Professor Manohar Bance, an ear surgeon at Cambridge University Hospitals NHS Foundation Trust who is leading the trial in the UK, said a decision by the NHS last year to start funding genetic testing for hearing loss had been crucial in “changing the whole landscape” for the development of gene therapies.
Gene therapies now held remarkable promise to restore hearing, he suggested. “It’s the dawn of a new era,” he added.
Auditory neuropathy can be due to a variation in a single gene—known as the OTOF gene—which produces a protein called otoferlin. This protein typically allows the inner hair cells in the ear to communicate with the hearing nerve.
Mutations in the OTOF gene can be identified by genetic testing. However, Bance said it was a condition often missed when newborn babies were screened for potential hearing problems. “This is one of the few conditions where everything works except the transmission between the hair cells and the nerve. So everything else looks fine when you test it, but they can’t hear anything. So these poor kids’ [difficulties] end up being missed,” Bance added.
The new gene therapy aims to deliver a working copy of the faulty OTOF gene using a modified, non-pathogenic virus. It will be delivered via an injection into the cochlea under general anesthetic.
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