| First Author | Shibata SB | Year | 2016 |
| Journal | Am J Hum Genet | Volume | 98 |
| Issue | 6 | Pages | 1101-1113 |
| PubMed ID | 27236922 | Mgi Jnum | J:252207 |
| Mgi Id | MGI:6093592 | Doi | 10.1016/j.ajhg.2016.03.028 |
| Citation | Shibata SB, et al. (2016) RNA Interference Prevents Autosomal-Dominant Hearing Loss. Am J Hum Genet 98(6):1101-1113 |
| abstractText | 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. |
