当前位置： SCI文献检索 > NUCLEIC ACIDS RESEARCH期刊下所有文献 > Short antisense-locked nucleic acids (all-LNAs) correct alternative splicing abnormalities in myotonic dystrophy.

Short antisense-locked nucleic acids (all-LNAs) correct alternative splicing abnormalities in myotonic dystrophy.

Abstract：

:Myotonic dystrophy type 1 (DM1) is an autosomal dominant multisystemic disorder caused by expansion of CTG triplet repeats in 3'-untranslated region of DMPK gene. The pathomechanism of DM1 is driven by accumulation of toxic transcripts containing expanded CUG repeats (CUG(exp)) in nuclear foci which sequester several factors regulating RNA metabolism, such as Muscleblind-like proteins (MBNLs). In this work, we utilized very short chemically modified antisense oligonucleotides composed exclusively of locked nucleic acids (all-LNAs) complementary to CUG repeats, as potential therapeutic agents against DM1. Our in vitro data demonstrated that very short, 8- or 10-unit all-LNAs effectively bound the CUG repeat RNA and prevented the formation of CUG(exp)/MBNL complexes. In proliferating DM1 cells as well as in skeletal muscles of DM1 mouse model the all-LNAs induced the reduction of the number and size of CUG(exp) foci and corrected MBNL-sensitive alternative splicing defects with high efficacy and specificity. The all-LNAs had low impact on the cellular level of CUG(exp)-containing transcripts and did not affect the expression of other transcripts with short CUG repeats. Our data strongly indicate that short all-LNAs complementary to CUG repeats are a promising therapeutic tool against DM1.

journal_name

Nucleic Acids Res

journal_title

Nucleic acids research

authors

Wojtkowiak-Szlachcic A,Taylor K,Stepniak-Konieczna E,Sznajder LJ,Mykowska A,Sroka J,Thornton CA,Sobczak K

doi

10.1093/nar/gkv163

subject

Has Abstract

pub_date

2015-03-31 00:00:00

pages

3318-31

issue

6

eissn

0305-1048

issn

1362-4962

pii

gkv163

journal_volume

43

pub_type

杂志文章

相关文献

NUCLEIC ACIDS RESEARCH文献大全
  • Structure and DNA binding activity of the mouse condensin hinge domain highlight common and diverse features of SMC proteins.

    abstract：:Structural Maintenance of Chromosomes (SMC) proteins are vital for a wide range of processes including chromosome structure and dynamics, gene regulation and DNA repair. Eukaryotes have three SMC complexes, consisting of heterodimeric pairs of six different SMC proteins along with several specific regulatory subunits....

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkq038

    authors： Griese JJ,Witte G,Hopfner KP

    更新日期：2010-06-01 00:00:00

  • Air2p is critical for the assembly and RNA-binding of the TRAMP complex and the KOW domain of Mtr4p is crucial for exosome activation.

    abstract：:Trf4/5p-Air1/2p-Mtr4p polyadenylation complex (TRAMP) is an essential component of nuclear RNA surveillance in yeast. It recognizes a variety of nuclear transcripts produced by all three RNA polymerases, adds short poly(A) tails to aberrant or unstable RNAs and activates the exosome for their degradation. Despite the ...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gks223

    authors： Holub P,Lalakova J,Cerna H,Pasulka J,Sarazova M,Hrazdilova K,Arce MS,Hobor F,Stefl R,Vanacova S

    更新日期：2012-07-01 00:00:00

  • Discovery and characterization of a thermostable bacteriophage RNA ligase homologous to T4 RNA ligase 1.

    abstract：:Thermophilic viruses represent a novel source of genetic material and enzymes with great potential for use in biotechnology. We have isolated a number of thermophilic viruses from geothermal areas in Iceland, and by combining high throughput genome sequencing and state of the art bioinformatics we have identified a nu...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkg914

    authors： Blondal T,Hjorleifsdottir SH,Fridjonsson OF,Aevarsson A,Skirnisdottir S,Hermannsdottir AG,Hreggvidsson GO,Smith AV,Kristjansson JK

