当前位置： SCI文献检索 > NUCLEIC ACIDS RESEARCH期刊下所有文献 > Targeted correction of a thalassemia-associated beta-globin mutation induced by pseudo-complementary peptide nucleic acids.

Targeted correction of a thalassemia-associated beta-globin mutation induced by pseudo-complementary peptide nucleic acids.

Abstract：

:Beta-thalassemia is a genetic disorder caused by mutations in the beta-globin gene. Triplex-forming oligonucleotides and triplex-forming peptide nucleic acids (PNAs) have been shown to stimulate recombination in mammalian cells via site-specific binding and creation of altered helical structures that provoke DNA repair. However, the use of these molecules for gene targeting requires homopurine tracts to facilitate triple helix formation. Alternatively, to achieve binding to mixed-sequence target sites for the induced gene correction, we have used pseudo-complementary PNAs (pcPNAs). Due to steric hindrance, pcPNAs are unable to form pcPNA-pcPNA duplexes but can bind to complementary DNA sequences via double duplex-invasion complexes. We demonstrate here that pcPNAs, when co-transfected with donor DNA fragments, can promote single base pair modification at the start of the second intron of the beta-globin gene. This was detected by the restoration of proper splicing of transcripts produced from a green fluorescent protein-beta globin fusion gene. We also demonstrate that pcPNAs are effective in stimulating recombination in human fibroblast cells in a manner dependent on the nucleotide excision repair factor, XPA. These results suggest that pcPNAs can be effective tools to induce heritable, site-specific modification of disease-related genes in human cells without purine sequence restriction.

journal_name

Nucleic Acids Res

journal_title

Nucleic acids research

authors

Lonkar P,Kim KH,Kuan JY,Chin JY,Rogers FA,Knauert MP,Kole R,Nielsen PE,Glazer PM

doi

10.1093/nar/gkp217

subject

Has Abstract

pub_date

2009-06-01 00:00:00

pages

3635-44

issue

11

eissn

0305-1048

issn

1362-4962

pii

gkp217

journal_volume

37

pub_type

杂志文章

相关文献

NUCLEIC ACIDS RESEARCH文献大全
  • Multiple RNA-RNA tertiary interactions are dispensable for formation of a functional U2/U6 RNA catalytic core in the spliceosome.

    abstract：:The active 3D conformation of the spliceosome's catalytic U2/U6 RNA core is stabilised by a network of secondary and tertiary RNA interactions, but also depends on spliceosomal proteins for its formation. To determine the contribution towards splicing of specific RNA secondary and tertiary interactions in the U2/U6 RN...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gky966

    authors： Bao P,Boon KL,Will CL,Hartmuth K,Lührmann R

    更新日期：2018-12-14 00:00:00

  • The proteasomal ATPase complex is required for stress-induced transcription in yeast.

    abstract：:Sug1 and Sug2 are two of six ATPases in the 19S regulatory particle of the 26S proteasome. We have shown previously that these proteins play a non-proteolytic role in the transcription of the GAL genes in yeast. In this study, we probe the requirement for these factors in stress-induced transcription in yeast. It is k...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkl012

    authors： Sulahian R,Sikder D,Johnston SA,Kodadek T

    更新日期：2006-03-03 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

  • Nucleotide sequences of two serine tRNAs with a GGA anticodon: the structure-function relationships in the serine family of E. coli tRNAs.

    abstract：:We have determined the nucleotide sequence of the major species of E. coli tRNASer and of a minor species having the same GGA anticodon. These two tRNAs should recognize the UCC and UCU codons, the most widely used codons for serine in the highly expressed genes of E. coli. The two sequences differ in only one positio...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/13.15.5697

    authors： Grosjean H,Nicoghosian K,Haumont E,Söll D,Cedergren R

    更新日期：1985-08-12 00:00:00

  • Complete sequence of the amphioxus (Branchiostoma lanceolatum) mitochondrial genome: relations to vertebrates.

    abstract：:The complete nucleotide sequence of the mitochondrial DNA of the amphioxus Branchiostoma lanceolatum has been determined. This mitochondrial genome is small (15 076 bp) because of the short size of the two rRNA genes and the tRNA genes. In addition, this genome contains a very short non-coding region (57 bp) with no s...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/26.13.3279

    authors： Spruyt N,Delarbre C,Gachelin G,Laudet V

    更新日期：1998-07-01 00:00:00

  • SPINE 2: a system for collaborative structural proteomics within a federated database framework.

