Short (16-mer) locked nucleic acid splice-switching oligonucleotides restore dystrophin production in Duchenne Muscular Dystrophy myotubes.
Identifieur interne : 000C17 ( PubMed/Corpus ); précédent : 000C16; suivant : 000C18Short (16-mer) locked nucleic acid splice-switching oligonucleotides restore dystrophin production in Duchenne Muscular Dystrophy myotubes.
Auteurs : Vanessa Borges Pires ; Ricardo Sim Es ; Kamel Mamchaoui ; Célia Carvalho ; Maria Carmo-FonsecaSource :
- PloS one [ 1932-6203 ] ; 2017.
English descriptors
- KwdEn :
- Cell Line, Dystrophin (biosynthesis), Dystrophin (genetics), Exons, Genetic Therapy (methods), Humans, Muscle Fibers, Skeletal (metabolism), Muscular Dystrophy, Duchenne (genetics), Muscular Dystrophy, Duchenne (metabolism), Muscular Dystrophy, Duchenne (therapy), Oligonucleotides (genetics), Oligonucleotides (therapeutic use), Oligonucleotides, Antisense (genetics), Oligonucleotides, Antisense (therapeutic use), Protein Isoforms (biosynthesis), Protein Isoforms (genetics), RNA Splicing.
- MESH :
- chemical , biosynthesis : Dystrophin, Protein Isoforms.
- chemical , genetics : Dystrophin, Oligonucleotides, Oligonucleotides, Antisense, Protein Isoforms.
- genetics : Muscular Dystrophy, Duchenne.
- metabolism : Muscle Fibers, Skeletal, Muscular Dystrophy, Duchenne.
- methods : Genetic Therapy.
- chemical , therapeutic use : Oligonucleotides, Oligonucleotides, Antisense.
- therapy : Muscular Dystrophy, Duchenne.
- Cell Line, Exons, Humans, RNA Splicing.
Abstract
Splice-switching antisense oligonucleotides (SSOs) offer great potential for RNA-targeting therapies, and two SSO drugs have been recently approved for treating Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA). Despite promising results, new developments are still needed for more efficient chemistries and delivery systems. Locked nucleic acid (LNA) is a chemically modified nucleic acid that presents several attractive properties, such as high melting temperature when bound to RNA, potent biological activity, high stability and low toxicity in vivo. Here, we designed a series of LNA-based SSOs complementary to two sequences of the human dystrophin exon 51 that are most evolutionary conserved and evaluated their ability to induce exon skipping upon transfection into myoblasts derived from a DMD patient. We show that 16-mers with 60% of LNA modification efficiently induce exon skipping and restore synthesis of a truncated dystrophin isoform that localizes to the plasma membrane of patient-derived myotubes differentiated in culture. In sum, this study underscores the value of short LNA-modified SSOs for therapeutic applications.
DOI: 10.1371/journal.pone.0181065
PubMed: 28742140
Links to Exploration step
pubmed:28742140Le document en format XML
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Mamchaoui</name><affiliation><nlm:affiliation>Center for Research in Myology, INSERM UMRS974, CNRS FRE3617, Sorbonne Universités, UPMC Univ Paris 06, Paris, France.</nlm:affiliation></affiliation></author><author><name sortKey="Carvalho, Celia" sort="Carvalho, Celia" uniqKey="Carvalho C" first="Célia" last="Carvalho">Célia Carvalho</name><affiliation><nlm:affiliation>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal.</nlm:affiliation></affiliation></author><author><name sortKey="Carmo Fonseca, Maria" sort="Carmo Fonseca, Maria" uniqKey="Carmo Fonseca M" first="Maria" last="Carmo-Fonseca">Maria Carmo-Fonseca</name><affiliation><nlm:affiliation>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal.</nlm:affiliation></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PubMed</idno><date when="2017">2017</date><idno type="RBID">pubmed:28742140</idno><idno 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Universidade de Lisboa, Lisboa, Portugal.</nlm:affiliation></affiliation></author><author><name sortKey="Mamchaoui, Kamel" sort="Mamchaoui, Kamel" uniqKey="Mamchaoui K" first="Kamel" last="Mamchaoui">Kamel Mamchaoui</name><affiliation><nlm:affiliation>Center for Research in Myology, INSERM UMRS974, CNRS FRE3617, Sorbonne Universités, UPMC Univ Paris 06, Paris, France.</nlm:affiliation></affiliation></author><author><name sortKey="Carvalho, Celia" sort="Carvalho, Celia" uniqKey="Carvalho C" first="Célia" last="Carvalho">Célia Carvalho</name><affiliation><nlm:affiliation>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal.</nlm:affiliation></affiliation></author><author><name sortKey="Carmo Fonseca, Maria" sort="Carmo Fonseca, Maria" uniqKey="Carmo Fonseca M" first="Maria" last="Carmo-Fonseca">Maria Carmo-Fonseca</name><affiliation><nlm:affiliation>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal.</nlm:affiliation></affiliation></author></analytic><series><title level="j">PloS one</title><idno type="eISSN">1932-6203</idno><imprint><date when="2017" type="published">2017</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Cell Line</term><term>Dystrophin (biosynthesis)</term><term>Dystrophin (genetics)</term><term>Exons</term><term>Genetic Therapy (methods)</term><term>Humans</term><term>Muscle Fibers, Skeletal (metabolism)</term><term>Muscular Dystrophy, Duchenne (genetics)</term><term>Muscular Dystrophy, Duchenne (metabolism)</term><term>Muscular Dystrophy, Duchenne (therapy)</term><term>Oligonucleotides (genetics)</term><term>Oligonucleotides (therapeutic use)</term><term>Oligonucleotides, Antisense (genetics)</term><term>Oligonucleotides, Antisense (therapeutic use)</term><term>Protein Isoforms (biosynthesis)</term><term>Protein Isoforms (genetics)</term><term>RNA Splicing</term></keywords><keywords scheme="MESH" type="chemical" qualifier="biosynthesis" xml:lang="en"><term>Dystrophin</term><term>Protein