Researchers Use Gene Therapy to Repair Inner-Ear Defects in Mice Scientists may have found a way to correct nonfunctioning stereocilia in the inner ears of newborn mice. In research from the National Institute on Deafness and Other Communication Disorders, otolaryngology surgeon and scientist Wade Chien and his team used gene therapy to fix the hair-like structures in mice who had ... Research in Brief
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Research in Brief  |   February 01, 2016
Researchers Use Gene Therapy to Repair Inner-Ear Defects in Mice
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Balance & Balance Disorders / Special Populations / Genetic & Congenital Disorders / Research Issues, Methods & Evidence-Based Practice / Research in Brief
Research in Brief   |   February 01, 2016
Researchers Use Gene Therapy to Repair Inner-Ear Defects in Mice
The ASHA Leader, February 2016, Vol. 21, online only. doi:10.1044/leader.RIB3.21022016.np
The ASHA Leader, February 2016, Vol. 21, online only. doi:10.1044/leader.RIB3.21022016.np
Scientists may have found a way to correct nonfunctioning stereocilia in the inner ears of newborn mice.
In research from the National Institute on Deafness and Other Communication Disorders, otolaryngology surgeon and scientist Wade Chien and his team used gene therapy to fix the hair-like structures in mice who had inherited deafness at birth, a type of deafness also found in humans. The study appears in the journal Molecular Therapy.
The newborn mice in the study had irregular stereocilia—unusually short and fat, and arranged in extra rows—because of a mutated gene of a hair cell protein called whirlin. The mutated hair cells die off quickly after birth, which often causes deafness or Usher syndrome in humans.

The mutated hair cells die off quickly after birth, which often causes deafness or Usher syndrome.

The researchers were able to restore the stereocilia to normal heights and amounts in the newborn mice with the whirlin mutation—though not in adult mice with the same mutation—by injecting a virus carrying the whirlin’s corrected gene. However, the newborn mice did not regain their hearing ability after treatment.
There is no available fix for defective hair cells and stereocilia in humans, but the study lends promise that scientists may be able to intervene in newborn humans who have inherited gene mutations. The scientists say they are now working on determining why correcting the stereocilia did not restore the mice’s hearing.
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February 2016
Volume 21, Issue 2