Exon Snipping in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a life-limiting neuromuscular disorder caused by mutations in the DMD gene encoding dystrophin. We discuss very recent studies that used CRISPR/Cas9 technology to ‘snip out’ mutated exons in DMD , restoring the reading frame of the gene. We also present cautionar...
Gespeichert in:
| Veröffentlicht in: | Trends in molecular medicine 2016-03, Vol.22 (3), p.187-189 |
|---|---|
| Hauptverfasser: | , |
| Format: | Artikel |
| Sprache: | eng |
| Schlagworte: | |
| Online-Zugang: | Volltext |
| Tags: |
Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
|
| container_end_page | 189 |
|---|---|
| container_issue | 3 |
| container_start_page | 187 |
| container_title | Trends in molecular medicine |
| container_volume | 22 |
| creator | Kemaladewi, Dwi U Cohn, Ronald D |
| description | Duchenne muscular dystrophy (DMD) is a life-limiting neuromuscular disorder caused by mutations in the DMD gene encoding dystrophin. We discuss very recent studies that used CRISPR/Cas9 technology to ‘snip out’ mutated exons in DMD , restoring the reading frame of the gene. We also present cautionary aspects of translating this exciting technology into clinical practice. |
| doi_str_mv | 10.1016/j.molmed.2016.01.007 |
| format | Article |
| fullrecord | <record><control><sourceid>proquest_cross</sourceid><recordid>TN_cdi_proquest_miscellaneous_1769625137</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><els_id>1_s2_0_S1471491416000186</els_id><sourcerecordid>1769625137</sourcerecordid><originalsourceid>FETCH-LOGICAL-c417t-70eda00f3921877d2e996356f47e4e55100fda214ebde939643f1639c388b81f3</originalsourceid><addsrcrecordid>eNqFkU1PwzAMhiME4vsfINQjB1biJk2aCxIanxKIw0DiFnWpCxldWpIVsX9Pqg0OXDjZll_b8vMScgQ0BQribJbO22aOVZrFKqWQUio3yC5wCSOu1Mvmbw58h-yFMKMUcimLbbKTiSIXGZO75PTqq3XJxNmus-41sS657M0bOofJQx9M35Q-uVyGhW-7t-UB2arLJuDhOu6T5-urp_Ht6P7x5m58cT8yHORiJClWJaU1UxkUUlYZKiVYLmoukWOeQ-xVZQYcpxUqpgRnNQimDCuKaQE12ycnq72dbz96DAs9t8Fg05QO2z5okEKJLAcmo5SvpMa3IXisdeftvPRLDVQPnPRMrzjpgZOmoCOnOHa8vtBPh97P0A-YKDhfCTD--WnR62AsOoOV9WgWumrtfxf-LjCNddaUzTsuMcza3rvIUIMOmaZ6Mng1WAWCRp8Kwb4BQ2ON8Q</addsrcrecordid><sourcetype>Aggregation Database</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>1769625137</pqid></control><display><type>article</type><title>Exon Snipping in Duchenne Muscular Dystrophy</title><source>MEDLINE</source><source>ScienceDirect Journals (5 years ago - present)</source><creator>Kemaladewi, Dwi U ; Cohn, Ronald D</creator><creatorcontrib>Kemaladewi, Dwi U ; Cohn, Ronald D</creatorcontrib><description>Duchenne muscular dystrophy (DMD) is a life-limiting neuromuscular disorder caused by mutations in the DMD gene encoding dystrophin. We discuss very recent studies that used CRISPR/Cas9 technology to ‘snip out’ mutated exons in DMD , restoring the reading frame of the gene. We also present cautionary aspects of translating this exciting technology into clinical practice.</description><identifier>ISSN: 1471-4914</identifier><identifier>EISSN: 1471-499X</identifier><identifier>DOI: 10.1016/j.molmed.2016.01.007</identifier><identifier>PMID: 26856237</identifier><language>eng</language><publisher>England: Elsevier Ltd</publisher><subject>Animals ; CRISPR-Cas Systems ; Dystrophin - genetics ; Exons - genetics ; Genetic Therapy - methods ; Male ; Muscle, Skeletal - metabolism ; Muscular Dystrophy, Duchenne - therapy ; Pathology</subject><ispartof>Trends in molecular medicine, 2016-03, Vol.22 (3), p.187-189</ispartof><rights>Elsevier Ltd</rights><rights>2016 Elsevier Ltd</rights><rights>Copyright © 2016 Elsevier Ltd. All rights reserved.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c417t-70eda00f3921877d2e996356f47e4e55100fda214ebde939643f1639c388b81f3</citedby><cites>FETCH-LOGICAL-c417t-70eda00f3921877d2e996356f47e4e55100fda214ebde939643f1639c388b81f3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://dx.doi.org/10.1016/j.molmed.2016.01.007$$EHTML$$P50$$Gelsevier$$H</linktohtml><link.rule.ids>314,780,784,3548,27923,27924,45994</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/26856237$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Kemaladewi, Dwi U</creatorcontrib><creatorcontrib>Cohn, Ronald D</creatorcontrib><title>Exon Snipping in Duchenne Muscular Dystrophy</title><title>Trends in molecular medicine</title><addtitle>Trends Mol Med</addtitle><description>Duchenne muscular dystrophy (DMD) is a life-limiting neuromuscular disorder caused by mutations in the DMD gene encoding dystrophin. We discuss very recent studies that used CRISPR/Cas9 technology to ‘snip out’ mutated exons in DMD , restoring the reading frame of the gene. We also present cautionary aspects of translating this exciting technology into clinical practice.