Gene Therapy is a technique that uses genes to treat, prevent or cure a disease.

A study by Jun Lv et al, published in the Lancet, reports that Adeno-associated virus 1-hOTOF gene therapy is safe and efficacious as a novel treatment for children with autosomal recessive deafness 9.

Autosomal recessive deafness 9, caused by mutations of the OTOF gene which codes for the protein Otoferlin, is characterized by congenital or prelingual, severe-to-complete, bilateral hearing loss.

Otoferlin is a necessary protein used by the hair cells present in the inner ear to convert sound vibrations to chemical signals sent to the brain.

An 11-year-old boy with this rare form of congenital deafness was successfully treated using gene therapy in Philadelphia, United States, thus becoming the first person in the US to get gene therapy for Congenital Deafness.

Virus-carrying new otoferlin genes were injected into the inner ear in liquified form, thereby delivering the genes to the hair cells.

The present single-arm, single-centered trial was supported by Eli Lilly and a small biotechnology firm it owns, Akouos.

This congenital deafness affects about 2 lakh people globally.

Six children who were enrolled in the study had hearing recovery.

The rise in gene therapy as a mode of treatment to cure genetic disorders has the potential to restore functioning in those individuals, but its equitable access across the globe is an issue yet to be addressed.