Antisense Therapy in Neurology
Identifieur interne : 000706 ( Pmc/Checkpoint ); précédent : 000705; suivant : 000707Antisense Therapy in Neurology
Auteurs : Joshua J. A. Lee ; Toshifumi Yokota [Canada]Source :
- Journal of Personalized Medicine [ 2075-4426 ] ; 2013.
Abstract
Antisense therapy is an approach to fighting diseases using short DNA-like molecules called antisense oligonucleotides. Recently, antisense therapy has emerged as an exciting and promising strategy for the treatment of various neurodegenerative and neuromuscular disorders. Previous and ongoing pre-clinical and clinical trials have provided encouraging early results. Spinal muscular atrophy (SMA), Huntington’s disease (HD), amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD), Fukuyama congenital muscular dystrophy (FCMD), dysferlinopathy (including limb-girdle muscular dystrophy 2B; LGMD2B, Miyoshi myopathy; MM, and distal myopathy with anterior tibial onset; DMAT), and myotonic dystrophy (DM) are all reported to be promising targets for antisense therapy. This paper focuses on the current progress of antisense therapies in neurology.
Url:
DOI: 10.3390/jpm3030144
PubMed: 25562650
PubMed Central: 4251390
Affiliations:
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<record><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">Antisense Therapy in Neurology</title><author><name sortKey="Lee, Joshua J A" sort="Lee, Joshua J A" uniqKey="Lee J" first="Joshua J. A." last="Lee">Joshua J. A. Lee</name><affiliation><nlm:aff id="af1-jpm-03-00144">Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, 8812-112 St, Edmonton T6G 2H7, Canada; E-Mail:<email>jjlee@ualberta.ca</email></nlm:aff></affiliation></author><author><name sortKey="Yokota, Toshifumi" sort="Yokota, Toshifumi" uniqKey="Yokota T" first="Toshifumi" last="Yokota">Toshifumi Yokota</name><affiliation><nlm:aff id="af1-jpm-03-00144">Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, 8812-112 St, Edmonton T6G 2H7, Canada; E-Mail:<email>jjlee@ualberta.ca</email></nlm:aff></affiliation><affiliation wicri:level="1"><nlm:aff id="af2-jpm-03-00144">The Friends of Garrett Cumming Research and Muscular Dystrophy Canada HM Toupin Neurological Science Research Chair, 8812-112 St, Edmonton T6G 2H7, Canada</nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea>The Friends of Garrett Cumming Research and Muscular Dystrophy Canada HM Toupin Neurological Science Research Chair, 8812-112 St, Edmonton T6G 2H7</wicri:regionArea><wicri:noRegion>Edmonton T6G 2H7</wicri:noRegion></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PMC</idno><idno type="pmid">25562650</idno><idno type="pmc">4251390</idno><idno type="url">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4251390</idno><idno type="RBID">PMC:4251390</idno><idno type="doi">10.3390/jpm3030144</idno><date when="2013">2013</date><idno type="wicri:Area/Pmc/Corpus">000C53</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Corpus" wicri:corpus="PMC">000C53</idno><idno type="wicri:Area/Pmc/Curation">000C53</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Curation">000C53</idno><idno type="wicri:Area/Pmc/Checkpoint">000706</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Checkpoint">000706</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en" level="a" type="main">Antisense Therapy in Neurology</title><author><name sortKey="Lee, Joshua J A" sort="Lee, Joshua J A" uniqKey="Lee J" first="Joshua J. A." last="Lee">Joshua J. A. Lee</name><affiliation><nlm:aff id="af1-jpm-03-00144">Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, 8812-112 St, Edmonton T6G 2H7, Canada; E-Mail:<email>jjlee@ualberta.ca</email></nlm:aff></affiliation></author><author><name sortKey="Yokota, Toshifumi" sort="Yokota, Toshifumi" uniqKey="Yokota T" first="Toshifumi" last="Yokota">Toshifumi Yokota</name><affiliation><nlm:aff id="af1-jpm-03-00144">Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, 8812-112 St, Edmonton T6G 2H7, Canada; E-Mail:<email>jjlee@ualberta.ca</email></nlm:aff></affiliation><affiliation wicri:level="1"><nlm:aff id="af2-jpm-03-00144">The Friends of Garrett Cumming Research and Muscular Dystrophy Canada HM Toupin Neurological Science Research Chair, 8812-112 St, Edmonton T6G 2H7, Canada</nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea>The Friends of Garrett Cumming Research and Muscular Dystrophy Canada HM Toupin Neurological Science Research Chair, 8812-112 St, Edmonton T6G 2H7</wicri:regionArea><wicri:noRegion>Edmonton T6G 2H7</wicri:noRegion></affiliation></author></analytic><series><title level="j">Journal of Personalized Medicine</title><idno type="eISSN">2075-4426</idno><imprint><date when="2013">2013</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en"><p>Antisense therapy is an approach to fighting diseases using short DNA-like molecules called antisense oligonucleotides. Recently, antisense therapy has emerged as an exciting and promising strategy for the treatment of various neurodegenerative and neuromuscular disorders. Previous and ongoing pre-clinical and clinical trials have provided encouraging early results. Spinal muscular atrophy (SMA), Huntington’s disease (HD), amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD), Fukuyama congenital muscular dystrophy (FCMD), dysferlinopathy (including limb-girdle muscular dystrophy 2B; LGMD2B, Miyoshi myopathy; MM, and distal myopathy with anterior tibial onset; DMAT), and myotonic dystrophy (DM) are all reported to be promising targets for antisense therapy. This paper focuses on the current progress of antisense therapies in neurology.