An innovative gene therapy allows children with hearing problems to hear
Five children who were born deaf can now hear in both ears thanks to a gene therapy trial that raises hope for a future treatment. The study participants, unable to hear due to an inherited genetic mutation that prevents the production of a protein vital for the transmission of auditory signals from the ear to the brain, Significant improvement is seen after treatment,
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Doctors at Fudan University in Shanghai treated both ears of children aged one to 11, hoping they would regain enough three-dimensional hearing to participate in conversations and determine the direction of sounds.
Within a few weeks of receiving the therapy, the children’s hearing improved, and they became better able to locate sound sources and recognize speech in noisy environments. Two kids were recorded dancing to the musicThe researchers reported Naturopathy,
Zheng-Yi ChenA scientist at Massachusetts Eye and Ear, a Harvard teaching hospital in Boston, who co-led the trial, called the results “amazing,” and said researchers saw dramatic progress in the children’s hearing abilities.
How the therapy works
The children in the trial suffer from a condition called DFNB9, caused by mutations in the Otof gene, which accounts for 2-8% of all cases of congenital hearing loss. In January, the same US-Chinese team reported improvements after treatment in children who were deaf in only one ear, But the intention was always to provide hearing ability in both ears,
The therapy uses an inactivated virus to insert functional copies of the affected gene, Otof, into the inner ear. Once inside, the ear’s cells use it as a template to make functional copies of the new genetic material Otoferlin Protein,
Video footage of the patients shows a two-year-old child responding to his name three weeks after treatment and dancing to music after 13 weeks, but he had shown no response before receiving the injections. Another patient, a three-year-old girl, did not react to sounds before treatment, but after 13 weeks she could understand sentences and speak some words.
The oldest patient, an 11-year-old girl, showed no response to tones of different frequencies before therapy, but she responded to all of them six weeks later and was able to participate in speech training from the 13th week onwards.
Future investigations
According to the World Health Organization, more than 5% of the world’s population, or 430 million people34 million suffer from disabling hearing loss, including children. Of the 26 million people with congenital hearing loss, 60% are caused by genetic factors.
Deficiency or inactivation of the otoferlin protein results from pathogenic mutations in the OTOF gene and leads to autosomal recessive deafness 9 (DFNB9). DFNB9 is characterized by severe to complete bilateral congenital or prelingual hearing loss and accounts for 2-8% of inherited deafness.
Larger trials will be needed to further evaluate the therapy’s benefits and risks. The gene therapy is injected during a minimally invasive surgical procedure, so treating both ears doubles the time patients spend in surgery. Regardless, we’re on the right track.