RNA Interference Prevents Autosomal-Dominant Hearing Loss
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 f...
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| Veröffentlicht in: | American journal of human genetics 2016-06, Vol.98 (6), p.1101-1113 |
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| container_title | American journal of human genetics |
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| creator | Shibata, Seiji B. Ranum, Paul T. Moteki, Hideaki Pan, Bifeng Goodwin, Alexander T. Goodman, Shawn S. Abbas, Paul J. Holt, Jeffrey R. Smith, Richard J.H. |
| description | 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. |
| doi_str_mv | 10.1016/j.ajhg.2016.03.028 |
| format | Article |
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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.</description><identifier>ISSN: 0002-9297</identifier><identifier>EISSN: 1537-6605</identifier><identifier>DOI: 10.1016/j.ajhg.2016.03.028</identifier><identifier>PMID: 27236922</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject>Animals ; Auditory Pathways ; Deafness ; Dependovirus - genetics ; Gene expression ; Hearing Loss - etiology ; Hearing Loss - pathology ; Hearing Loss - prevention & control ; Humans ; Mechanotransduction, Cellular ; Membrane Proteins - antagonists & inhibitors ; Membrane Proteins - physiology ; Mice ; Mice, Inbred C3H ; Mice, Knockout ; MicroRNAs - administration & dosage ; MicroRNAs - genetics ; Mutation ; Mutation, Missense - genetics ; Ribonucleic acid ; RNA ; RNA Interference ; Rodents</subject><ispartof>American journal of human genetics, 2016-06, Vol.98 (6), p.1101-1113</ispartof><rights>2016 The Authors</rights><rights>Copyright © 2016 The Authors. Published by Elsevier Inc. All rights reserved.</rights><rights>Copyright Cell Press Jun 2, 2016</rights><rights>2016 The Authors 2016</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c516t-bf6fffce21673557b01d9b7a67003ff1f1c1fb31a90598df63b2228693fd5f023</citedby><cites>FETCH-LOGICAL-c516t-bf6fffce21673557b01d9b7a67003ff1f1c1fb31a90598df63b2228693fd5f023</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC4908151/pdf/$$EPDF$$P50$$Gpubmedcentral$$Hfree_for_read</linktopdf><linktohtml>$$Uhttps://www.sciencedirect.com/science/article/pii/S0002929716300945$$EHTML$$P50$$Gelsevier$$Hfree_for_read</linktohtml><link.rule.ids>230,314,723,776,780,881,3537,27901,27902,53766,53768,65306</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/27236922$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Shibata, Seiji B.</creatorcontrib><creatorcontrib>Ranum, Paul T.</creatorcontrib><creatorcontrib>Moteki, Hideaki</creatorcontrib><creatorcontrib>Pan, Bifeng</creatorcontrib><creatorcontrib>Goodwin, Alexander T.</creatorcontrib><creatorcontrib>Goodman, Shawn S.</creatorcontrib><creatorcontrib>Abbas, Paul J.</creatorcontrib><creatorcontrib>Holt, Jeffrey R.</creatorcontrib><creatorcontrib>Smith, Richard J.H.</creatorcontrib><title>RNA Interference Prevents Autosomal-Dominant Hearing Loss</title><title>American journal of human genetics</title><addtitle>Am J Hum Genet</addtitle><description>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.</description><subject>Animals</subject><subject>Auditory Pathways</subject><subject>Deafness</subject><subject>Dependovirus - genetics</subject><subject>Gene expression</subject><subject>Hearing Loss - etiology</subject><subject>Hearing Loss - pathology</subject><subject>Hearing Loss - prevention & control</subject><subject>Humans</subject><subject>Mechanotransduction, Cellular</subject><subject>Membrane Proteins - antagonists & inhibitors</subject><subject>Membrane Proteins - physiology</subject><subject>Mice</subject><subject>Mice, Inbred C3H</subject><subject>Mice, Knockout</subject><subject>MicroRNAs - administration & dosage</subject><subject>MicroRNAs - genetics</subject><subject>Mutation</subject><subject>Mutation, Missense - genetics</subject><subject>Ribonucleic acid</subject><subject>RNA</subject><subject>RNA Interference</subject><subject>Rodents</subject><issn>0002-9297</issn><issn>1537-6605</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2016</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqFkU1rFTEUhoMo9lr9Ay5kwI2bGU8Sk0xAhEv9aOFSRXQdMpnkNsNMUpOZC_57M9xabBd2lUCe83LyPgi9xNBgwPzt0Ojhat-Qcm-ANkDaR2iDGRU158Aeow0AkFoSKU7Qs5wHAIxboE_RCRGEcknIBsnvl9vqIsw2OZtsMLb6luzBhjlX22WOOU56rD_GyQcd5urc6uTDvtrFnJ-jJ06P2b64OU_Rz8-ffpyd17uvXy7OtrvaMMznunPcOWcswVxQxkQHuJed0FwAUOewwwa7jmItgcm2d5x2hJCWS-p65oDQU_ThmHu9dJPtTdkt6VFdJz_p9FtF7dXdl-Cv1D4e1DsJLWa4BLy5CUjx12LzrCafjR1HHWxcsiqdtBxzLvjDqJBUAjBGC_r6HjrEJYXSxEpxIVpGZaHIkTKpVJasu90bg1olqkGtEtUqUQFVRWIZevXvj29H_lorwPsjYEvvB2-Tysav8nqfrJlVH_3_8v8AzdOsbg</recordid><startdate>20160602</startdate><enddate>20160602</enddate><creator>Shibata, Seiji B.</creator><creator>Ranum, Paul T.</creator><creator>Moteki, Hideaki</creator><creator>Pan, Bifeng</creator><creator>Goodwin, Alexander T.</creator><creator>Goodman, Shawn S.</creator><creator>Abbas, Paul J.</creator><creator>Holt, Jeffrey R.</creator><creator>Smith, Richard J.H.</creator><general>Elsevier Inc</general><general>Cell Press</general><general>Elsevier</general><scope>6I.</scope><scope>AAFTH</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7QP</scope><scope>7TK</scope><scope>7TM</scope><scope>7U7</scope><scope>8FD</scope><scope>C1K</scope><scope>FR3</scope><scope>K9.</scope><scope>NAPCQ</scope><scope>P64</scope><scope>RC3</scope><scope>7X8</scope><scope>5PM</scope></search><sort><creationdate>20160602</creationdate><title>RNA Interference Prevents Autosomal-Dominant Hearing Loss</title><author>Shibata, Seiji B. ; 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| source | MEDLINE; Cell Press Free Archives; Elsevier ScienceDirect Journals; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; PubMed Central |
| subjects | Animals Auditory Pathways Deafness Dependovirus - genetics Gene expression Hearing Loss - etiology Hearing Loss - pathology Hearing Loss - prevention & control Humans Mechanotransduction, Cellular Membrane Proteins - antagonists & inhibitors Membrane Proteins - physiology Mice Mice, Inbred C3H Mice, Knockout MicroRNAs - administration & dosage MicroRNAs - genetics Mutation Mutation, Missense - genetics Ribonucleic acid RNA RNA Interference Rodents |
| title | RNA Interference Prevents Autosomal-Dominant Hearing Loss |
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