Gene therapies have restored hearing in several children with inherited Deafness

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Clinical trial results show that two new gene therapies have helped several children with a rare inherited form deafness to hear.

Both therapies target the gene. otoferlinA protein in the inner-ear that allows nerve cells to translate sound vibrations into electrical signals that the brain can interpret. Mutations of the otoferlin genes cause About 1% – 8% of casesCongenital deafness is when a child is born without hearing. Even so, mutations of the gene are rare and affect only a small percentage of people. An estimated 200,000 people worldwide.

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