“The FDA has approved a gene therapy for a form of congenital deafness caused by a faulty OTOF gene. The treatment uses a virus to deliver a working copy of the gene, a technique that has shown very promising results in recent trials,” reports The Doomslayer.
Live Science explains:
The U.S. Food and Drug Administration (FDA) has approved the first-ever gene therapy for inherited deafness.
The therapy, called Otarmeni, is approved to treat a form of hearing loss caused by mutations in the OTOF gene, which codes for a protein called otoferlin. Cells in the inner ear need otoferlin to translate vibrations into signals that can be interpreted by the brain. When people carry two defective copies of the OTOF gene — one from each parent — this line of communication between the inner ear and brain is cut, resulting in severe-to-profound hearing loss.
Otarmeni is a one-time treatment that uses harmless viruses to deliver working copies of OTOF into the ear. In a trial including 20 participants, 16 showed improved hearing within six months, and one additional person showed improvement within a year of treatment.
The Harvard Gazette adds:
An experimental gene therapy for people with an inherited form of deafness led to durable hearing improvements, a new study shows, with associated gains in patients’ ability to recognize speech.
The research corrected mutations in the OTOF gene, one of about 200 genes whose mutations are known to cause deafness from birth. Patients 18 and younger saw the strongest gains in hearing and ability to recognize speech. Adults receiving the therapy also saw improvements, though the effect was smaller. Overall, 90 percent of recipients saw their hearing improve, with half reaching normal levels by the study’s end at 2½ years.
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