USH Talks: Gene Therapy for Usher Syndrome Type 1C
February 6, 2017
Gwenaëlle Géléoc, PhD
In this USH Talk, Dr. Gwen Géléoc shares exciting news on progress made towards gene therapy for Usher syndrome type 1c. Working with a mouse model of a human mutation, Dr. Géléoc and colleagues delivered a normal copy of the Ush1c gene to the inner ear soon after the mice were born. This led to robust improvements enabling profoundly deaf and dizzy mice to hear sounds at the level of whispers and recover proper balance function. Dr. Géléoc is cautiously optimistic that successes in the lab will someday lead to novel therapeutic approaches in the clinic.
Press: Gene therapy restores hearing in deaf mice...down to a whisper (published online February 6, 2017)
Gwenaëlle Géléoc received a PhD, in 1996, from the University of Montpellier II, France. She completed two postdoctoral fellowships, one in the Department of Physiology at University College London and subsequently in the Department of Neurobiology at Harvard Medical School. In 2001, she joined the faculty at the University of Virginia and in 2011, returned to Boston and joined the Department of Otolaryngology at Boston Children’s Hospital and Harvard Medical School.
Dr. Géléoc's research has been largely focused on the physiology of the sensory cells (so called hair cells) of the auditory and balance organs of the inner ear as well as the role played by numerous hair cell genes, many implicated in severe to profound hearing loss and balance disorders and some also expressed in the eye that are implicated in retinal degeneration and blindness leading to Usher syndrome. More recently, a focus of her research has been on the restoration of hearing using gene therapy to target genes responsible for Usher syndrome.