Document Type

Article

Peer Reviewed

1

Publication Date

6-2-2016

NLM Title Abbreviation

Am J Hum Genet

Journal/Book/Conference Title

American journal of human genetics

PubMed ID

27236922

DOI of Published Version

10.1016/j.ajhg.2016.03.028

Abstract

Hearing impairment is the most common sensory deficit. It is frequently caused by the expression of an allele carrying a single dominant missense mutation. Herein, we show that a single intracochlear injection of an artificial microRNA carried in a viral vector can slow progression of hearing loss for up to 35 weeks in the Beethoven mouse, a murine model of non-syndromic human deafness caused by a dominant gain-of-function mutation in Tmc1 (transmembrane channel-like 1). This outcome is noteworthy because it demonstrates the feasibility of RNA-interference-mediated suppression of an endogenous deafness-causing allele to slow progression of hearing loss. Given that most autosomal-dominant non-syndromic hearing loss in humans is caused by this mechanism of action, microRNA-based therapeutics might be broadly applicable as a therapy for this type of deafness.

Keywords

OAfund

Journal Article Version

Version of Record

Published Article/Book Citation

Am J Hum Genet. 2016 Jun 2;98(6):1101-13. doi: 10.1016/j.ajhg.2016.03.028. Epub 2016 May 26.

Rights

© 2016 The Authors

Creative Commons License

Creative Commons License
This work is licensed under a Creative Commons Attribution-Noncommercial-No Derivative Works 4.0 License.

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URL

https://ir.uiowa.edu/oto_pubs/1