09 Feb Gene Therapy Restores Hearing Down To A Whisper, in Mice
MedicalResearch.com Interview with:
Gwenaelle Geleoc, PhD
Department of Otolaryngology
F.M. Kirby Neurobiology Center
Children’s Hospital and Harvard Medical School
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: We seek to develop gene therapy to restore auditory and balance function in a mouse model of Usher syndrome. Usher syndrome is a rare genetic disorder which causes deafness, progressive blindness and in some cases balance deficits. We used a novel viral vector developed by Luk Vandenberghe and package gene sequences encoding for Ush1c and applied it to young mice suffering from Usher syndrome. These mice mimic a mutation found in patients of Acadian descent. We assessed recovery of hearing and balance function in young adult mice which had received the treatment. Otherwise deaf and dizzy, we found that the treated mice had recovered hearing down to soft sounds equivalent to a whisper and normal balance function.
MedicalResearch.com: What should readers take away from your report?
Response: This work demonstrates that gene therapy treatments can efficiently restore auditory and balance function. The level of recovery that we have obtained has never been seen before. Having identified a potent vehicle and applying the treatment at the right time was crucial in our study.
MedicalResearch.com: What recommendations do you have for future research as a result of this study?
Response: We need to extend this work to other deafness genes that lead to congenital or progressive deafness. The difficulty will arise when looking at genes that extend beyond the capacity of the vector we used for this study. Any gene over 5kb will not fit in our vector. Other strategies will therefore be required.
MedicalResearch.com: Is there anything else you would like to add?
Response: Our goal is to advance research to develop new treatments for deafness and balance disorders. I welcome collaborations and material sharing with anyone who wish to work with us for this purpose.
MedicalResearch.com: Thank you for your contribution to the MedicalResearch.com community.
Gwenaëlle S Géléoc et al. Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c. Nature Biotechnology, February 2017
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