🧏 She was deaf at birth, 18-month-old Opal Sandy had her hearing restored after a world-unique gene therapy.

• 18-month-old Opal Sandy has had her hearing almost fully restored through a first-of-its-kind gene therapy.
• The treatment involved a 16-minute operation where she received a functional copy of a gene that is crucial for hearing.
• The study will continue with more children to test different doses of the gene therapy.

A major breakthrough in gene therapy

Opal Sandy, who suffers from auditory neuropathy, a condition that disrupts nerve impulses from the inner ear to the brain, underwent a successful gene therapy trial, reports The Guardian.

This condition is caused by a defect in the OTOF gene, which is necessary for the cells in the ear to communicate with the auditory nerve. To correct this, the new treatment from the biotech company Regeneron uses a functional copy of the gene delivered directly to the ear. A harmless virus is used to transport the functional gene to the patient.

Opal Sandy can now hear sounds as soft as a whisper.

The study is divided into three parts. In the first part, three children receive a low dose of the gene therapy in one ear. Another group of three children will receive a higher dose on one side. If this proves to be safe, additional children will receive doses in both ears simultaneously. A total of 18 children globally will be recruited for the study.

Potential cure for this type of deafness

Professor Manohar Bance, an ear surgeon and lead investigator for the study, commented that the results from Opal Sandy are "almost like normal hearing restoration" and expressed hope that this could be a potential cure for this type of deafness.

With Opal's hearing restored, her parents now face a new challenge: managing. Her new favorite activity is banging utensils on the table to make as much noise as possible.

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