Gene therapy to restore the architecture of the inner ear



Deafness is the most common inherited sensory impairment, with the majority of cases associated with no other signs or symptoms. For many people, deafness is caused by defects in the actin-rich stereocilia bundles on the surfaces of sensory hair cells—defects which could not be corrected. While hearing aids and cochlear implants are possible treatment options, they do not work for all patients and do not offer a biological cure.


IRP researchers led by Lisa Cunningham, Ph.D., and Wade Chien, M.D., investigated the potential for gene therapy to restore the morphology of defective stereocilia bundles. They discovered that when sensory hair cells received gene therapy delivered through a virus injected into the inner ears of mice, defective bundles were restored to normal length and morphology.


In finding a viable option for reviving normal stereocilia architecture, the team’s research has demonstrated the feasibility of gene therapy for restoring structural defects in the inner ear. It also suggests that gene therapy holds promise for correcting hereditary deafness caused by structural flaws, should it be shown to be safe and effective in human patients.


Chien WW, Isgrig K, Roy S, Belyantseva IA, Drummond MC, May LA, Fitzgerald TS, Friedman TB, Cunningham LL. (2016). Gene Therapy Restores Hair Cell Stereocilia Morphology in Inner Ears of Deaf Whirler Mice. Mol Ther. 24(1): 17-25.

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