Medical News
Gene therapy can reverse hearing loss
The technique promises to cure people with the inability to hear since birth
One UK baby and a child from Spain who were born deaf had their hearing restored after undergoing a new gene therapy treatment designed to treat their genetic deafness , according to two separate studies . In the most recent trial , Opal Sandy , a UK girl , received the treatment before turning one and after six months managed to hear soft whispers and say words like “ Mama ” and “ Dada ,” the BBC reports .
Her deafness was caused by a faulty gene called otoferlin . The revolutionary approach involves the infusion of a functional copy of the otoferlin gene — along with a harmless virus to deliver it — into the ears . This leads to the repair of damaged ear cells , allowing patients to hear .
“ It ’ s wonderful seeing her respond to sound ,” chief investigator and ear surgeon Professor Manohar Bance told BBC News .
One of the standard treatments for this type of hearing loss is a cochlear implant , an electronic device placed inside the ear that improves hearing by sending sound signals past the damaged part of the ear and directly to the hearing nerve . However , this approach often requires additional interventions .
“ What I am hoping is that we can start to use gene therapy in young children ... where we actually restore the hearing and they don ’ t have to have cochlear implants and other technologies that have to be replaced ,” he said .
Data about Opal ’ s treatment and other participants in the trial were presented at the annual meeting of the American Society of Gene and Cell Therapy , which was held in Baltimore in May .
In a previous study , Aissam Dam , an 11-year-old boy from Spain who never heard any sound , was able to hear for the first time after being treated at the Children ’ s Hospital of Philadelphia ( CHOP ) with a similar gene therapy approach to replace the abnormal gene with a working one . Aissam ’ s father said his son was able to hear traffic noise a few days after being treated , while his hearing in the treated ear was almost normal two months after therapy , according to The New York Times .
Dr Dylan K . Chan , a paediatric otolaryngologist at the University of California , San Francisco , who was not involved in the research , told The New York Times this was a “ groundbreaking ” study .
“ There has never been a biological or medical or surgical way to correct the underlying biological changes that cause the inner ear to not function ,” he said .
The treatment of hearing loss lends itself to gene therapy because most gene mutations causing the dysfunction don ’ t affect other parts of the body , meaning that fixing the faulty gene will have no effects on other organs or tissues , Manny Simons , chief executive and co-founder of Akouos and senior vice president of gene therapy at Lilly , the companies supporting the trial , told The New York Times .
However , despite the therapy being successful , researchers believe Aissam may never learn to understand or speak a language . While the brain has the ability to learn to speak between the ages of two and five , explained �r �ohn Germiller , an otolaryngologist who ’ s leading the study at CHOP , after age five , the window for learning to speak closes . But the ability to hear could still help Aissam understand when people are trying to communicate or improve lip reading , he added .
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