当前位置： SCI文献检索 > BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS期刊下所有文献 > In vivo gene correction with targeted sequence substitution through microhomology-mediated end joining.

In vivo gene correction with targeted sequence substitution through microhomology-mediated end joining.

Abstract：

:Genome editing technology using programmable nucleases has rapidly evolved in recent years. The primary mechanism to achieve precise integration of a transgene is mainly based on homology-directed repair (HDR). However, an HDR-based genome-editing approach is less efficient than non-homologous end-joining (NHEJ). Recently, a microhomology-mediated end-joining (MMEJ)-based transgene integration approach was developed, showing feasibility both in vitro and in vivo. We expanded this method to achieve targeted sequence substitution (TSS) of mutated sequences with normal sequences using double-guide RNAs (gRNAs), and a donor template flanking the microhomologies and target sequence of the gRNAs in vitro and in vivo. Our method could realize more efficient sequence substitution than the HDR-based method in vitro using a reporter cell line, and led to the survival of a hereditary tyrosinemia mouse model in vivo. The proposed MMEJ-based TSS approach could provide a novel therapeutic strategy, in addition to HDR, to achieve gene correction from a mutated sequence to a normal sequence.

journal_name

Biochem Biophys Res Commun

journal_title

Biochemical and biophysical research communications

authors

Shin JH,Jung S,Ramakrishna S,Kim HH,Lee J

doi

10.1016/j.bbrc.2018.05.130

subject

Has Abstract

pub_date

2018-07-07 00:00:00

pages

116-122

issue

1

eissn

0006-291X

issn

1090-2104

pii

S0006-291X(18)31199-9

journal_volume

502

pub_type

杂志文章

相关文献

BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS文献大全
  • Evidence that TSG101 aberrant transcripts are PCR artifacts.

    abstract：:Critical analysis of the data published so far concerning the TSG101 gene revealed some inconsistencies leading us to its re-evaluation in 80 breast, brain, colon, and neuroectodermal tumors and 37 normal tissue specimens. In this study, the occurrence of TSG101 cDNA aberrant transcripts was verified, but in addition ...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1006/bbrc.1998.9038

    authors： Hampl M,Hampl J,Plaschke J,Fitze G,Schackert G,Saeger HD,Schackert HK

    更新日期：1998-07-30 00:00:00

  • Two isoforms of the rat kidney EP3 receptor derived by alternative RNA splicing: intrarenal expression co-localization.

    abstract：:Two different clones, named rEP3A (approximately 2.2 kb) and rEP3B (approximately 5.2 kb), were isolated from a rat kidney cDNA library by a homology screening approach. rEP3A was shown to encode the rat kidney prostaglandin E receptor EP3 subtype (rEP3A receptor) (Takeuchi, K. et al. B.B.R.C. (1993) 194: 885). rEP3B ...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1006/bbrc.1994.1304

    authors： Takeuchi K,Takahashi N,Abe T,Abe K

    更新日期：1994-03-15 00:00:00

  • Crystal structure of the Epithiospecifier Protein, ESP from Arabidopsis thaliana provides insights into its product specificity.

    abstract：:Specifier proteins are important components of the glucosinolate-myrosinase system, which mediate plant defense against herbivory and pathogen attacks. Upon tissue disruption, glucosinolates are hydrolyzed to instable aglucones by myrosinases, and then aglucones will rearrange to form defensive isothiocyanates. Specif...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/j.bbrc.2016.08.019

    authors： Zhang W,Wang W,Liu Z,Xie Y,Wang H,Mu Y,Huang Y,Feng Y

    更新日期：2016-09-16 00:00:00

  • Leptin enhances CC-chemokine ligand expression in cultured murine macrophage.

    abstract：:Despite accumulating evidence, the role of leptin in chemokine expression is poorly understood. In this study, we evaluated the effects of leptin on CC-chemokine ligands (CCLs), CCL3, CCL4, and CCL5 gene expression in cultured murine macrophage, J774A.1 cells. Expression of all these CCLs mRNA was gradually increased ...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/j.bbrc.2009.04.121

    authors： Kiguchi N,Maeda T,Kobayashi Y,Fukazawa Y,Kishioka S

    更新日期：2009-07-03 00:00:00

  • Role of apoptosis in growth and differentiation of proximal tubule cells in primary cultures.

