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A revolutionary gene therapy technique has successfully restored hearing in multiple children who were born with inherited deafness in China, representing a significant achievement in the field of hearing loss treatment.
During the 26-week clinical trial, six children with a form of hereditary deafness caused by a genetic mutation in the OTOF gene were studied.
According to the study published on Wednesday in The Lancet medical journal, five out of six children who received the experimental treatment showed improvement in hearing and speech recognition without any reported harmful effects.
This study is the first of its kind to test gene therapy as a treatment for this genetic disorder in humans. It included the largest number of patients and the longest period of follow-up to date.
“According to study co-author Zheng-Yi Chen, DPhil, there has not been an effective treatment for deafness since the invention of cochlear implants 60 years ago. This marks a significant milestone and signifies a new chapter in the battle against all forms of hearing loss.”
A report from the House Committee in the United States claims that China intentionally postponed the disclosure of vital gene sequencing information related to COVID-19.
More than 1.5 billion people across the globe have hearing impairments, with approximately 26 million of them being born with the condition.
According to scientists, over 60% of hearing loss cases in children are caused by genetic factors.
There is a hereditary hearing loss known as DFNB9, which is the result of mutations in the OTOF gene. These mutations prevent the production of a functional protein essential for transmitting sound signals from the ear to the brain.
According to scientists, at present, there are no medications available to address this type of inherited hearing loss which can potentially lead to abnormal brain development in children if left untreated.
Dr. Chen reported that the findings of this research are incredibly impressive. Throughout the weeks, there was a significant improvement in the hearing and speech abilities of children.
During the research, a group of six children with complete hearing loss for more than 26 weeks were observed at the Eye & ENT Hospital of Fudan University. They were given an injection of adeno-associated virus (AAV) containing a modified form of the human OTOF gene.
The process was created to precisely insert the altered virus containing the OTOF gene into the inner ears of the patients using a specialized surgical technique.
Various amounts of the one-time injection were administered.
The study revealed significant advancements in understanding speech and regained capability for typical conversation among the kids.
Scientists have reported that their findings provide support for the safety and efficacy of gene therapies as a possible remedy for genetic hearing loss.
“We are the pioneers in conducting the clinical trial for OTOF gene therapy. It is exciting that our team was able to apply our findings from studying DFNB9 in animal models to successfully restore hearing in children with DFNB9,” stated Yilai Shu, the lead author of the study.
Dr. Shu expressed enthusiasm for our future endeavors in addressing various types of genetic hearing loss and providing treatments to a larger number of patients.
Source: independent.co.uk