{"title":"Pre-mRNA splicing as a target for antisense oligonucleotides","authors":"Ryszard Kole, Ram R. Shukla, Saghir Akhtar","doi":"10.1016/0169-409X(91)90021-4","DOIUrl":null,"url":null,"abstract":"<div><div>Splicing is one of the major post-transcriptional modifications a eukaryotic mRNA precursor (pre-mRNA) has to undergo to yield the mature mRNA. During pre-mRNA splicing the non-coding sequences (introns) of the precursor are removed and coding sequences (exons) are joined. This process takes place within a complex called a spliceosome and requires the presence of a number of splicing factors such as small nuclear ribonucleoprotein particles (snRNPs). Oligonucleotides containing sequences complementary (antisense) to unique sequences within the pre-mRNA can be used to modify splicing and, thus, gene expression. Likewise, snRNPs provide another important target for using antisense oligonucleotides as investigative tools to further study the mechanism of splicing. This article reviews the available literature on the use of antisense oligonucleotides targeted against pre-mRNA and those targeted against small nuclear ribonucleoprotein particles (snRNPs) within the spliceosomal complex.</div></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":"6 3","pages":"Pages 271-286"},"PeriodicalIF":17.6000,"publicationDate":"1991-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Advanced drug delivery reviews","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/0169409X91900214","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2002/11/20 0:00:00","PubModel":"Epub","JCR":"Q1","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
引用次数: 0
Abstract
Splicing is one of the major post-transcriptional modifications a eukaryotic mRNA precursor (pre-mRNA) has to undergo to yield the mature mRNA. During pre-mRNA splicing the non-coding sequences (introns) of the precursor are removed and coding sequences (exons) are joined. This process takes place within a complex called a spliceosome and requires the presence of a number of splicing factors such as small nuclear ribonucleoprotein particles (snRNPs). Oligonucleotides containing sequences complementary (antisense) to unique sequences within the pre-mRNA can be used to modify splicing and, thus, gene expression. Likewise, snRNPs provide another important target for using antisense oligonucleotides as investigative tools to further study the mechanism of splicing. This article reviews the available literature on the use of antisense oligonucleotides targeted against pre-mRNA and those targeted against small nuclear ribonucleoprotein particles (snRNPs) within the spliceosomal complex.
期刊介绍：
The aim of the Journal is to provide a forum for the critical analysis of advanced drug and gene delivery systems and their applications in human and veterinary medicine. The Journal has a broad scope, covering the key issues for effective drug and gene delivery, from administration to site-specific delivery.
In general, the Journal publishes review articles in a Theme Issue format. Each Theme Issue provides a comprehensive and critical examination of current and emerging research on the design and development of advanced drug and gene delivery systems and their application to experimental and clinical therapeutics. The goal is to illustrate the pivotal role of a multidisciplinary approach to modern drug delivery, encompassing the application of sound biological and physicochemical principles to the engineering of drug delivery systems to meet the therapeutic need at hand. Importantly the Editorial Team of ADDR asks that the authors effectively window the extensive volume of literature, pick the important contributions and explain their importance, produce a forward looking identification of the challenges facing the field and produce a Conclusions section with expert recommendations to address the issues.
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