    abstract：:In this study, we assessed the profile of cell proliferation, Na,K-ATPase activity and apoptosis in primary cultures of proximal tubule cells. Freshly isolated proximal tubules exhibited apoptosis very early in culture: floated cells within 6 hr; attached tubule cells within 20 hr. The onset of apoptosis in either att...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1006/bbrc.1996.0118

    authors： Tang MJ,Cheng YR,Lin HH

    更新日期：1996-01-26 00:00:00

  • Conformational microheterogeneity in a DNA double helix: structure of restriction endonuclease Bam H1 recognition site.

    abstract：:Structural studies using 500 MHz 1H NMR spectroscopy on Bam H1 recognition site d(GGATCC)2 in solution at 19 degrees is reported. The resonances from the sugar ring and base protons have been assigned from the 2D-COSY and NOESY spectra. Analyses of the NOESY cross-peaks between the base protons H8/H6 and sugar protons...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/0006-291x(85)91798-x

    authors： Sarma MH,Dhingra MM,Gupta G,Sarma RH

    更新日期：1985-08-30 00:00:00

  • Molecular characterization and expression of the low-density lipoprotein receptor-related protein-10, a new member of the LDLR gene family.

    abstract：:We report the characterization of a new member of the low-density lipoprotein receptor (LDLR) gene family designated LRP10. Human LRP10 cDNA encodes a 1905 amino acid type I membrane protein consisting of five functional domains characteristic of the LDLR gene family. CHO-ldlA7 cells transfected with human LRP10 cDNA ...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/j.bbrc.2009.12.033

    authors： Jeong YH,Ishikawa K,Someya Y,Hosoda A,Yoshimi T,Yokoyama C,Kiryu-Seo S,Kang MJ,Tchibana T,Kiyama H,Fukumura T,Kim DH,Saeki S

    更新日期：2010-01-01 00:00:00

  • Immunoreactive growth hormone (GH) secretion by human lymphocytes: augmented release by exogenous GH.

    abstract：:Peripheral blood mononuclear cells (PBMCs) from normal adults secreted small amounts of human growth hormone (GH; 0.2-0.6 pg/10(5) cells/7 days culture) as measured by a highly sensitive enzyme immunoassay. Stimulation of PBMCs with phytohemagglutinin (PHA) consistently showed a 4-6 fold increase in GH secretion. Tran...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/0006-291x(90)92334-v

    authors： Hattori N,Shimatsu A,Sugita M,Kumagai S,Imura H

    更新日期：1990-04-30 00:00:00

  • Identification and partial purification of a chromatin bound calmodulin activated histone 3 kinase from calf thymus.

    abstract：:A calcium-calmodulin (Ca2(+)-CaM) stimulated histone H3 phosphorylating activity was identified as a component of a nuclear protein complex purified from a 150 mM NaCl extract of calf thymus chromatin. This activity bound to a CaM-Sepharose affinity column in a Ca2+ dependent manner and was eluted off the column in th...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/0006-291x(90)91359-z

    authors： Wakim BT,Picken MM,DeLange RJ

    更新日期：1990-08-31 00:00:00

  • Formation of HIV-1 envelope-hepatitis B core antigen hybrids with high affinity for CD4.

    abstract：:We have identified an acceptor site on HIV gp120, where foreign protein sequences can be inserted while retaining the native conformation of gp120. The resulting hybrids showed dual antigenicity, normal glycosylation, and high affinity binding of the CD4 receptor. This site allows insertion of highly immunogenic prote...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1006/bbrc.2001.5227

    authors： Patterson LJ,Aberdeen A,Kone J,Haben M,Raymond M,Berkower I

    更新日期：2001-07-20 00:00:00

  • Activation of glutathione peroxidase via Nrf1 mediates genistein's protection against oxidative endothelial cell injury.

