A team led by Mass Eye and Ear researchers has demonstrated for the first time a successful restoration of hearing through a novel, in vivo genome editing approach in an adult mouse model with a form of inherited deafness caused by mutations in microRNA. The researchers note that mouse and human microRNAs have identical sequences, and accordingly, hope their new study lays the foundation for translational research into applications in humans with deafness caused by these types of mutations. The study, led by Zheng-Yi Chen, DPhil, an associate scientist in the Eaton-Peabody Laboratories at Mass Eye and Ear (a member of the Mass General Brigham healthcare system), was published July 10 in Science Translational Medicine .
"Our findings provide a promising pathway for developing treatments by editing for many forms of genetic hearing loss," said Chen, who is the Ines and Fredrick Yeatts Chair in Otolaryngology at Mass Eye and Ear and an associate professor in Otolaryngology–Head and Neck Surgery at Harvard Medical School. With further study, our intervention using genome editing could potentially halt or reverse hearing loss progression in affected individuals, including adults." Zheng-Yi Chen, Associate Scientist, Eaton-Peabody Laboratories, Mass Eye and Ear About one in 500 newborns suffer from genetic hearing loss and currently there are not any approved therapeutics to treat deafness.
In the new study, researchers targeted a specific mutation in the microRNA-96 (MiR-96) gen.
