Damage and loss of hair cells in the inner ear is the most frequent cause of hearing loss (HL), since mammalian hair cells are not replenished once lost. To date, the treatment of HL consists of hearing aids or cochlear implants, but both options restore hearing with limited success. Gene therapy is an attractive option as a means for treating sensorineural hearing impairment, although at present it is investigational. The finding that hair cells can regenerate in nonmammalian vertebrates has triggered many studies, with the hope of simulating a similar process in humans. Current research that focuses on the auditory pathways, the genes involved in HL, efficient ways to transfer therapeutic agents into the inner ear, and optimization of gene manipulation, gene therapy, and stem cell treatment raises hope for restoring hearing and curing deafness.Learning Outcomes: As a result of this activity, the participant will be able to (1) list the different approaches being studied for the treatment of sensorineural hearing loss, and (2) list the methods used to transfer therapeutic genes into the inner ear.Hearing impairment affects approximately one in 1000 newborns and 4% of people under the age of 45. 1 Progressive hearing loss (HL) affects a greater proportion of the population; in the United States 10% of the population suffers from HL by age 65 and 50% by age 80. 2 Similar proportions exist in other parts of the world, except where high rates of consanguinity are endemic, such as the Middle East, India, and Pakistan. 3 In these regions, large kindreds have been identified with a higher rate of deafness, particularly in children.Various genetic and environmental causes are involved in the etiology of sensorineural