    更新日期：2003-12-15 00:00:00

  • Analysis of pooled DNA samples on high density arrays without prior knowledge of differential hybridization rates.

    abstract：:Array based DNA pooling techniques facilitate genome-wide scale genotyping of large samples. We describe a structured analysis method for pooled data using internal replication information in large scale genotyping sets. The method takes advantage of information from single nucleotide polymorphisms (SNPs) typed in par...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkl136

    authors： Macgregor S,Visscher PM,Montgomery G

    更新日期：2006-04-20 00:00:00

  • CHOP gene expression in response to endoplasmic-reticular stress requires NFY interaction with different domains of a conserved DNA-binding element.

    abstract：:The transcription factor CHOP/GADD153 gene is induced by cellular stress and is involved in mediating apoptosis. We report the identification of a conserved region in the promoter of the CHOP gene responsible for its inducibility by endoplasmic reticulum (ER) stress. Deletion mutants of the human CHOP promoter identif...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/28.24.4987

    authors： Ubeda M,Habener JF

    更新日期：2000-12-15 00:00:00

  • Ribosomal protein eL24, involved in two intersubunit bridges, stimulates translation initiation and elongation.

    abstract：:Interactions between subunits in the Saccharomyces cerevisiae ribosome are mediated by universal and eukaryote-specific intersubunit bridges. Universal bridges are positioned close to the ribosomal functional centers, while eukaryote-specific bridges are mainly located on the periphery of the ribosome. Two bridges, eB...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gky1083

    authors： Kisly I,Remme J,Tamm T

    更新日期：2019-01-10 00:00:00

  • A novel regulatory circuit in base excision repair involving AP endonuclease 1, Creb1 and DNA polymerase beta.

    abstract：:DNA repair is required to maintain genome stability in stem cells and early embryos. At critical junctures, oxidative damage to DNA requires the base excision repair (BER) pathway. Since early zebrafish embryos lack the major polymerase in BER, DNA polymerase ß, repair proceeds via replicative polymerases, even though...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkq1142

    authors： Pei DS,Yang XJ,Liu W,Guikema JE,Schrader CE,Strauss PR

    更新日期：2011-04-01 00:00:00

  • Tumor-associated mutations of rat mitochondrial transfer RNA genes.

    abstract：:Mitochondrial DNA is a sensitive target of chemical carcinogens (Backer and Weinstein (1980) Science 209, 297-299), suggesting that mutations of the mitochondrial genome occur in tumor cells. We examined this point by comparing mitochondrial DNA sequences in four rat tumors with those of normal rat liver. Some novel m...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/11.6.1635

    authors： Taira M,Yoshida E,Kobayashi M,Yaginuma K,Koike K

    更新日期：1983-03-25 00:00:00

  • Activation of the cryptic DNA binding function of mutant forms of p53.

    abstract：:Wild type p53 assembles into a latent multiprotein complex which can be activated for sequence-specific DNA binding in vitro by proteins targeting the carboxy-terminal domain. Using an optimized system coupling the post-translational modification of wild type p53 to activation of sequence specific DNA binding, we exam...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/21.14.3167

    authors： Hupp TR,Meek DW,Midgley CA,Lane DP

    更新日期：1993-07-11 00:00:00

  • Noncoding RNAs database (ncRNAdb).

    abstract：:The noncoding RNA database (ncRNAdb) was created as a source of information on RNA molecules, which do not possess protein-coding capacity. It is now widely accepted that, in addition to constitutively expressed, housekeeping or infrastructural RNAs, there is a wide variety of RNAs participating in mechanisms involved...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkl994

    authors： Szymanski M,Erdmann VA,Barciszewski J

    更新日期：2007-01-01 00:00:00

  • Structural basis for transcription inhibition by E. coli SspA.