Isoforms</term></keywords><keywords scheme="MESH" type="chemical" qualifier="genetics" xml:lang="en"><term>Dystrophin</term><term>Oligonucleotides</term><term>Oligonucleotides, Antisense</term><term>Protein Isoforms</term></keywords><keywords scheme="MESH" qualifier="genetics" xml:lang="en"><term>Muscular Dystrophy, Duchenne</term></keywords><keywords scheme="MESH" qualifier="metabolism" xml:lang="en"><term>Muscle Fibers, Skeletal</term><term>Muscular Dystrophy, Duchenne</term></keywords><keywords scheme="MESH" qualifier="methods" xml:lang="en"><term>Genetic Therapy</term></keywords><keywords scheme="MESH" type="chemical" qualifier="therapeutic use" xml:lang="en"><term>Oligonucleotides</term><term>Oligonucleotides, Antisense</term></keywords><keywords scheme="MESH" qualifier="therapy" xml:lang="en"><term>Muscular Dystrophy, Duchenne</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Cell Line</term><term>Exons</term><term>Humans</term><term>RNA Splicing</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Splice-switching antisense oligonucleotides (SSOs) offer great potential for RNA-targeting therapies, and two SSO drugs have been recently approved for treating Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA). Despite promising results, new developments are still needed for more efficient chemistries and delivery systems. Locked nucleic acid (LNA) is a chemically modified nucleic acid that presents several attractive properties, such as high melting temperature when bound to RNA, potent biological activity, high stability and low toxicity in vivo. Here, we designed a series of LNA-based SSOs complementary to two sequences of the human dystrophin exon 51 that are most evolutionary conserved and evaluated their ability to induce exon skipping upon transfection into myoblasts derived from a DMD patient. We show that 16-mers with 60% of LNA modification efficiently induce exon skipping and restore synthesis of a truncated dystrophin isoform that localizes to the plasma membrane of patient-derived myotubes differentiated in culture. In sum, this study underscores the value of short LNA-modified SSOs for therapeutic applications.</div></front></TEI><pubmed><MedlineCitation Status="MEDLINE" Owner="NLM"><PMID Version="1">28742140</PMID><DateCompleted><Year>2017</Year><Month>09</Month><Day>25</Day></DateCompleted><DateRevised><Year>2018</Year><Month>11</Month><Day>13</Day></DateRevised><Article PubModel="Electronic-eCollection"><Journal><ISSN IssnType="Electronic">1932-6203</ISSN><JournalIssue CitedMedium="Internet"><Volume>12</Volume><Issue>7</Issue><PubDate><Year>2017</Year></PubDate></JournalIssue><Title>PloS one</Title><ISOAbbreviation>PLoS ONE</ISOAbbreviation></Journal><ArticleTitle>Short (16-mer) locked nucleic acid splice-switching oligonucleotides restore dystrophin production in Duchenne Muscular Dystrophy myotubes.</ArticleTitle><Pagination><MedlinePgn>e0181065</MedlinePgn></Pagination><ELocationID EIdType="doi" ValidYN="Y">10.1371/journal.pone.0181065</ELocationID><Abstract><AbstractText>Splice-switching antisense oligonucleotides (SSOs) offer great potential for RNA-targeting therapies, and two SSO drugs have been recently approved for treating Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA). Despite promising results, new developments are still needed for more efficient chemistries and delivery systems. Locked nucleic acid (LNA) is a chemically modified nucleic acid that presents several attractive properties, such as high melting temperature when bound to RNA, potent biological activity, high stability and low toxicity in vivo. Here, we designed a series of LNA-based SSOs complementary to two sequences of the human dystrophin exon 51 that are most evolutionary conserved and evaluated their ability to induce exon skipping upon transfection into myoblasts derived from a DMD patient. We show that 16-mers with 60% of LNA modification efficiently induce exon skipping and restore synthesis of a truncated dystrophin isoform that localizes to the plasma membrane of patient-derived myotubes differentiated in culture. In sum, this study underscores the value of short LNA-modified SSOs for therapeutic applications.</AbstractText></Abstract><AuthorList CompleteYN="Y"><Author ValidYN="Y"><LastName>Pires</LastName><ForeName>Vanessa Borges</ForeName><Initials>VB</Initials><AffiliationInfo><Affiliation>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Simões</LastName><ForeName>Ricardo</ForeName><Initials>R</Initials><AffiliationInfo><Affiliation>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Mamchaoui</LastName><ForeName>Kamel</ForeName><Initials>K</Initials><AffiliationInfo><Affiliation>Center for Research in Myology, INSERM UMRS974, CNRS FRE3617, Sorbonne Universités, UPMC Univ Paris 06, Paris, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Carvalho</LastName><ForeName>Célia</ForeName><Initials>C</Initials><Identifier Source="ORCID">http://orcid.org/0000-0002-5980-595X</Identifier><AffiliationInfo><Affiliation>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Carmo-Fonseca</LastName><ForeName>Maria</ForeName><Initials>M</Initials><AffiliationInfo><Affiliation>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal.</Affiliation></AffiliationInfo></Author></AuthorList><Language>eng</Language><PublicationTypeList><PublicationType UI="D016428">Journal Article</PublicationType></PublicationTypeList><ArticleDate DateType="Electronic"><Year>2017</Year><Month>07</Month><Day>24</Day></ArticleDate></Article><MedlineJournalInfo><Country>United States</Country><MedlineTA>PLoS 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