    abstract：:We present version 2 of the SPINE system for structural proteomics. SPINE is available over the web at http://nesg.org. It serves as the central hub for the Northeast Structural Genomics Consortium, allowing collaborative structural proteomics to be carried out in a distributed fashion. The core of SPINE is a laborato...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkg397

    authors： Goh CS,Lan N,Echols N,Douglas SM,Milburn D,Bertone P,Xiao R,Ma LC,Zheng D,Wunderlich Z,Acton T,Montelione GT,Gerstein M

    更新日期：2003-06-01 00:00:00

  • Chicken erythrocyte polynucleosomes which are soluble at physiological ionic strength and contain linker histones are highly enriched in beta-globin gene sequences.

    abstract：:Mature chicken erythrocyte polynucleosomes which are soluble at physiological ionic strength are enriched in beta-globin DNA sequences. Vitellogenin chromatin, which is not expressed in this tissue, is found in aggregation prone, salt insoluble chromatin. There is a direct correlation between the size of soluble fragm...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/15.3.1081

    authors： Ridsdale JA,Davie JR

    更新日期：1987-02-11 00:00:00

  • mrsFAST-Ultra: a compact, SNP-aware mapper for high performance sequencing applications.

    abstract：:High throughput sequencing (HTS) platforms generate unprecedented amounts of data that introduce challenges for processing and downstream analysis. While tools that report the 'best' mapping location of each read provide a fast way to process HTS data, they are not suitable for many types of downstream analysis such a...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gku370

    authors： Hach F,Sarrafi I,Hormozdiari F,Alkan C,Eichler EE,Sahinalp SC

    更新日期：2014-07-01 00:00:00

  • The European Nucleotide Archive in 2018.

    abstract：:The European Nucleotide Archive (ENA; https://www.ebi.ac.uk/ena), provided from EMBL-EBI, has for more than three decades been responsible for archiving the world's public sequencing data and presenting this important resource to the scientific community to support and accelerate the global research effort. Here, we o...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gky1078

    authors： Harrison PW,Alako B,Amid C,Cerdeño-Tárraga A,Cleland I,Holt S,Hussein A,Jayathilaka S,Kay S,Keane T,Leinonen R,Liu X,Martínez-Villacorta J,Milano A,Pakseresht N,Rajan J,Reddy K,Richards E,Rosello M,Silvester N,Smi

    更新日期：2019-01-08 00:00:00

  • DNA topology of the ordered chromatin domain 5' to the human c-myc gene.

    abstract：:DNA restriction fragments located 5' to the human c-myc gene display anomalous electrophoretic mobility on polyacrylamide gels. Computer modeling of the c-myc flanking DNA suggests that the slow-moving DNA fragments spanning nucleotides -1690 to -1054 (relative to c-myc promoter P1) and -718 to -452 form large left ha...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/17.7.2819

    authors： Kumar S,Leffak M

    更新日期：1989-04-11 00:00:00

  • Evidence that RNA editing modulates splice site selection in the 5-HT2C receptor gene.

    abstract：:Adenosine to inosine editing of mRNA from the human 5-HT2C receptor gene (HTR2C) occurs at five exonic positions (A-E) in a stable stem-loop that includes the normal 5' splice site of intron 5 and is flanked by two alternative splice sites. Using in vitro editing, we identified a novel editing site (F) located in the ...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkh536

    authors： Flomen R,Knight J,Sham P,Kerwin R,Makoff A

    更新日期：2004-04-15 00:00:00

  • Identification of endoribonuclease specific cleavage positions reveals novel targets of RNase III in Streptococcus pyogenes.

    abstract：:A better understanding of transcriptional and post-transcriptional regulation of gene expression in bacteria relies on studying their transcriptome. RNA sequencing methods are used not only to assess RNA abundance but also the exact boundaries of primary and processed transcripts. Here, we developed a method, called i...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkw1316

    authors： Le Rhun A,Lécrivain AL,Reimegård J,Proux-Wéra E,Broglia L,Della Beffa C,Charpentier E

    更新日期：2017-03-17 00:00:00

  • Conserved elements containing NF-E2 and tandem GATA binding sites are required for erythroid-specific chromatin structure reorganization within the human beta-globin locus control region.

    abstract：:Proper expression of the genes of the human beta-globin gene locus requires the associated locus control region (LCR). Structurally, the LCR is defined by the presence of four domains of erythroid-specific chromatin structure. These domains, which have been characterized as DNase I hypersensitive sites (HSs), comprise...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/26.24.5684

    authors： Pomerantz O,Goodwin AJ,Joyce T,Lowrey CH

    更新日期：1998-12-15 00:00:00

  • A nucleobase-binding pocket in a viral RNA-dependent RNA polymerase contributes to elongation complex stability.