    abstract：:Stringent starvation protein A (SspA) is an RNA polymerase (RNAP)-associated protein involved in nucleotide metabolism, acid tolerance and virulence of bacteria. Despite extensive biochemical and genetic analyses, the precise regulatory role of SspA in transcription is still unknown, in part, because of a lack of stru...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkaa672

    authors： Wang F,Shi J,He D,Tong B,Zhang C,Wen A,Zhang Y,Feng Y,Lin W

    更新日期：2020-09-25 00:00:00

  • Mutational analysis of the reverse transcriptase and ribonuclease H domains of the human foamy virus.

    abstract：:Human foamy or spuma virus (HFV) codes for a distinct set of pol gen products. To determine the minimal requirements for the HFV enzymatic activities, defined residues of the reverse transcriptase (RT) and ribo-nuclease H (RNase H) domain of the HFV pol gene were mutated by site-specific PCR mutagenesis. The mutant ge...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/23.14.2621

    authors： Kögel D,Aboud M,Flügel RM

    更新日期：1995-07-25 00:00:00

  • The PARP promoter of Trypanosoma brucei is developmentally regulated in a chromosomal context.

    abstract：:African trypanosomes are extracellular protozoan parasites that are transmitted from one mammalian host to the next by tsetse flies. Bloodstream forms express variant surface glycoprotein (VSG); the tsetse fly (procyclic) forms express instead the procyclic acidic repetitive protein (PARP). PARP mRNA is abundant in pr...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/24.7.1202

    authors： Biebinger S,Rettenmaier S,Flaspohler J,Hartmann C,Peña-Diaz J,Wirtz LE,Hotz HR,Barry JD,Clayton C

    更新日期：1996-04-01 00:00:00

  • Base excision repair processing of abasic site/single-strand break lesions within clustered damage sites associated with XRCC1 deficiency.

    abstract：:Ionizing radiation induces clustered DNA damage, which presents a challenge to the cellular repair machinery. The repair efficiency of a single-strand break (SSB) is approximately 4x less than that for repair of an abasic (AP) site when in a bistranded cluster containing 8-oxoG. To explore whether this difference in r...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkm947

    authors： Mourgues S,Lomax ME,O'Neill P

    更新日期：2007-01-01 00:00:00

  • Maturation of a hypermodified nucleoside in transfer RNA.

    abstract：:E. coli C6 rel- met- cys- was cultured in a fully supplemented medium and in media lacking cysteine or methionine. tRNA isolated from the three cultures containted, respectively, a normal complement of modified nucleosides; a deficiency in thiolated nucleosides and a deficiency in methylated nucleosides. Both sulfur-d...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/2.5.691

    authors： Agris PF,Armstrong DJ,Schäfer KP,Söll D

    更新日期：1975-05-01 00:00:00

  • ASD: a comprehensive database of allosteric proteins and modulators.

    abstract：:Allostery is the most direct, rapid and efficient way of regulating protein function, ranging from the control of metabolic mechanisms to signal-transduction pathways. However, an enormous amount of unsystematic allostery information has deterred scientists who could benefit from this field. Here, we present the AlloS...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkq1022

    authors： Huang Z,Zhu L,Cao Y,Wu G,Liu X,Chen Y,Wang Q,Shi T,Zhao Y,Wang Y,Li W,Li Y,Chen H,Chen G,Zhang J

    更新日期：2011-01-01 00:00:00

  • EMdeCODE: a novel algorithm capable of reading words of epigenetic code to predict enhancers and retroviral integration sites and to identify H3R2me1 as a distinctive mark of coding versus non-coding genes.

    abstract：:Existence of some extra-genetic (epigenetic) codes has been postulated since the discovery of the primary genetic code. Evident effects of histone post-translational modifications or DNA methylation over the efficiency and the regulation of DNA processes are supporting this postulation. EMdeCODE is an original algorit...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gks1214

    authors： Santoni FA

    更新日期：2013-02-01 00:00:00

  • Error-free and error-prone lesion bypass by human DNA polymerase kappa in vitro.

