Promising gene therapy to restore hearing to deaf children

Promising gene therapy to restore hearing to deaf children

An advanced clinical trial using gene therapy has restored hearing to five children born deaf. After six months, the children were able to recognize speech and conduct conversations, raising hopes for its wider use in the near future, according to what was published by the New Atlas website, citing the journal Science Advances.

Hereditary condition

Patients in the trial suffered from a genetic condition called autosomal recessive deafness 9 (DFNB9), which results from a mutation in a gene called OTOF, which produces the protein otoferlin, which helps transmit electrical impulses from the cochlea to the brain, where it can be interpreted as sound – but without it. Those signals will never get there. Because it is caused by a single mutation and does not involve any physical damage to the cells, the team says DFNB9 is an ideal candidate for this type of gene therapy.

In the study conducted by researchers from Harvard Medical School, Massachusetts Eye and Ear, and Fudan in China, gene therapy involves packaging the OTOF gene into viral carriers and injecting the mixture into the inner ear fluid. The viruses then searched for cells in the cochlea and inserted the gene into them, allowing them to start making the missing autoferlin protein and restore hearing.

Cochlear implant

Six children, aged between one and seven years, whose DFNB9 had left them completely deaf also participated in the study. Four patients were fitted with cochlear implants, which bypassed the problem and could allow them to learn to recognize speech and other sounds. In this case, the transplants were stopped.

remarqable improvement

After gene therapy, the children were followed for 26 weeks. At that time, five out of six showed significant improvement, with the three older children able to understand and respond to speech, while two were able to pick it up in a noisy room and carry on a conversation on the phone. Some of the children were too young to undergo the usual tests, but they were found to respond to sounds, and even began to say simple words such as “mama.” The improvements were gradual, but the team reported that the children started showing results before the first test four weeks later.

Genetic causes and aging

Yilai Xu, the study's lead researcher, said that participants in this trial will continue to be monitored, while follow-up studies will be conducted on other people. The team says that approval of the treatment in the United States may take between three to five years. Similar gene therapies have been tested for genetic or age-related hearing loss.

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