</p></div></front><back><div1 type="bibliography"><listBibl><biblStruct><analytic><author><name sortKey="Kuzmiak, H A" uniqKey="Kuzmiak H">H.A. Kuzmiak</name></author><author><name sortKey="Maquat, L E" uniqKey="Maquat L">L.E. 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<front><journal-meta><journal-id journal-id-type="nlm-ta">J Pers Med</journal-id><journal-id journal-id-type="iso-abbrev">J Pers Med</journal-id><journal-id journal-id-type="publisher-id">jpm</journal-id><journal-title-group><journal-title>Journal of Personalized Medicine</journal-title></journal-title-group><issn pub-type="epub">2075-4426</issn><publisher><publisher-name>MDPI</publisher-name></publisher></journal-meta><article-meta><article-id pub-id-type="pmid">25562650</article-id><article-id pub-id-type="pmc">4251390</article-id><article-id pub-id-type="doi">10.3390/jpm3030144</article-id><article-id pub-id-type="publisher-id">jpm-03-00144</article-id><article-categories><subj-group subj-group-type="heading"><subject>Review</subject></subj-group></article-categories><title-group><article-title>Antisense Therapy in Neurology</article-title></title-group><contrib-group><contrib contrib-type="author"><name><surname>Lee</surname><given-names>Joshua J.A.</given-names></name><xref ref-type="aff" rid="af1-jpm-03-00144">1</xref></contrib><contrib contrib-type="author"><name><surname>Yokota</surname><given-names>Toshifumi</given-names></name><xref ref-type="aff" rid="af1-jpm-03-00144">1</xref><xref ref-type="aff" rid="af2-jpm-03-00144">2</xref><xref rid="c1-jpm-03-00144" ref-type="corresp">*</xref></contrib></contrib-group><aff id="af1-jpm-03-00144"><label>1</label>Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, 8812-112 St, Edmonton T6G 2H7, Canada; E-Mail:<email>jjlee@ualberta.ca</email></aff><aff id="af2-jpm-03-00144"><label>2</label>The Friends of Garrett Cumming Research and Muscular Dystrophy Canada HM Toupin Neurological Science Research Chair, 8812-112 St, Edmonton T6G 2H7, Canada</aff><author-notes><corresp id="c1-jpm-03-00144"><label>*</label>Author to whom correspondence should be addressed; E-Mail: <email>toshifum@ualberta.ca</email>; Tel.: +1-780-492-1102; Fax: +1-780-492-1998.</corresp></author-notes><pub-date pub-type="epub"><day>02</day><month>8</month><year>2013</year></pub-date><pub-date pub-type="collection"><month>9</month><year>2013</year></pub-date><volume>3</volume><issue>3</issue><fpage>144</fpage><lpage>176</lpage><history><date date-type="received"><day>27</day><month>5</month><year>2013</year></date><date date-type="rev-recd"><day>26</day><month>7</month><year>2013</year></date><date date-type="accepted"><day>29</day><month>7</month><year>2013</year></date></history><permissions><copyright-statement>© 2013 by the authors; licensee MDPI, Basel, Switzerland.</copyright-statement><copyright-year>2013</copyright-year><license><license-p><pmc-comment>CREATIVE COMMONS</pmc-comment>This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution license (<ext-link ext-link-type="uri" xlink:href="http://creativecommons.org/licenses/by/3.0/">http://creativecommons.org/licenses/by/3.0/</ext-link>).</license-p></license></permissions><abstract><p>Antisense therapy is an approach to fighting diseases using short DNA-like molecules called antisense oligonucleotides. Recently, antisense therapy has emerged as an exciting and promising strategy for the treatment of various neurodegenerative and neuromuscular disorders. Previous and ongoing pre-clinical and clinical trials have provided encouraging early results. Spinal muscular atrophy (SMA), Huntington’s disease (HD), amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD), Fukuyama congenital muscular dystrophy (FCMD), dysferlinopathy (including limb-girdle muscular dystrophy 2B; LGMD2B, Miyoshi myopathy; MM, and distal myopathy with anterior tibial onset; DMAT), and myotonic dystrophy (DM) are all reported to be promising targets for antisense therapy. This paper focuses on the current progress of antisense therapies in neurology.</p></abstract><kwd-group><kwd>Duchenne muscular dystrophy (DMD)</kwd><kwd>Fukuyama congenital muscular dystrophy (FCMD)</kwd><kwd>myotonic dystrophy (DM)</kwd><kwd>spinal muscular atrophy (SMA)</kwd><kwd>Huntington’s disease (HD)</kwd><kwd>amyotrophic lateral sclerosis (ALS)</kwd><kwd>limb-girdle muscular dystrophy 2B (LGMD2B)</kwd><kwd>Miyoshi myopathy (MM)</kwd><kwd>distal myopathy with anterior tibial onset (DMAT)</kwd><kwd>antisense therapy</kwd></kwd-group></article-meta></front></pmc><affiliations><list><country><li>Canada</li></country></list><tree><noCountry><name sortKey="Lee, Joshua J A" sort="Lee, Joshua J A" uniqKey="Lee J" first="Joshua J. A." last="Lee">Joshua J. A. Lee</name></noCountry><country name="Canada"><noRegion><name sortKey="Yokota, Toshifumi" sort="Yokota, Toshifumi" uniqKey="Yokota T" first="Toshifumi" last="Yokota">Toshifumi Yokota</name></noRegion></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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