CHILDREN in Spain who have been deaf from birth are hearing for the first time after taking part in gene therapy trials.
Four of them suffered from a defect in the otoferlin gene but have had very positive results- just a month after starting treatment
Three of Spain’s top ear experts are coordinating gene therapy trials with youngsters from Madrid, Pamplona, and Gran Canaria taking part.
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Two of the children are aged just one year, while the others are four and 16-years-old.
One of them is named Daniela, and her father said: “We are seeing a super positive response from her in recovering a good part of her hearing and we hope the progress continues so that she will be able to develop her speech.”
A genetic mutation prevents otoferlin from being produced naturally or making abnormal versions of it.
Otoferlin is essential for the proper functioning of the inner ear cells and without it, hearing is virtually impossible.
Angel Ramos from the Las Palmas Children’s Hospital said: “What gene therapy does is to repair the genetic damage caused by a lack of otoferlin.”
“Through surgery, a virus with modified genetic material is introduced enabling a child to hear for the first time.”
Severe hearing loss was previously treated with cochlear implants or hearing aids but the trials now open up the chance of a totally different and far more successful approach.
Medics emphasised that the trials are still in the early stage and deals with a specific kind of deafness, but doors are now being opened for further research to help more children.
Trials are also taking place in China, with 20 children involved.