Jan 25, 2024 20:00:00

Succeeded in restoring the hearing of a child who was born deaf with gene therapy

In recent years, research into

gene therapy using genome editing has been progressing as a treatment for diseases and disorders. A research team at Fudan University Hospital in Shanghai has reported that they have successfully restored the hearing in five children with congenital hearing loss through gene therapy. A similar case has also been reported at Children's Hospital of Philadelphia, USA.

AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial - The Lancet
https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(23)02874-X/fulltext

Children's Hospital of Philadelphia Performs First in US Gene Therapy Procedure to Treat Genetic Hearing Loss

https://www.prnewswire.com/news-releases/childrens-hospital-of-philadelphia-performs-first-in-us-gene-therapy-procedure-to-treat-genetic-hearing-loss-302042233.html

Gene therapy breakthrough enables deaf boy to hear for the first time - The Week
https://www.theweek.in/news/health/2024/01/24/gene-therapy-breakthrough-enables-deaf-boy-to-hear-for-the-first.html

Gene therapies restore hearing in several kids with inherited deafness | Live Science
https://www.livescience.com/health/genetics/gene-therapies-restore-hearing-in-several-kids-with-inherited-deafness

Gene therapy is a technology that treats diseases by modifying an individual's genes, such as replacing disease-causing genes with healthy copies or inactivating malfunctioning genes.

Congenital hearing loss is thought to be caused by mutations in a protein called otoferlin . Therefore, a research team at Fudan University Hospital administered an adeno-associated virus in which the protein was replaced with otoferrin to six children aged 1 to 6 who were born deaf.

The research team administered the gene to the membrane that separates the middle ear and inner ear of the children. Dosing was given to one ear, and treatment showed that five out of six people regained their hearing within 26 weeks.

According to the research team, hearing improvements began to appear about 4 to 6 weeks after treatment. At the same time, it has also been reported that children's speaking ability has improved.

Three of the five children who showed the effects of the treatment had

a cochlear implant , which aids their hearing, in the opposite ear where the treatment was performed. However, after treatment, these children were able to perceive spoken words and communicate verbally even with their cochlear implants switched off. In addition, the remaining two children who did not have cochlear implants went from being completely unable to perceive speech due to hearing loss to being able to hear speech for the first time through treatment.

On the other hand, the research team reported, ``Although there were no serious side effects with this treatment, mild side effects were confirmed, such as fever and a temporary increase in white blood cell count.'' 'Further large-scale studies are needed to determine which doses are effective and whether they are safe.'

Similar research is being conducted in the United States, and on October 4, 2023, a research team at Children's Hospital of Philadelphia tested 11-year-old Aisam Dam by partially lifting his eardrum, the membrane that separates the middle ear from the inner ear. We performed gene therapy using injections.

As a result of the treatment, Dam's hearing improved dramatically. Before the treatment, Mr. Dam was diagnosed with ``severe hearing loss,'' but after about four months, the diagnosis has changed to ``mild to moderate hearing loss.''

These successful examples of gene therapy are expected to bring hope not only to patients around the world suffering from hearing loss caused by genetic mutations, but also to the medical community as a whole. 'Gene therapy for hearing loss has been something that doctors and scientists specializing in hearing loss have been working on for more than 20 years,' said John Germiller, an otorhinolaryngologist at Children's Hospital of Philadelphia. gene therapy has become a reality.'

For safety reasons, the U.S. Food and Drug Administration (FDA) insists that the efficacy and safety of treatments should first be evaluated in older patients before conducting treatment or research in younger patients. doing.