当前位置： SCI文献检索 > INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES期刊下所有文献 > Synergy between Readthrough and Nonsense Mediated Decay Inhibition in a Murine Model of Cystic Fibrosis Nonsense Mutations.

Synergy between Readthrough and Nonsense Mediated Decay Inhibition in a Murine Model of Cystic Fibrosis Nonsense Mutations.

Abstract：

:Many heritable genetic disorders arise from nonsense mutations, which generate premature termination codons (PTCs) in transcribed mRNA. PTCs ablate protein synthesis by prematurely terminating the translation of mutant mRNA, as well as reducing mutant mRNA quantity through targeted degradation by nonsense-mediated decay (NMD) mechanisms. Therapeutic strategies for nonsense mutations include facilitating ribosomal readthrough of the PTC and/or inhibiting NMD to restore protein function. However, the efficacy of combining readthrough agents and NMD inhibitors has not been thoroughly explored. In this study, we examined combinations of known NMD inhibitors and readthrough agents using functional analysis of the CFTR protein in primary cells from a mouse model carrying a G542X nonsense mutation in Cftr. We observed synergy between an inhibitor of the NMD component SMG-1 (SMG1i) and the readthrough agents G418, gentamicin, and paromomycin, but did not observe synergy with readthrough caused by amikacin, tobramycin, PTC124, escin, or amlexanox. These results indicate that treatment with NMD inhibitors can increase the quantity of functional protein following readthrough, and that combining NMD inhibitors and readthrough agents represents a potential therapeutic option for treating nonsense mutations.

journal_name

Int J Mol Sci

journal_title

International journal of molecular sciences

authors

McHugh DR,Cotton CU,Hodges CA

doi

10.3390/ijms22010344

subject

Has Abstract

pub_date

2020-12-31 00:00:00

issue

1

issn

1422-0067

pii

ijms22010344

journal_volume

22

pub_type

杂志文章

相关文献

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES文献大全
  • Circulating Tumour Cell Expression of Immune Markers as Prognostic and Therapeutic Biomarkers in Head and Neck Squamous Cell Carcinoma: A Systematic Review and Meta-Analysis.

    abstract：:Rates of loco-regional recurrence and distant metastasis remain high among head and neck squamous cell carcinoma (HNSCC) patients, despite advancing cancer treatment modalities and therapeutic agents. One area that has generated considerable interest is the immune landscape of the tumour, heralding a wave of immune ch...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章,评审

    doi：10.3390/ijms21218229

    authors： Payne K,Pugh M,Brooks J,Batis N,Taylor G,Nankivell P,Mehanna H

    更新日期：2020-11-03 00:00:00

  • Alpha-Synuclein RNA Expression is Increased in Major Depression.

    abstract：:Alpha-synuclein (SNCA) is a small membrane protein that plays an important role in neuro-psychiatric diseases. It is best known for its abnormal subcellular aggregation in Lewy bodies that serves as a hallmark of Parkinson's disease (PD). Due to the high comorbidity of PD with depression, we investigated the role of S...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms20082029

    authors： Rotter A,Lenz B,Pitsch R,Richter-Schmidinger T,Kornhuber J,Rhein C

    更新日期：2019-04-25 00:00:00

  • Potential mechanisms linking atherosclerosis and increased cardiovascular risk in COPD: focus on Sirtuins.

    abstract：:The development of atherosclerosis is a multi-step process, at least in part controlled by the vascular endothelium function. Observations in humans and experimental models of atherosclerosis have identified monocyte recruitment as an early event in atherogenesis. Chronic inflammation is associated with ageing and its...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章,评审

    doi：10.3390/ijms140612696

    authors： Corbi G,Bianco A,Turchiarelli V,Cellurale M,Fatica F,Daniele A,Mazzarella G,Ferrara N

    更新日期：2013-06-17 00:00:00

  • The Soybean GmNAC019 Transcription Factor Mediates Drought Tolerance in Arabidopsis in an Abscisic Acid-Dependent Manner.

    abstract：:Being master regulators of gene expression, transcription factors (TFs) play important roles in determining plant growth, development and reproduction. To date, many TFs have been shown to positively mediate plant responses to environmental stresses. In the current study, the biological functions of a stress-responsiv...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms21010286

    authors： Hoang XLT,Nguyen NC,Nguyen YH,Watanabe Y,Tran LP,Thao NP

    更新日期：2019-12-31 00:00:00

  • Tumour-Derived Extracellular Vesicles (EVs): A Dangerous "Message in A Bottle" for Bone.

    abstract：:Several studies have shown the importance of Extracellular Vesicles (EVs) in the intercellular communication between tumour and resident cells. Through EVs, tumour cells can trigger cell-signalling molecules and shuttle exogenous information to target cells, thus promoting spread of the disease. In fact, many processe...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章,评审

    doi：10.3390/ijms20194805

    authors： Cappariello A,Rucci N

    更新日期：2019-09-27 00:00:00

  • Protective Effects of α-Tocopherol, γ-Tocopherol and Oleic Acid, Three Compounds of Olive Oils, and No Effect of Trolox, on 7-Ketocholesterol-Induced Mitochondrial and Peroxisomal Dysfunction in Microglial BV-2 Cells.