    abstract：:The enterovirus 71 (EV71) 3Dpol is an RNA-dependent RNA polymerase (RdRP) that plays the central role in the viral genome replication, and is an important target in antiviral studies. Here, we report a crystal structure of EV71 3Dpol elongation complex (EC) at 1.8 Å resolution. The structure reveals that the 5'-end gu...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkz1170

    authors： Shi W,Ye HQ,Deng CL,Li R,Zhang B,Gong P

    更新日期：2020-02-20 00:00:00

  • GeneMark: web software for gene finding in prokaryotes, eukaryotes and viruses.

    abstract：:The task of gene identification frequently confronting researchers working with both novel and well studied genomes can be conveniently and reliably solved with the help of the GeneMark web software (http://opal.biology.gatech.edu/GeneMark/). The website provides interfaces to the GeneMark family of programs designed ...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gki487

    authors： Besemer J,Borodovsky M

    更新日期：2005-07-01 00:00:00

  • Transmission of dynamic supercoiling in linear and multi-way branched DNAs and its regulation revealed by a fluorescent G-quadruplex torsion sensor.

    abstract：:DNA supercoiling is an important regulator of gene activity. The transmission of transcription-generated supercoiling wave along a DNA helix provides a way for a gene being transcribed to communicate with and regulate its neighboring genes. Currently, the dynamic behavior of supercoiling transmission remains unclear o...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gky534

    authors： Xia Y,Zheng KW,He YD,Liu HH,Wen CJ,Hao YH,Tan Z

    更新日期：2018-08-21 00:00:00

  • DNA analysis with multiplex microarray-enhanced PCR.

    abstract：:We have developed a highly sensitive method for DNA analysis on 3D gel element microarrays, a technique we call multiplex microarray-enhanced PCR (MME-PCR). Two amplification strategies are carried out simultaneously in the reaction chamber: on or within gel elements, and in bulk solution over the gel element array. M...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gnh184

    authors： Pemov A,Modi H,Chandler DP,Bavykin S

    更新日期：2005-01-20 00:00:00

  • Solution structure of a novel zinc finger motif in the SAP30 polypeptide of the Sin3 corepressor complex and its potential role in nucleic acid recognition.

    abstract：:Giant chromatin-modifying complexes regulate gene transcription in eukaryotes by acting on chromatin substrates and 'setting' the histone code. The histone deacetylase (HDAC)-associated mammalian Sin3 corepressor complex regulates a wide variety of genes involved in all aspects of cellular physiology. The recruitment ...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkp051

    authors： He Y,Imhoff R,Sahu A,Radhakrishnan I

    更新日期：2009-04-01 00:00:00

  • Multi-forms of human MTH1 polypeptides produced by alternative translation initiation and single nucleotide polymorphism.

    abstract：:The human MTH1 gene for 8-oxo-7,8-dihydrodeoxyguanosine triphosphatase, produces seven types (types 1, 2A, 2B, 3A, 3B, 4A and 4B) of mRNAs. The B-type mRNAs with exon 2b-2c segments have three additional in-frame AUGs in their 5' regions. We report here that these transcripts produce three forms of MTH1 polypeptides (...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/27.22.4335

    authors： Oda H,Taketomi A,Maruyama R,Itoh R,Nishioka K,Yakushiji H,Suzuki T,Sekiguchi M,Nakabeppu Y

    更新日期：1999-11-15 00:00:00

  • Isolation and characterization of the DNA fraction of rat liver chromatin which binds polylysine.

    abstract：:The structure of eukaryotic chromatin has been investigated by isolating and analyzing the "accessible" DNA fraction of rat liver chromatin. This DNA fraction has been isolated by titrating the chromatin with the protese-resistant D isomer of polylysine to bind the "accessible" DNA sites. After removal of chromosomal ...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/2.5.667

    authors： Arnold EA,Wahn U,Young KE

    更新日期：1975-05-01 00:00:00

  • An oligodeoxynucleotide affinity column for the isolation of sequence specific DNA binding proteins.

    abstract：:A nucleic acid affinity matrix containing a short oligodeoxynucleotide ligand has been prepared as an example of a material which can be used for the rapid and effective isolation of sequence specific DNA binding proteins. Two complementary oligodeoxynucleotides have been employed, one of which contains a small 5'-spa...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/16.21.10283

    authors： Blanks R,McLaughlin LW

    更新日期：1988-11-11 00:00:00

  • Validation of kinetics similarity in qPCR.

    abstract：:Quantitative real-time PCR (qPCR) is the method of choice for specific and sensitive quantification of nucleic acids. However, data validation is still a major issue, partially due to the complex effect of PCR inhibition on the results. If undetected PCR inhibition may severely impair the accuracy and sensitivity of r...

