Stanford Initiative to Cure Hearing Loss

Gene Therapy

Recent advances in genetic testing have shown that 70 percent of the most common forms of hearing loss are caused by mutations in only three genes. A simple approach is to use safe and highly sophisticated gene therapy vectors to override the defective gene with an intact healthy one.

One aspect of research in this regard focuses directly on patients with genetic causes of hearing loss. Here, our researchers work on generating disease models from patient-derived stem cells that can be used in the laboratory to develop safe and effective gene therapy treatments. The goal is to translate these findings into first clinical trials within a time frame of ten years.

A second focus of research focuses on genes that play important roles in bonding nerves to cochlear hair cells to form synaptic connections. This research will further the understanding of how the auditory system develops and may lead to new strategies for stimulating the innervation of newly generated hair cells.

Finally, researchers found a gene called ATOH1, which is active in developing hair cells. When the gene was removed from a developing mouse, no hair cells formed in the mouse's cochlea. But if animal cochlea cells were forced to express ATOH1, the cells developed as hair cells even if they would normally have become other cells. The findings imply that if ATOH1 can be activated in cells within an adult human ear, the gene might force those cells to develop as new hair cells. Using the tools of gene therapy may allow scientists to activate ATOH1 and rebuild a damaged ear by replicating the steps that took place during embryonic development.

While preliminary research has been promising, important follow-up experiments still must be done to determine whether expression of ATOH1 is all that is required to make a new hair cell in an adult ear.



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