</description><subject>Animals</subject><subject>CRISPR-Cas Systems</subject><subject>Dystrophin - genetics</subject><subject>Exons - genetics</subject><subject>Genetic Therapy - methods</subject><subject>Male</subject><subject>Muscle, Skeletal - metabolism</subject><subject>Muscular Dystrophy, Duchenne - therapy</subject><subject>Pathology</subject><issn>1471-4914</issn><issn>1471-499X</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2016</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqFkU1PwzAMhiME4vsfINQjB1biJk2aCxIanxKIw0DiFnWpCxldWpIVsX9Pqg0OXDjZll_b8vMScgQ0BQribJbO22aOVZrFKqWQUio3yC5wCSOu1Mvmbw58h-yFMKMUcimLbbKTiSIXGZO75PTqq3XJxNmus-41sS657M0bOofJQx9M35Q-uVyGhW-7t-UB2arLJuDhOu6T5-urp_Ht6P7x5m58cT8yHORiJClWJaU1UxkUUlYZKiVYLmoukWOeQ-xVZQYcpxUqpgRnNQimDCuKaQE12ycnq72dbz96DAs9t8Fg05QO2z5okEKJLAcmo5SvpMa3IXisdeftvPRLDVQPnPRMrzjpgZOmoCOnOHa8vtBPh97P0A-YKDhfCTD--WnR62AsOoOV9WgWumrtfxf-LjCNddaUzTsuMcza3rvIUIMOmaZ6Mng1WAWCRp8Kwb4BQ2ON8Q</recordid><startdate>20160301</startdate><enddate>20160301</enddate><creator>Kemaladewi, Dwi U</creator><creator>Cohn, Ronald D</creator><general>Elsevier Ltd</general><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>7X8</scope></search><sort><creationdate>20160301</creationdate><title>Exon Snipping in Duchenne Muscular Dystrophy</title><author>Kemaladewi, Dwi U ; Cohn, Ronald D</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c417t-70eda00f3921877d2e996356f47e4e55100fda214ebde939643f1639c388b81f3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2016</creationdate><topic>Animals</topic><topic>CRISPR-Cas Systems</topic><topic>Dystrophin - genetics</topic><topic>Exons - genetics</topic><topic>Genetic Therapy - methods</topic><topic>Male</topic><topic>Muscle, Skeletal - metabolism</topic><topic>Muscular Dystrophy, Duchenne - therapy</topic><topic>Pathology</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Kemaladewi, Dwi U</creatorcontrib><creatorcontrib>Cohn, Ronald D</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><jtitle>Trends in molecular medicine</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Kemaladewi, Dwi U</au><au>Cohn, Ronald D</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Exon Snipping in Duchenne Muscular Dystrophy</atitle><jtitle>Trends in molecular medicine</jtitle><addtitle>Trends Mol Med</addtitle><date>2016-03-01</date><risdate>2016</risdate><volume>22</volume><issue>3</issue><spage>187</spage><epage>189</epage><pages>187-189</pages><issn>1471-4914</issn><eissn>1471-499X</eissn><abstract>Duchenne muscular dystrophy (DMD) is a life-limiting neuromuscular disorder caused by mutations in the DMD gene encoding dystrophin. We discuss very recent studies that used CRISPR/Cas9 technology to ‘snip out’ mutated exons in DMD , restoring the reading frame of the gene. We also present cautionary aspects of translating this exciting technology into clinical practice.</abstract><cop>England</cop><pub>Elsevier Ltd</pub><pmid>26856237</pmid><doi>10.1016/j.molmed.2016.01.007</doi><tpages>3</tpages></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 1471-4914 |
| ispartof | Trends in molecular medicine, 2016-03, Vol.22 (3), p.187-189 |
| issn | 1471-4914 1471-499X |
| language | eng |
| recordid | cdi_proquest_miscellaneous_1769625137 |
| source | MEDLINE; ScienceDirect Journals (5 years ago - present) |
| subjects | Animals CRISPR-Cas Systems Dystrophin - genetics Exons - genetics Genetic Therapy - methods Male Muscle, Skeletal - metabolism Muscular Dystrophy, Duchenne - therapy Pathology |
| title | Exon Snipping in Duchenne Muscular Dystrophy |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2025-01-10T11%3A08%3A14IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-proquest_cross&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Exon%20Snipping%20in%20Duchenne%20Muscular%20Dystrophy&rft.jtitle=Trends%20in%20molecular%20medicine&rft.au=Kemaladewi,%20Dwi%20U&rft.date=2016-03-01&rft.volume=22&rft.issue=3&rft.spage=187&rft.epage=189&rft.pages=187-189&rft.issn=1471-4914&rft.eissn=1471-499X&rft_id=info:doi/10.1016/j.molmed.2016.01.007&rft_dat=%3Cproquest_cross%3E1769625137%3C/proquest_cross%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_pqid=1769625137&rft_id=info:pmid/26856237&rft_els_id=1_s2_0_S1471491416000186&rfr_iscdi=true |