    abstract：:Cellular actions of isoflavones may mediate the beneficial health effects associated with high soy consumption. We have investigated protection by genistein and daidzein against oxidative stress-induced endothelial injury. Genistein but not daidzein protected endothelial cells from damage induced by oxidative stress. ...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/j.bbrc.2006.05.197

    authors： Hernandez-Montes E,Pollard SE,Vauzour D,Jofre-Montseny L,Rota C,Rimbach G,Weinberg PD,Spencer JP

    更新日期：2006-08-04 00:00:00

  • Steroid-lipid interactions in sonicated dipalmitoyl phosphatidyl choline vesicles: a steady-state and time-resolved fluorescence anisotropy study with all trans-1,6-diphenyl-1,3,5-hexatriene as probe.

    abstract：:The effect of steroid chemical structure on steroid-lipid interaction is studied in sonicated dipalmitoyl phosphatidylcholine vesicles by steady-state and time-resolved fluorescence anisotropy techniques using diphenylhexatriene as probe. A large number of cholesterol derivatives and steroids, some of them being precu...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/0006-291x(83)91070-7

    authors： Vincent M,Gallay J

    更新日期：1983-06-29 00:00:00

  • The fate of internalized alpha-2-macroglobulin: alpha-galactosidase conjugate in fibroblasts from Fabry's hemizygote.

    abstract：:In our previous report we described the endocytotic incorporation of coffee bean alpha-galactosidase conjugated to human alpha-2-macroglobulin (alpha-2-M) into cultured fibroblasts derived from a patient with Fabry's disease (1). The fate of internalized alpha-galactosidase according to the method described in the abo...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/0006-291x(87)90343-3

    authors： Osada T,Kuroda Y,Ikai A

    更新日期：1987-03-30 00:00:00

  • Auxin-regulated changes in protein phosphorylation in pea epicotyls.

    abstract：:Auxins regulate various aspects of plant growth and development. However, the mechanism by which these hormones elicit diverse physiological processes is not clear. We present evidence for the role of auxin in protein phosphorylation and the possible involvement of calmodulin in auxin-induced changes. In the presence ...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/s0006-291x(87)80055-4

    authors： Reddy AS,Chengappa S,Poovaiah BW

    更新日期：1987-04-29 00:00:00

  • On the enigma of pain and hyperalgesia: A molecular perspective.

    abstract：:Pain is a common symptom of injuries and inflammatory-related conditions. The perception of pain, commonly known as nociception, depends on integrated receptors and molecular pathways. Inflammatory mediators are involved in the genesis, persistence, and severity of pain. Noxious stimuli can trigger a cascade of inflam...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章,评审

    doi：10.1016/j.bbrc.2006.12.032

    authors： Haddad JJ

    更新日期：2007-02-09 00:00:00

  • CXCR4 and PTEN are involved in the anti-metastatic regulation of anethole in DU145 prostate cancer cells.

    abstract：:Anethole has been known to have chemopreventive activities as a suppressor of the incidence and multiplicity of both invasive and noninvasive carcinomas. The goal of this study was to understand the anti-metastatic effect of anethole through C-X-C chemokine receptor type 4 (CXCR4)/tumor suppressor phosphatase and tens...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/j.bbrc.2014.01.121

    authors： Rhee YH,Chung PS,Kim SH,Ahn JC

    更新日期：2014-05-16 00:00:00

  • T7 DNA polymerase is not a zinc-metalloenzyme and the polymerase and exonuclease activities are inhibited by zinc ions.

    abstract：:Phage T7 DNA polymerase purified to homogeneity by an antithioredoxin immunoadsorbent technique was resolved into its active subunits the gene 5 protein and Escherichia coli thioredoxin by a novel technique involving chromatography on Sephadex G-50 at pH 11.5. Analysis of the metal content of the holoenzyme by atomic ...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/0006-291x(84)91248-8

    authors： Slaby I,Lind B,Holmgren A

    更新日期：1984-08-16 00:00:00

  • Identification of an unconventional nuclear localization signal in human ribosomal protein S2.

