A breakthrough clinical trial using gene therapy has restored hearing to five children born deaf. After six months, the children were able to recognize speech and hold conversations, raising hopes for wider use in the near future.
The patients in the trial suffered from a genetic condition called autosomal recessive deafness 9 (DFNB9), which is caused by a mutation in a gene called OTOF. This gene produces the otoferlin protein, which helps transmit electrical pulses from the cochlea to the brain, where it can be interpreted as sound – but without it, those signals never get there. Because it’s caused by a single mutation, and doesn’t involve any physical damage to cells, the team says DFNB9 was the perfect candidate for this kind of gene therapy.
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The gene therapy involves packaging the OTOF gene into viral carriers, and injecting the mix into the inner ear fluid. The viruses then sought out cells in the cochlea and inserted the gene into them, which allows them to start manufacturing the missing otoferlin protein and restore hearing.
Continue here: New Atlas
