Gene therapy has shown promise for treating sensorineural hearing loss, supported by numerous successful preclinical studies. From the perspective of translation to humans, researchers have focused more on the genetic...Gene therapy has shown promise for treating sensorineural hearing loss, supported by numerous successful preclinical studies. From the perspective of translation to humans, researchers have focused more on the genetic causes of profound sensorineural hearing loss, where the sensory epithelium remains viable and intact for a considerable time after birth in humans. A key human deafness gene that best fits such a context is OTOF (GenBank AF183185.1), of which protein products, otoferlin, is essential for synaptic exocytosis and vesicle replenishment at the inner hair cell level in the cochlea.展开更多
基金supported by the Basic Science Research Program through the National Research Foundation of Korea,funded by the Ministry of Education,South Korea(No.2021R1A2C2092038 to B.Y.C.,2022R1I1A3072036 to E.Y.)Bio Core Facility center program through the NRF(No.2022M3A9G1014007 to B.Y.C.)+2 种基金also by the Basic Research Laboratory program through the NRF,funded by the Ministry of Science&ICT(MSIT)(No.RS-2023-0021971031482092640001 to B.Y.C.)the Technology Innovation Program(No.K_G012002572001 to B.Y.C.)funded by the Korean Ministry of TradeIndustry and Energy.This study is also funded by the Seoul National University Bundang Hospital intramural research fund(No.16-2020-0009,16-2022-0005,13-2023-0002,16-2023-0002,18-2023-0004,and 13-2024-0004 to B.Y.C.).
文摘Gene therapy has shown promise for treating sensorineural hearing loss, supported by numerous successful preclinical studies. From the perspective of translation to humans, researchers have focused more on the genetic causes of profound sensorineural hearing loss, where the sensory epithelium remains viable and intact for a considerable time after birth in humans. A key human deafness gene that best fits such a context is OTOF (GenBank AF183185.1), of which protein products, otoferlin, is essential for synaptic exocytosis and vesicle replenishment at the inner hair cell level in the cochlea.