    abstract：:Error-free lesion bypass and error-prone lesion bypass are important cellular responses to DNA damage during replication, both of which require a DNA polymerase (Pol). To identify lesion bypass DNA polymerases, we have purified human Polkappa encoded by the DINB1 gene and examined its response to damaged DNA templates...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/28.21.4138

    authors： Zhang Y,Yuan F,Wu X,Wang M,Rechkoblit O,Taylor JS,Geacintov NE,Wang Z

    更新日期：2000-11-01 00:00:00

  • Isolation of polymorphic DNA segments from human chromosome 21.

    abstract：:A somatic cell hybrid line containing only human chromosome 21 on a mouse background has been used as the source of DNA for construction of a recombinant phage library. Individual phages containing human inserts have been identified. Repeat-free human DNA subclones have been prepared and used to screen for restriction...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/13.17.6075

    authors： Watkins PC,Tanzi RE,Gibbons KT,Tricoli JV,Landes G,Eddy R,Shows TB,Gusella JF

    更新日期：1985-09-11 00:00:00

  • EBI metagenomics--a new resource for the analysis and archiving of metagenomic data.

    abstract：:Metagenomics is a relatively recently established but rapidly expanding field that uses high-throughput next-generation sequencing technologies to characterize the microbial communities inhabiting different ecosystems (including oceans, lakes, soil, tundra, plants and body sites). Metagenomics brings with it a number ...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkt961

    authors： Hunter S,Corbett M,Denise H,Fraser M,Gonzalez-Beltran A,Hunter C,Jones P,Leinonen R,McAnulla C,Maguire E,Maslen J,Mitchell A,Nuka G,Oisel A,Pesseat S,Radhakrishnan R,Rocca-Serra P,Scheremetjew M,Sterk P,Vaughan D,

    更新日期：2014-01-01 00:00:00

  • Identification of family-determining residues in PHD fingers.

    abstract：:Histone modifications are fundamental to chromatin structure and transcriptional regulation, and are recognized by a limited number of protein folds. Among these folds are PHD fingers, which are present in most chromatin modification complexes. To date, about 15 PHD finger domains have been structurally characterized,...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkq947

    authors： Slama P,Geman D

    更新日期：2011-03-01 00:00:00

  • Azidopolynucleotides as photoaffinity reagents.

    abstract：:Polynucleotides containing adenosine and 8-azidoadenosine or inosine and 8-azidoinosine residues have been prepared from mixtures of nucleoside diphosphates using polynucleotide phosphorylase from Escherichia coli. These copolymers can form complexes with polyuridylic or polycytidylic acids respectively. Single strand...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/8.7.1675

    authors： Cartwright IL,Hutchinson DW

    更新日期：1980-04-11 00:00:00

  • A thermostable d-polymerase for mirror-image PCR.

    abstract：:Biological evolution resulted in a homochiral world in which nucleic acids consist exclusively of d-nucleotides and proteins made by ribosomal translation of l-amino acids. From the perspective of synthetic biology, however, particularly anabolic enzymes that could build the mirror-image counterparts of biological mac...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkx079

    authors： Pech A,Achenbach J,Jahnz M,Schülzchen S,Jarosch F,Bordusa F,Klussmann S

    更新日期：2017-04-20 00:00:00

  • Synthesis and properties of DNA-PNA chimeric oligomers.

    abstract：:Adenine, thymine and cytosine PNA monomers have been prepared using 3-amino-1,2-propanediol as a starting material. The benzoyl group was used to protect the exocyclic amines of the heterocyclic bases of A and C PNA monomers and the backbone primary amine was protected with the monomethoxytrityl group. The thymine and...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/24.17.3357

    authors： Finn PJ,Gibson NJ,Fallon R,Hamilton A,Brown T

    更新日期：1996-09-01 00:00:00

  • Simple, multiplexed, PCR-based barcoding of DNA enables sensitive mutation detection in liquid biopsies using sequencing.

