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Researchers in China report that a gene therapy trial has successfully allowed deaf children to gain the ability to hear.
According to the journal Science, the inner ears of deaf children were implanted with a harmless virus containing a novel genetic code.
According to a team of researchers from Fudan University in China, their experimental therapy has resulted in 80% of children being able to hear.
The trial participants were children who were born with hearing impairments.
According to The MIT Technology Review, they received two faulty versions of a gene that aids in producing a protein responsible for allowing hair cells in the inner ear to send sound signals to the brain.
The report includes a video clip of a six-year-old participant in the trial conversing with her mother after taking out her cochlear implant.
The specific protein, Otoferlin (Otof), is associated with deafness and is a rare condition, accounting for only 1-3% of inherited deafness cases.
It is estimated that there are approximately 20,000 individuals in the US and EU, specifically in the UK, Germany, France, Spain, and Italy, who may have auditory neuropathy caused by abnormal forms of the Otof protein.
Healthcare professionals have observed that children who have hearing impairments may encounter obstacles in developing their communication abilities and could potentially fall behind in reaching developmental milestones without appropriate assistance from the beginning.
According to the researchers, the latest trial findings provide additional optimism for potential treatment options for individuals who have had hearing loss since birth.
Currently, there are several trials in progress that aim to use gene therapy as a treatment for inherited deafness.
Cambridge’s Addenbrooke’s Hospital is currently involved in a groundbreaking study to determine if gene therapy can restore hearing in children who have inherited severe to profound hearing loss.
“According to ear surgeon Manohar Bance from Cambridge University Hospitals, children who have a mutation in the Otof gene are typically born with a significant level of hearing loss. However, they may pass the standard hearing test for newborns, leading others to believe they have normal hearing.”
According to Dr. Bance, the hair cells are functioning, but they are not communicating with the nerve.
“Treating hearing loss in young children with otoferlin deficiency through gene therapy is a promising initial step, as it is a relatively straightforward approach compared to other methods. It is crucial for all other aspects to be functioning properly.”
If gene therapy treatments for Otof gene-related hearing loss are proven to be highly effective, they could provide a potential solution for hearing loss caused by other prevalent genetic disorders.
Dr. Bance emphasized the significance of ensuring the success of the initial gene therapy treatment, as it will pave the way for treating other genetic disorders.