    abstract：:Ribosomal proteins must be imported into the nucleus after being synthesized in the cytoplasm. Since the rpS2 amino acid sequence does not contain a typical nuclear localization signal, we used deletion mutant analysis and rpS2-beta-galactosidase chimeric proteins to identify the nuclear targeting domains in rpS2. Nuc...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/j.bbrc.2005.07.069

    authors： Antoine M,Reimers K,Wirz W,Gressner AM,Müller R,Kiefer P

    更新日期：2005-09-16 00:00:00

  • A brain-specific decrease of the tyrosine hydroxylase protein in sepiapterin reductase-null mice--as a mouse model for Parkinson's disease.

    abstract：:Sepiapterin reductase (SPR) is an enzyme that acts in the third and final step of tetrahydrobiopterin (BH4) biosynthesis. The human Spr gene locates within the region of 2.5MB mapped to PARK3, an autosomal dominant form of familial Parkinson's diseases. In order to explore the role of SPR in the metabolism of BH4, we ...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/j.bbrc.2008.01.028

    authors： Takazawa C,Fujimoto K,Homma D,Sumi-Ichinose C,Nomura T,Ichinose H,Katoh S

    更新日期：2008-03-21 00:00:00

  • Up-regulation of long noncoding RNA MINCR promotes non-small cell of lung cancer growth by negatively regulating miR-126/SLC7A5 axis.

    abstract：:A growing body of evidence suggests that MYC induced long noncoding RNA (MINCR) is involved in the initiation and progression of various tumors. However, little is known about the biological function and clinical value of MINCR in non-small cell lung cancer (NSCLC). In the present study, results found that MINCR over ...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/j.bbrc.2018.11.162

    authors： Wang J,Ding M,Zhu H,Cao Y,Zhao W

    更新日期：2019-01-15 00:00:00

  • Adhesion of mouse hepatocytes to type I collagen: role of supramolecular forms and effect of proteolytic degradation.

    abstract：:Mouse hepatocytes were found to adhere much stronger to intact native type I collagen than to native, telopeptide-devoid collagen. Removal of telopeptides (by pepsin treatment) caused disintegration of supramolecular collagen aggregates. Similar differences in hepatocyte adhesion were observed between oligomeric and m...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1006/bbrc.1993.1827

    authors： Berman A,Morozevich G,Karmansky I,Gleiberman A,Bychkova V

    更新日期：1993-07-15 00:00:00

  • Dehydrogenase binding to the 3'-untranslated region of GLUT1 mRNA.

    abstract：:Employing RNA gel mobility shift assays we detected specific binding events between several dehydrogenases and various regions of the GLUT1 mRNA 3'-untranslated region. In particular, the enzymes glyceraldehyde 3-phosphate dehydrogenase (G3PDH), lactate dehydrogenase (LDH), and glucose 6-phosphate dehydrogenase (G6PDH...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1006/bbrc.1996.0541

    authors： McGowan K,Pekala PH

    更新日期：1996-04-05 00:00:00

  • Molecular cloning of cDNA for human poly(ADP-ribose) polymerase and expression of its gene during HL-60 cell differentiation.

    abstract：:Human placental poly(ADP-ribose) polymerase was purified and the NH2-terminal amino acid sequences of 16 KDa and 40 KDa chymotryptic peptides were determined. Screening of a lambda gt11 cDNA library of normal human placenta with a 51-mer oligodeoxyribonucleotide yielded one clone with a 1.8 Kb insert and two clones wi...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/0006-291x(87)90543-2

    authors： Suzuki H,Uchida K,Shima H,Sato T,Okamoto T,Kimura T,Miwa M

    更新日期：1987-07-31 00:00:00

  • Hydrogen-rich medium protects human skin fibroblasts from high glucose or mannitol induced oxidative damage.