    abstract：:Detection of cell-free DNA in liquid biopsies offers great potential for use in non-invasive prenatal testing and as a cancer biomarker. Fetal and tumor DNA fractions however can be extremely low in these samples and ultra-sensitive methods are required for their detection. Here, we report an extremely simple and fast...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkw224

    authors： Ståhlberg A,Krzyzanowski PM,Jackson JB,Egyud M,Stein L,Godfrey TE

    更新日期：2016-06-20 00:00:00

  • Human protein reference database--2006 update.

    abstract：:Human Protein Reference Database (HPRD) (http://www.hprd.org) was developed to serve as a comprehensive collection of protein features, post-translational modifications (PTMs) and protein-protein interactions. Since the original report, this database has increased to >20 000 proteins entries and has become the largest...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkj141

    authors： Mishra GR,Suresh M,Kumaran K,Kannabiran N,Suresh S,Bala P,Shivakumar K,Anuradha N,Reddy R,Raghavan TM,Menon S,Hanumanthu G,Gupta M,Upendran S,Gupta S,Mahesh M,Jacob B,Mathew P,Chatterjee P,Arun KS,Sharma S,Chand

    更新日期：2006-01-01 00:00:00

  • PSSweb: protein structural statistics web server.

    abstract：:With the increasing number of protein structures available, there is a need for tools capable of automating the comparison of ensembles of structures, a common requirement in structural biology and bioinformatics. PSSweb is a web server for protein structural statistics. It takes as input an ensemble of PDB files of p...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkw332

    authors： Gaillard T,Stote RH,Dejaegere A

    更新日期：2016-07-08 00:00:00

  • Analytical approaches to RNA profiling data for the identification of genes enriched in specific cells.

    abstract：:We have recently developed a novel method for the affinity purification of the complete suite of translating mRNA from genetically labeled cell populations. This method permits comprehensive quantitative comparisons of the genes employed by each specific cell type. We provide a detailed description of tools for analys...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkq130

    authors： Dougherty JD,Schmidt EF,Nakajima M,Heintz N

    更新日期：2010-07-01 00:00:00

  • Optogenetic control of Neisseria meningitidis Cas9 genome editing using an engineered, light-switchable anti-CRISPR protein.

    abstract：:Optogenetic control of CRISPR-Cas9 systems has significantly improved our ability to perform genome perturbations in living cells with high precision in time and space. As new Cas orthologues with advantageous properties are rapidly being discovered and engineered, the need for straightforward strategies to control th...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkaa1198

    authors： Hoffmann MD,Mathony J,Upmeier Zu Belzen J,Harteveld Z,Aschenbrenner S,Stengl C,Grimm D,Correia BE,Eils R,Niopek D

    更新日期：2020-12-16 00:00:00

  • Genome sequence of Shigella flexneri 2a: insights into pathogenicity through comparison with genomes of Escherichia coli K12 and O157.

    abstract：:We have sequenced the genome of Shigella flexneri serotype 2a, the most prevalent species and serotype that causes bacillary dysentery or shigellosis in man. The whole genome is composed of a 4 607 203 bp chromosome and a 221 618 bp virulence plasmid, designated pCP301. While the plasmid shows minor divergence from th...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkf566

    authors： Jin Q,Yuan Z,Xu J,Wang Y,Shen Y,Lu W,Wang J,Liu H,Yang J,Yang F,Zhang X,Zhang J,Yang G,Wu H,Qu D,Dong J,Sun L,Xue Y,Zhao A,Gao Y,Zhu J,Kan B,Ding K,Chen S,Cheng H,Yao Z,He B,Chen R,Ma D,Qiang B,

    更新日期：2002-10-15 00:00:00