Gene-Therapy used in treating deafness

Recently, a UK girl born deaf could hear unaided, after a gene-therapy treatment.

She (Opal Sandy) had the therapy in her right ear, under general anaesthetic, and a cochlear implant put into her left ear.
A cochlear implant is an electronic device that improves hearing.
She is the youngest person to have their hearing restored after undergoing new gene therapy.
She had a genetic form of auditory neuropathy caused by the disruption of nerve impulses traveling from the inner ear to the brain.

The gene therapy known as ‘DB-OTO’ is specifically designed for children with OTOF mutations.
In this method, a safe virus is used to inject the patient with the functional gene.
Auditory neuropathy can be caused by a fault in the OTOF gene.
The OTOF gene produces a protein called otoferlin, which is crucial for the inner hair cells in the ear to communicate with hearing nerves.
By inserting a functional copy of the gene into the ear, Regeneron’s “new era” gene therapy fixes the defect.
The therapy uses a modified, harmless virus to deliver a working copy of the OTOF gene into these cells.
Hearing loss caused by a variation in the OTOF gene is not commonly detected until children are two or three years old.

This successful case exemplifies the transformative potential of gene therapy in addressing genetic forms of hearing loss thus offering hope for affected individuals worldwide.
This case also pave the way for further advancements in the related fields of medical genetics and gene therapy.
Doctors in other countries are also exploring similar treatments for the OTOF gene mutation.
Gene therapies are also being used to treat a few other diseases like: – an eye disorder called Leber congenital amaurosis and a muscle disorder called spinal muscular atrophy.