Using Gene Therapy to Restore Hearing

Filed in archive Gene Therapy on June 18, 2007

Researchers may have discovered a way to restore hearing in individuals with a genetic hearing loss resulting form a mutation of a gene known as KCNQ4, which affects the formation of hair-like projections that line the cochlea. They engineered a correct form of the gene and using gene therapy delivery techniques, the KCNQ4 gene has been successfully transferred into human hair cells in the inner ears of patients with hearing loss.

"Our results show that gene therapy reagents are effective in human inner ear tissue. Taken together with the results from another group of scientists who showed that similar gene therapy compounds can produce new hair cells and restore hearing function in guinea pigs suggest that the future of gene therapy in the human inner ear is sound," Holt said.

The study has been published in the online issue of the scientific journal, Gene Therapy.