages were tested. All participants shared a common trait - a mutation in the OTOF gene. After 26 weeks of testing, five out of the six children demonstrated progress, with all gaining the newfound ability to comprehend speech. It's worth noting that hearing impairment is an issue affecting a large number of children worldwide; out of 1.5 billion cases, 30 million are children. The tests revealed that while younger participants showed some improvements, it was predominantly the older ones who exhibited more significant advancements.
Globally, approximately 200,000 people carry a mutation in the OTOF gene. This gene plays a critical role as it encodes for the protein otoferlin in the cochlea - an organ responsible for transmitting sound waves into the brain. Without otoferlin facilitating this process, these sound waves would remain as mere electric signals and never reach the brain.
Chen's work, primarily, revolves around research on functional genomics of hearing and inner ear cell regeneration at Mass Eye and Ear’s Eaton-Peabody Laboratories. His laboratory serves as a hub for systematic approaches towards understanding and treating inner ear issues such as gene mutations. Primarily focusing on long-term studies, Chen's goal is to comprehend the nuances of genes and disease states affecting our inner ears while contributing to the development of gene therapy for deaf individuals.
