Gene Therapy: Children Born Deaf Regain Hearing

Source: FSSPX News

There are wonderful advances in gene therapy. It is a “clean” therapy that has no need of the manipulation of embryos or embryonic stem cells, which are often obtained at the cost of the lives of unborn children.

This therapy allowed a little girl of 18 months, Opal Sandy, to gain the sense of hearing. The child suffers from auditory neuropathy due to mutation of the OTOF gene. The defect impairs the production of otoferlin, a protein which allows the ciliated cells of the inner ear to convert sound vibrations into chemical signals. The absence of this protein causes total deafness.

Researchers from the University of Cambridge practiced the therapy on the child when she was 11 months old. A viral vector carrying a functional copy of the OTOF gene was injected into her right ear. Four weeks after the operation, the little girl could “respond to sounds,” “even while the cochlear implant in her left ear was turned off.”

As Genethique reports, “Then, 24 weeks after her operation, Opal attained an ‘almost normal’ level of hearing in her right ear. She can ‘respond to weak sounds like murmurs and is now able to speak.’”

Previous Attempts

In October 2023, Gènéthique continues, “scientists of Fudan University in Shanghai indicated having restored hearing for four children, also thanks to this gene therapy.” “Following the therapy, the hearing of all four children improved. ‘They gained perhaps 60 to 65% of normal hearing,’ Dr. Yilai Shu indicated.

“’Before the treatment, if you put them in a movie theater with the loudest sound, they would not have heard it,’ Dr. Zheng-Yi Chen, specialist professor at Mass Eye and Ear, a Harvard hospital which helped devise the study, clarified. ‘Now, they can hear a conversation nearly normal,’ adds the scientist.”

On January 23, 2024, the children’s hospital of Philadelphia announced that one of its patients, Aissam Dam, 11 years old and “born profoundly deaf” due to a defect in the OTOF gene, heard thanks to a gene therapy.

However, Opal is the youngest to have received this therapy. Moreover, she was the only one to have learned to speak thanks to it. Opal should soon enter phase two of the trial. She will be injected with a stronger dose in the right ear. During phase three, both of her ears will be treated.

A Rare Form of Deafness

Genethique reports, “The therapy only concerns one specific form of deafness. It only applies in fact to people presenting an anomaly of the gene coding otoferline. These anomalies are the origin of around 1 to 3% of cases of congenital deafness. [...]

“This type of deafness was especially targeted, because the ciliated auditory cells respond relatively well to gene therapy and easily absord new DNA. Moreover, these cells do not grow, and are not replaced throughout life. When the ‘replacement’ gene is added to the cells, it can therefore remain active throughout all one’s life.”

The hearing restored by gene therapy gives a better result than cochlear implants--an electronic device which uses a receiver and electrodes to directly stimulate the auditory nerve. It has allowed some children to learn how to speak, but as soon as it is turned off, the child can no longer hear anything.