Making sense of antisense oligonucleotides: A narrative review
ABSTRACT Synthetic nucleic acid sequences that bind to ribonucleic acid (RNA) through Watson‐Crick base pairing are known as antisense oligonucleotides (ASOs) because they are complementary to “sense strand” nucleic acids. ASOs bind to selected sequences of RNA and regulate the expression of genes b...
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| Veröffentlicht in: | Muscle & nerve 2018-03, Vol.57 (3), p.356-370 |
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| container_title | Muscle & nerve |
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| creator | Goyal, Neelam Narayanaswami, Pushpa |
| description | ABSTRACT
Synthetic nucleic acid sequences that bind to ribonucleic acid (RNA) through Watson‐Crick base pairing are known as antisense oligonucleotides (ASOs) because they are complementary to “sense strand” nucleic acids. ASOs bind to selected sequences of RNA and regulate the expression of genes by several mechanisms depending on their chemical properties and targets. They can be used to restore deficient protein expression, reduce the expression of a toxic protein, modify functional effects of proteins, or reduce toxicity of mutant proteins. Two ASOs were approved by the U.S. Food and Drug Administration in 2016: eteplirsen for Duchenne muscular dystrophy and nusinersen for spinal muscular atrophy. Clinical trials in amyotrophic lateral sclerosis and familial amyloid polyneuropathy are ongoing. We review the chemistry, pharmacology, and mechanisms of action of ASOs, preclinical data, and clinical trials in neuromuscular diseases and discuss some ethical, regulatory, and policy considerations in the clinical development and use of ASOs. Muscle Nerve 57: 356–370, 2018 |
| doi_str_mv | 10.1002/mus.26001 |
| format | Article |
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Synthetic nucleic acid sequences that bind to ribonucleic acid (RNA) through Watson‐Crick base pairing are known as antisense oligonucleotides (ASOs) because they are complementary to “sense strand” nucleic acids. ASOs bind to selected sequences of RNA and regulate the expression of genes by several mechanisms depending on their chemical properties and targets. They can be used to restore deficient protein expression, reduce the expression of a toxic protein, modify functional effects of proteins, or reduce toxicity of mutant proteins. Two ASOs were approved by the U.S. Food and Drug Administration in 2016: eteplirsen for Duchenne muscular dystrophy and nusinersen for spinal muscular atrophy. Clinical trials in amyotrophic lateral sclerosis and familial amyloid polyneuropathy are ongoing. We review the chemistry, pharmacology, and mechanisms of action of ASOs, preclinical data, and clinical trials in neuromuscular diseases and discuss some ethical, regulatory, and policy considerations in the clinical development and use of ASOs. Muscle Nerve 57: 356–370, 2018</description><identifier>ISSN: 0148-639X</identifier><identifier>EISSN: 1097-4598</identifier><identifier>DOI: 10.1002/mus.26001</identifier><identifier>PMID: 29105153</identifier><language>eng</language><publisher>United States: Wiley Subscription Services, Inc</publisher><subject>Amyloid ; Amyotrophic lateral sclerosis ; Antisense oligonucleotides ; Atrophy ; Chemical properties ; Clinical trials ; Duchenne muscular dystrophy ; Duchenne's muscular dystrophy ; Dystrophy ; eteplirsen ; Ethics ; Gene expression ; Gene sequencing ; Medical research ; Muscles ; Muscular dystrophy ; Neuromuscular diseases ; Nucleic acids ; nusinersen ; Oligonucleotides ; Pharmacology ; Polyneuropathy ; Proteins ; Ribonucleic acid ; RNA ; Spinal muscular atrophy ; Toxicity</subject><ispartof>Muscle & nerve, 2018-03, Vol.57 (3), p.356-370</ispartof><rights>2017 Wiley Periodicals, Inc.</rights><rights>2018 Wiley Periodicals, Inc.