    abstract：:Reactive oxygen species (ROS) are an important factor in the development of skin lesions in diabetes. A new antioxidant, hydrogen, can selectively neutralize hydroxyl radicals (()OH) and peroxynitrite (ONOO(-)) in cell-free systems, whereas it seldom reacts with other ROS. Fibroblasts are a key component of skin. In t...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/j.bbrc.2011.05.024

    authors： Yu P,Wang Z,Sun X,Chen X,Zeng S,Chen L,Li S

    更新日期：2011-06-03 00:00:00

  • Is cAMP the obligatory second messenger in the action of lutropin on Leydig cell steroidogenesis.

    abstract：:Two adenylate cyclase inhibitors: 9-(tetrahydro-2-furyl)adenine and 2'5'-dideoxyadenosine decreased cAMP levels in LH-stimulated immature rat Leydig cells by 20-40%, independent of the concentration of LH. Steroid production was not correlated with this decrease in cAMP, but was increased (146%). The phorbol ester 4 b...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/0006-291x(85)91063-0

    authors： Themmen AP,Hoogerbrugge JW,Rommerts FF,van der Molen HJ

    更新日期：1985-05-16 00:00:00

  • Negative and positive elements in the promoter region of the human apoferritin L gene.

    abstract：:We have characterized the promoter of the human gene coding for the apoferritin L subunit. Transient transfections of 5' and 3' deletion mutants indicate that the efficiency of the L promoter depends on both negative and positive cis-elements, located upstream and downstream of the transcription start point. DNaseI fo...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1006/bbrc.1995.2470

    authors： D'Agostino P,Faniello MC,Quaresima B,Bevilacqua MA,Tiano MT,Ammendola R,Cimino F,Costanzo F

    更新日期：1995-10-04 00:00:00

  • Investigation of antigen delivery route in vivo and imune-boosting effects mediated by pH-sensitive liposomes encapsulated with K(b)-restricted CTL epitope.

    abstract：:Using fluorescein isothiocyanate (FITC)-conjugated H-2K(b) CTL epitope (SIINFEKL) as a model system, we investigated the antigen delivery route by pH-sensitive liposomes in vivo. Fluorescence was initially detected in lymph nodes at 3 h after immunization, and its intensity reached a peak value in superticial inguinal...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1006/bbrc.2002.6711

    authors： Lee KY,Chun E,Seong BL

    更新日期：2002-04-05 00:00:00

  • Isolation and identification of C-type natriuretic peptide in human monocytic cell line, THP-1.

    abstract：:In a survey for unknown bioactive peptides in mammalian cell lines, we isolated peptides exhibiting a strong relaxant effect on chick rectum from a phorbol ester-supplemented culture medium of the human monocytic cell line, THP-1. The peptide was deduced to be a C-terminal 29-residue peptide derived from a human C-typ...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/0006-291x(92)92257-x

    authors： Ishizaka Y,Kangawa K,Minamino N,Ishii K,Takano S,Eto T,Matsuo H

    更新日期：1992-12-15 00:00:00

  • The role of apolipoprotein E in the elimination of liposomes from blood by hepatocytes in the mouse.

    abstract：:We evaluated the role of apolipoprotein E (apoE) in the clearance of neutral and negatively charged liposomes by hepatocytes in apoE-deficient mice. Negatively charged liposomes were cleared at identical rates in apoE-deficient and wild-type mice; neutral liposomes were cleared at a 3.6-fold slower rate in apoE-defici...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1016/j.bbrc.2004.12.137

    authors： Yan X,Kuipers F,Havekes LM,Havinga R,Dontje B,Poelstra K,Scherphof GL,Kamps JA

    更新日期：2005-03-04 00:00:00

  • Distinct characteristics of receptor-operated Ca2+ influx and refilling in pancreatic acinar cells.

    abstract：:Ca2+ influx from the extracellular space in nonexcitable cells occurs via receptor-operated and refilling processes. However, they showed different characteristics with respect to the Mn2+ permeability, depletion of intracellular Ca2+ stores, and sensitivity to the K+ ionophore valinomycin in rat pancreatic acinar cel...

    journal_title：Biochemical and biophysical research communications

    pub_type： 杂志文章

    doi：10.1006/bbrc.1999.0305

    authors： Tsunoda Y,Tashiro Y

    更新日期：1999-03-24 00:00:00