A groundbreaking study has shown that a new form of gene therapy can successfully restore hearing in teenagers and adults born with congenital deafness. Researchers used a modified virus to deliver a working copy of the OTOF gene, which produces a protein critical for hearing, directly into the inner ear’s hearing cells.
The study focused on toddlers and young adults with OTOF-related deafness, a condition caused by mutations in the OTOF gene that produces the otoferlin protein. The researchers used a modified virus as a delivery system to carry the working copy of the OTOF gene, which was injected into the inner ears of ten participants aged 1 to 24 years.
The results showed rapid and significant hearing improvements, especially in younger participants. Within the first month of treatment, the average total hearing improvement reached 62% on objective brainstem response tests and 78% on behavioral hearing assessments. Two participants achieved near-normal speech perception.
While the study confirmed the favourable safety profile of this virus-based gene therapy, it also revealed important insights into the ideal window for treatment, with children between the ages of 5 and 8 showing the most pronounced benefit. However, younger children and older participants showed improvement but not as dramatically as expected.
The findings mark a significant milestone in the treatment of genetic deafness, bridging the gap between animal studies and human trials. With further research and advancements in gene therapy, the possibility of curing genetic hearing loss becomes increasingly realistic.
Source: https://www.livescience.com/health/genetics/in-a-first-congenital-deafness-in-teens-and-adults-treated-with-new-gene-therapy