Gene Therapy to reverse Hearing Loss

According to a study published in the journal Science Advances, researchers at Boston Children’s Hospital discovered that restoring a defective protein in the inner ear cured severe hearing loss in mice. In the inner ear, a mutant protein called stereocilin (STRC) was replaced in a first-of-its-kind Gene therapy procedure. According to a news release, the researchers claimed that it corrected severe hearing loss in mice and, in some cases, returned hearing to normal levels.

Hearing loss has been linked to mutations in at least 100 distinct Gene, according to the researchers in their press release. According to the researchers, the Gene STRC, which is also the second most frequent Gene cause of hearing loss, may be responsible for up to 16 percent of hereditary hearing loss. “Patients with STRC mutations have no cochlear amplification and, as a result, have decreased auditory sensitivity, trouble with frequency discrimination, and speech perception,” the investigators said in a peer-reviewed journal article.

The sensory hair cells in the inner ear must make touch with the tectorial membrane in order to perceive sound, according to the researchers. The membrane vibrates in response to sound, turning the vibrations into impulses that are sent to the brain. The stereocilin protein, according to the researchers, functions as a scaffold, allowing the hair cells to stand up in an ordered bundle, allowing the hair cell tips to contact the tectorial membrane.

According to the paper, the researchers used a mouse model to develop a dual-vector protein-recombination approach to replace full-length wild-type Strc in the outer hair cells of mice with STRC mutations. According to the article, the researchers detected “strong restoration of full–length stereocilin protein” in the mice, as well as hair bundles that seemed normal and were able to contact the tectorial membrane.