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c4191-1d12bbf7bd6c6c244273a682a215322e42521f109689a3b1bf59e48b66ce77f33</citedby><cites>FETCH-LOGICAL-c4191-1d12bbf7bd6c6c244273a682a215322e42521f109689a3b1bf59e48b66ce77f33</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1002%2Fmus.26001$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1002%2Fmus.26001$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>314,777,781,1412,27905,27906,45555,45556</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/29105153$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Goyal, Neelam</creatorcontrib><creatorcontrib>Narayanaswami, Pushpa</creatorcontrib><title>Making sense of antisense oligonucleotides: A narrative review</title><title>Muscle & nerve</title><addtitle>Muscle Nerve</addtitle><description>ABSTRACT
Synthetic nucleic acid sequences that bind to ribonucleic acid (RNA) through Watson‐Crick base pairing are known as antisense oligonucleotides (ASOs) because they are complementary to “sense strand” nucleic acids. ASOs bind to selected sequences of RNA and regulate the expression of genes by several mechanisms depending on their chemical properties and targets. They can be used to restore deficient protein expression, reduce the expression of a toxic protein, modify functional effects of proteins, or reduce toxicity of mutant proteins. Two ASOs were approved by the U.S. Food and Drug Administration in 2016: eteplirsen for Duchenne muscular dystrophy and nusinersen for spinal muscular atrophy. Clinical trials in amyotrophic lateral sclerosis and familial amyloid polyneuropathy are ongoing. We review the chemistry, pharmacology, and mechanisms of action of ASOs, preclinical data, and clinical trials in neuromuscular diseases and discuss some ethical, regulatory, and policy considerations in the clinical development and use of ASOs. Muscle Nerve 57: 356–370, 2018</description><subject>Amyloid</subject><subject>Amyotrophic lateral sclerosis</subject><subject>Antisense oligonucleotides</subject><subject>Atrophy</subject><subject>Chemical properties</subject><subject>Clinical trials</subject><subject>Duchenne muscular dystrophy</subject><subject>Duchenne's muscular dystrophy</subject><subject>Dystrophy</subject><subject>eteplirsen</subject><subject>Ethics</subject><subject>Gene expression</subject><subject>Gene sequencing</subject><subject>Medical research</subject><subject>Muscles</subject><subject>Muscular dystrophy</subject><subject>Neuromuscular diseases</subject><subject>Nucleic acids</subject><subject>nusinersen</subject><subject>Oligonucleotides</subject><subject>Pharmacology</subject><subject>Polyneuropathy</subject><subject>Proteins</subject><subject>Ribonucleic acid</subject><subject>RNA</subject><subject>Spinal muscular atrophy</subject><subject>Toxicity</subject><issn>0148-639X</issn><issn>1097-4598</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2018</creationdate><recordtype>article</recordtype><recordid>eNp1kE1Lw0AQhhdRbK0e_AMS8NRD2p1Nssl6EErxC1o8aMHbskkmZWua1N2kpf_eranePA0DD8-88xJyDXQElLLxurUjximFE9IHKmI_jERySvoUwsTngfjokQtrV9QRCY_PSY8JoBFEQZ_cz9WnrpaexcqiVxeeqhp9XEq9rKs2K7FudI72zpt4lTJGNXqLnsGtxt0lOStUafHqOAdk8fjwPn32Z69PL9PJzM9CEOBDDixNizjNecYzFoYsDhRPmGIuBGMYsohB4aLzRKgghbSIBIZJynmGcVwEwYDcdt6Nqb9atI1c1a2p3EnJDl_FApIDNeyozNTWGizkxui1MnsJVB6akq4p-dOUY2-OxjZdY_5H_lbjgHEH7HSJ-_9Ncr5465Tf7-NxBA</recordid><startdate>201803</startdate><enddate>201803</enddate><creator>Goyal, Neelam</creator><creator>Narayanaswami, Pushpa</creator><general>Wiley Subscription Services, Inc</general><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7T5</scope><scope>7TK</scope><scope>7TM</scope><scope>7TS</scope><scope>7U7</scope><scope>7U9</scope><scope>C1K</scope><scope>H94</scope><scope>K9.