Usher syndrome is a devastating genetic disorder that causes profound hearing loss, balance disorders and progressive blindness. In the ear, mutations in the Usher 1c (USH1C) gene lead to damage and loss of the inner ear’s sensory cells, causing deafness.

Scientists from Harvard Medical School recently reported an unprecedented recovery of hearing in a USH1C mutant mouse model of deafness using gene therapy. Deaf mice recovered hearing to a level of about 25 decibels, the equivalent of a whisper or rustling leaves.

The findings suggest a possible therapeutic strategy for treating patients with USH1C and other genetic disorders of inner ear function.