Groundbreaking Gene Therapy Restores Hearing in Deaf Toddler

Toddler's ear

In a remarkable breakthrough, 18-month-old Opal Sandy from Britain has become the first and youngest person worldwide to regain hearing through innovative gene therapy.

Interesting Engineering reports that Opal, who was born completely deaf due to a rare genetic condition called auditory neuropathy, received the groundbreaking treatment at Addenbrooke’s in Cambridge. The therapy, known as DB-OTO, targets cases with OTOF mutations, which affect the production of the protein otoferlin, crucial for communication between the inner ear and the auditory nerve.

The CHORD trial, which began in May 2022, aims to evaluate the safety, tolerability, and efficacy of DB-OTO gene therapy. Opal underwent the procedure under general anesthesia, where a harmless AAV1 virus containing the genetic material was injected into her cochlea. This virus delivers a copy of the gene to the cells, helping restore hearing functions before being naturally eliminated from the body.

Opal’s left ear received the gene therapy, while a cochlear implant was fitted in her right ear. Remarkably, about four weeks after the treatment, Opal started responding to sounds, even with the cochlear implant switched off. Further monitoring revealed significant improvements, with Opal being able to hear whispers at 24 weeks post-treatment.

Manohar Bance, a leading researcher, stated, “Opal’s experience marks a new era in the treatment for deafness and supports the development of gene therapies that may prove to make a difference in other hearing conditions.” The trial, consisting of three phases, involves administering increasing doses of the gene therapy to ensure safety and efficacy.

Opal’s parents, Jo and James Sandy, are thrilled with the success of the therapy and proud that their daughter can now hear even quieter sounds. They hope that this breakthrough will help other children and families, as it significantly improves the quality of life, especially in situations where cochlear implants cannot be worn, such as while bathing.

Read more at Interesting Engineering here.

Lucas Nolan is a reporter for Breitbart News covering issues of free speech and online censorship.


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