    journal_title：Nucleic acids research

    pub_type： 杂志文章,评审

    doi：10.1093/nar/gkr778

    authors： Bar T,Kubista M,Tichopad A

    更新日期：2012-02-01 00:00:00

  • Infectious TYMV RNA from cloned cDNA: effects in vitro and in vivo of point substitutions in the initiation codons of two extensively overlapping ORFs.

    abstract：:Full-length cDNA of the 6.3 kb turnip yellow mosaic virus (TYMV) genome was placed between a T7 promoter and a unique Hind III site. In vitro transcription of Hind III-linearized DNA of clone pTYMC yielded full-length RNA transcripts. In inoculations of Chinese cabbage protoplasts and plants, capped transcripts and vi...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/17.12.4675

    authors： Weiland JJ,Dreher TW

    更新日期：1989-06-26 00:00:00

  • Recent improvements to Binding MOAD: a resource for protein-ligand binding affinities and structures.

    abstract：:For over 10 years, Binding MOAD (Mother of All Databases; http://www.BindingMOAD.org) has been one of the largest resources for high-quality protein-ligand complexes and associated binding affinity data. Binding MOAD has grown at the rate of 1994 complexes per year, on average. Currently, it contains 23,269 complexes ...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gku1088

    authors： Ahmed A,Smith RD,Clark JJ,Dunbar JB Jr,Carlson HA

    更新日期：2015-01-01 00:00:00

  • Dual enhancer activities of the cyclic-AMP responsive element with cell type and promoter specificity.

    abstract：:The role of the cyclic-AMP (cAMP) responsive element (CRE) in eukaryotic gene transcription was investigated in several cell lines transfected by constructs containing the chloramphenicol acetyltransferase (CAT) gene linked to the three different promoters, simian virus (SV) 40, human c-Ha-ras-1, or chicken beta-actin...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/17.4.1521

    authors： Kanei-Ishii C,Ishii S

    更新日期：1989-02-25 00:00:00

  • H-DNA and Z-DNA in the mouse c-Ki-ras promoter.

    abstract：:The mouse c-Ki-ras protooncogene promoter contains a homopurine-homopyrimidine domain that exhibits S1 nuclease sensitivity in vitro. We have studied the structure of this DNA region in a supercoiled state using a number of chemical probes for non-B DNA conformations including diethyl pyrocarbonate, osmium tetroxide, ...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/19.23.6527

    authors： Pestov DG,Dayn A,Siyanova EYu,George DL,Mirkin SM

    更新日期：1991-12-11 00:00:00

  • A periodic pattern of mRNA secondary structure created by the genetic code.

    abstract：:Single-stranded mRNA molecules form secondary structures through complementary self-interactions. Several hypotheses have been proposed on the relationship between the nucleotide sequence, encoded amino acid sequence and mRNA secondary structure. We performed the first transcriptome-wide in silico analysis of the huma...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkl287

    authors： Shabalina SA,Ogurtsov AY,Spiridonov NA

    更新日期：2006-05-08 00:00:00

  • G-quadruplexes as novel cis-elements controlling transcription during embryonic development.

    abstract：:G-quadruplexes are dynamic structures folded in G-rich single-stranded DNA regions. These structures have been recognized as a potential nucleic acid based mechanism for regulating multiple cellular processes such as replication, transcription and genomic maintenance. So far, their transcriptional role in vivo during ...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gkw011

    authors： David AP,Margarit E,Domizi P,Banchio C,Armas P,Calcaterra NB

    更新日期：2016-05-19 00:00:00

  • OrysPSSP: a comparative platform for small secreted proteins from rice and other plants.

    abstract：:Plants have large diverse families of small secreted proteins (SSPs) that play critical roles in the processes of development, differentiation, defense, flowering, stress response, symbiosis, etc. Oryza sativa is one of the major crops worldwide and an excellent model for monocotyledonous plants. However, there had no...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gks1090

    authors： Pan B,Sheng J,Sun W,Zhao Y,Hao P,Li X

    更新日期：2013-01-01 00:00:00

  • ProtoNet 4.0: a hierarchical classification of one million protein sequences.

    abstract：:ProtoNet is an automatic hierarchical classification of the protein sequence space. In 2004, the ProtoNet (version 4.0) presents the analysis of over one million proteins merged from SwissProt and TrEMBL databases. In addition to rich visualization and analysis tools to navigate the clustering hierarchy, we incorporat...

    journal_title：Nucleic acids research

    pub_type： 杂志文章

    doi：10.1093/nar/gki007

    authors： Kaplan N,Sasson O,Inbar U,Friedlich M,Fromer M,Fleischer H,Portugaly E,Linial N,Linial M

    更新日期：2005-01-01 00:00:00