</scope><scope>NAPCQ</scope></search><sort><creationdate>201803</creationdate><title>Making sense of antisense oligonucleotides: A narrative review</title><author>Goyal, Neelam ; Narayanaswami, Pushpa</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c4191-1d12bbf7bd6c6c244273a682a215322e42521f109689a3b1bf59e48b66ce77f33</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2018</creationdate><topic>Amyloid</topic><topic>Amyotrophic lateral sclerosis</topic><topic>Antisense oligonucleotides</topic><topic>Atrophy</topic><topic>Chemical properties</topic><topic>Clinical trials</topic><topic>Duchenne muscular dystrophy</topic><topic>Duchenne's muscular dystrophy</topic><topic>Dystrophy</topic><topic>eteplirsen</topic><topic>Ethics</topic><topic>Gene expression</topic><topic>Gene sequencing</topic><topic>Medical research</topic><topic>Muscles</topic><topic>Muscular dystrophy</topic><topic>Neuromuscular diseases</topic><topic>Nucleic acids</topic><topic>nusinersen</topic><topic>Oligonucleotides</topic><topic>Pharmacology</topic><topic>Polyneuropathy</topic><topic>Proteins</topic><topic>Ribonucleic acid</topic><topic>RNA</topic><topic>Spinal muscular atrophy</topic><topic>Toxicity</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Goyal, Neelam</creatorcontrib><creatorcontrib>Narayanaswami, Pushpa</creatorcontrib><collection>PubMed</collection><collection>CrossRef</collection><collection>Immunology Abstracts</collection><collection>Neurosciences Abstracts</collection><collection>Nucleic Acids Abstracts</collection><collection>Physical Education Index</collection><collection>Toxicology Abstracts</collection><collection>Virology and AIDS Abstracts</collection><collection>Environmental Sciences and Pollution Management</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Nursing & Allied Health Premium</collection><jtitle>Muscle & nerve</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Goyal, Neelam</au><au>Narayanaswami, Pushpa</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Making sense of antisense oligonucleotides: A narrative review</atitle><jtitle>Muscle & nerve</jtitle><addtitle>Muscle Nerve</addtitle><date>2018-03</date><risdate>2018</risdate><volume>57</volume><issue>3</issue><spage>356</spage><epage>370</epage><pages>356-370</pages><issn>0148-639X</issn><eissn>1097-4598</eissn><abstract>ABSTRACT
Synthetic nucleic acid sequences that bind to ribonucleic acid (RNA) through Watson‐Crick base pairing are known as antisense oligonucleotides (ASOs) because they are complementary to “sense strand” nucleic acids. ASOs bind to selected sequences of RNA and regulate the expression of genes by several mechanisms depending on their chemical properties and targets. They can be used to restore deficient protein expression, reduce the expression of a toxic protein, modify functional effects of proteins, or reduce toxicity of mutant proteins. Two ASOs were approved by the U.S. Food and Drug Administration in 2016: eteplirsen for Duchenne muscular dystrophy and nusinersen for spinal muscular atrophy. Clinical trials in amyotrophic lateral sclerosis and familial amyloid polyneuropathy are ongoing. We review the chemistry, pharmacology, and mechanisms of action of ASOs, preclinical data, and clinical trials in neuromuscular diseases and discuss some ethical, regulatory, and policy considerations in the clinical development and use of ASOs. Muscle Nerve 57: 356–370, 2018</abstract><cop>United States</cop><pub>Wiley Subscription Services, Inc</pub><pmid>29105153</pmid><doi>10.1002/mus.26001</doi><tpages>15</tpages></addata></record> |
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| ispartof | Muscle & nerve, 2018-03, Vol.57 (3), p.356-370 |
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| language | eng |
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| source | Wiley Online Library Journals Frontfile Complete |
| subjects | Amyloid Amyotrophic lateral sclerosis Antisense oligonucleotides Atrophy Chemical properties Clinical trials Duchenne muscular dystrophy Duchenne's muscular dystrophy Dystrophy eteplirsen Ethics Gene expression Gene sequencing Medical research Muscles Muscular dystrophy Neuromuscular diseases Nucleic acids nusinersen Oligonucleotides Pharmacology Polyneuropathy Proteins Ribonucleic acid RNA Spinal muscular atrophy Toxicity |
| title | Making sense of antisense oligonucleotides: A narrative review |
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