Short (16-mer) locked nucleic acid splice-switching oligonucleotides restore dystrophin production in Duchenne Muscular Dystrophy myotubes
Identifieur interne : 000B93 ( Main/Exploration ); précédent : 000B92; suivant : 000B94Short (16-mer) locked nucleic acid splice-switching oligonucleotides restore dystrophin production in Duchenne Muscular Dystrophy myotubes
Auteurs : Vanessa Borges Pires [Portugal] ; Ricardo Sim Es [Portugal] ; Kamel Mamchaoui [France] ; Célia Carvalho [Portugal] ; Maria Carmo-Fonseca [Portugal]Source :
- PLoS ONE [ 1932-6203 ] ; 2017.
Descripteurs français
- KwdFr :
- Dystrophine (biosynthèse), Dystrophine (génétique), Exons, Fibres musculaires squelettiques (métabolisme), Humains, Isoformes de protéines (biosynthèse), Isoformes de protéines (génétique), Lignée cellulaire, Myopathie de Duchenne (), Myopathie de Duchenne (génétique), Myopathie de Duchenne (métabolisme), Oligonucléotides (génétique), Oligonucléotides (usage thérapeutique), Oligonucléotides antisens (génétique), Oligonucléotides antisens (usage thérapeutique), Thérapie génétique (), Épissage des ARN.
- MESH :
- biosynthèse : Dystrophine, Isoformes de protéines.
- génétique : Dystrophine, Isoformes de protéines, Myopathie de Duchenne, Oligonucléotides, Oligonucléotides antisens.
- métabolisme : Fibres musculaires squelettiques, Myopathie de Duchenne.
- usage thérapeutique : Oligonucléotides, Oligonucléotides antisens.
- Exons, Humains, Lignée cellulaire, Myopathie de Duchenne, Thérapie génétique, Épissage des ARN.
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.
Url:
DOI: 10.1371/journal.pone.0181065
PubMed: 28742140
PubMed Central: 5524367
Affiliations:
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Le document en format XML
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Dystrophy myotubes</title><author><name sortKey="Pires, Vanessa Borges" sort="Pires, Vanessa Borges" uniqKey="Pires V" first="Vanessa Borges" last="Pires">Vanessa Borges Pires</name><affiliation wicri:level="1"><nlm:aff id="aff001"><addr-line>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal</addr-line></nlm:aff><country xml:lang="fr">Portugal</country><wicri:regionArea>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa</wicri:regionArea><wicri:noRegion>Lisboa</wicri:noRegion></affiliation></author><author><name sortKey="Sim Es, Ricardo" sort="Sim Es, Ricardo" uniqKey="Sim Es R" first="Ricardo" last="Sim Es">Ricardo Sim Es</name><affiliation wicri:level="1"><nlm:aff id="aff001"><addr-line>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal</addr-line></nlm:aff><country xml:lang="fr">Portugal</country><wicri:regionArea>Instituto de Medicina Molecular, Faculdade 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(génétique)</term><term>Exons</term><term>Fibres musculaires squelettiques (métabolisme)</term><term>Humains</term><term>Isoformes de protéines (biosynthèse)</term><term>Isoformes de protéines (génétique)</term><term>Lignée cellulaire</term><term>Myopathie de Duchenne ()</term><term>Myopathie de Duchenne (génétique)</term><term>Myopathie de Duchenne (métabolisme)</term><term>Oligonucléotides (génétique)</term><term>Oligonucléotides (usage thérapeutique)</term><term>Oligonucléotides antisens (génétique)</term><term>Oligonucléotides antisens (usage thérapeutique)</term><term>Thérapie génétique ()</term><term>Épissage des ARN</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 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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" qualifier="usage thérapeutique" xml:lang="fr"><term>Oligonucléotides</term><term>Oligonucléotides antisens</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Cell Line</term><term>Exons</term><term>Humans</term><term>RNA Splicing</term></keywords><keywords scheme="MESH" xml:lang="fr"><term>Exons</term><term>Humains</term><term>Lignée cellulaire</term><term>Myopathie de Duchenne</term><term>Thérapie génétique</term><term>Épissage des ARN</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en"><p>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.</p></div></front><back><div1 type="bibliography"><listBibl><biblStruct><analytic><author><name sortKey="Mcclorey, G" uniqKey="Mcclorey G">G McClorey</name></author><author><name sortKey="Wood, Mj" uniqKey="Wood M">MJ Wood</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Lim, Krq" uniqKey="Lim K">KRQ Lim</name></author><author><name sortKey="Maruyama, R" uniqKey="Maruyama R">R Maruyama</name></author><author><name sortKey="Yokota, T" uniqKey="Yokota T">T Yokota</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Corey, Dr" uniqKey="Corey D">DR Corey</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Hoffman, Ep" uniqKey="Hoffman E">EP Hoffman</name></author><author><name sortKey="Brown, Rh" uniqKey="Brown R">RH Brown</name></author><author><name sortKey="Kunkel, Lm" uniqKey="Kunkel L">LM 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Santos</name></author><author><name sortKey="Petri, A" uniqKey="Petri A">A Petri</name></author><author><name sortKey="Heidenblad, M" uniqKey="Heidenblad M">M Heidenblad</name></author><author><name sortKey="Broom, O" uniqKey="Broom O">O Broom</name></author><author><name sortKey="Ruse, C" uniqKey="Ruse C">C Ruse</name></author></analytic></biblStruct></listBibl></div1></back></TEI><affiliations><list><country><li>France</li><li>Portugal</li></country><region><li>Île-de-France</li></region><settlement><li>Paris</li></settlement></list><tree><country name="Portugal"><noRegion><name sortKey="Pires, Vanessa Borges" sort="Pires, Vanessa Borges" uniqKey="Pires V" first="Vanessa Borges" last="Pires">Vanessa Borges Pires</name></noRegion><name sortKey="Carmo Fonseca, Maria" sort="Carmo Fonseca, Maria" uniqKey="Carmo Fonseca M" first="Maria" last="Carmo-Fonseca">Maria Carmo-Fonseca</name><name sortKey="Carvalho, Celia" sort="Carvalho, Celia" uniqKey="Carvalho C" first="Célia" last="Carvalho">Célia Carvalho</name><name sortKey="Sim Es, Ricardo" sort="Sim Es, Ricardo" uniqKey="Sim Es R" first="Ricardo" last="Sim Es">Ricardo Sim Es</name></country><country name="France"><region name="Île-de-France"><name sortKey="Mamchaoui, Kamel" sort="Mamchaoui, Kamel" uniqKey="Mamchaoui K" first="Kamel" last="Mamchaoui">Kamel Mamchaoui</name></region></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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