    abstract：:Lipid peroxidation products, such as 7-ketocholesterol (7KC), may be increased in the body fluids and tissues of patients with neurodegenerative diseases and trigger microglial dysfunction involved in neurodegeneration. It is therefore important to identify synthetic and natural molecules able to impair the toxic effe...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms17121973

    authors： Debbabi M,Nury T,Zarrouk A,Mekahli N,Bezine M,Sghaier R,Grégoire S,Martine L,Durand P,Camus E,Vejux A,Jabrane A,Bretillon L,Prost M,Moreau T,Ammou SB,Hammami M,Lizard G

    更新日期：2016-11-25 00:00:00

  • Increased Cytotoxicity of Herpes Simplex Virus Thymidine Kinase Expression in Human Induced Pluripotent Stem Cells.

    abstract：:Human induced pluripotent stem cells (iPSCs) hold enormous promise for regenerative medicine. The major safety concern is the tumorigenicity of transplanted cells derived from iPSCs. A potential solution would be to introduce a suicide gene into iPSCs as a safety switch. The herpes simplex virus type 1 thymidine kinas...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms20040810

    authors： Iwasawa C,Tamura R,Sugiura Y,Suzuki S,Kuzumaki N,Narita M,Suematsu M,Nakamura M,Yoshida K,Toda M,Okano H,Miyoshi H

    更新日期：2019-02-14 00:00:00

  • Nucleotides-Induced Changes in the Mechanical Properties of Living Endothelial Cells and Astrocytes, Analyzed by Atomic Force Microscopy.

    abstract：:Endothelial cells and astrocytes preferentially express metabotropic P2Y nucleotide receptors, which are involved in the maintenance of vascular and neural function. Among these, P2Y1 and P2Y2 receptors appear as main actors, since their stimulation induces intracellular calcium mobilization and activates signaling ca...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms22020624

    authors： Gil-Redondo JC,Iturri J,Ortega F,Pérez-Sen R,Weber A,Miras-Portugal MT,Toca-Herrera JL,Delicado EG

    更新日期：2021-01-10 00:00:00

  • Coactivator recruitment of AhR/ARNT1.

    abstract：:A common feature of nuclear receptors (NRs) is the transformation of external cell signals into specific transcriptions of the signal molecule. Signal molecules function as ligands for NRs and, after their uptake, activated NRs form homo- or heterodimers at promoter recognition sequences of the specific genes in the n...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章,评审

    doi：10.3390/ijms150611100

    authors： Endler A,Chen L,Shibasaki F

    更新日期：2014-06-19 00:00:00

  • Combinatorial Drug Treatments Reveal Promising Anticytomegaloviral Profiles for Clinically Relevant Pharmaceutical Kinase Inhibitors (PKIs).

    abstract：:Human cytomegalovirus (HCMV) is a human pathogenic herpesvirus associated with a variety of clinical symptoms. Current antiviral therapy is not always effective, so that improved drug classes and drug-targeting strategies are needed. Particularly host-directed antivirals, including pharmaceutical kinase inhibitors (PK...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms22020575

    authors： Wild M,Kicuntod J,Seyler L,Wangen C,Bertzbach LD,Conradie AM,Kaufer BB,Wagner S,Michel D,Eickhoff J,Tsogoeva SB,Bäuerle T,Hahn F,Marschall M

    更新日期：2021-01-08 00:00:00

  • Production of MCM-41 Nanoparticles with Control of Particle Size and Structural Properties: Optimizing Operational Conditions during Scale-Up.

    abstract：:The synthesis of Mobil Composition of Matter 41 (MCM-41) mesoporous silica nanoparticles (MSNs) of controlled sizes and porous structure has been performed at laboratory and pilot plant scales. Firstly, the effects of the main operating conditions (TEOS -Tetraethyl ortosilicate- addition rate, nanoparticle maturation ...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms21217899

    authors： Castillo RR,de la Torre L,García-Ochoa F,Ladero M,Vallet-Regí M

    更新日期：2020-10-24 00:00:00

  • Unravelling genes and pathways implicated in working memory of schizophrenia in Han Chinese.

    abstract：:Working memory deficit is the core neurocognitive disorder in schizophrenia patients. To identify the factors underlying working memory deficit in schizophrenia patients and to explore the implication of possible genes in the working memory using genome-wide association study (GWAS) of schizophrenia, computerized dela...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms16012145

    authors： Ren H,Zhang C,Huang C,Li N,Li M,Li Y,Deng W,Ma X,Xiang B,Wang Q,Li T

    更新日期：2015-01-20 00:00:00

  • Hub promiscuity in protein-protein interaction networks.

    abstract：:Hubs are proteins with a large number of interactions in a protein-protein interaction network. They are the principal agents in the interaction network and affect its function and stability. Their specific recognition of many different protein partners is of great interest from the structural viewpoint. Over the last...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章,评审

    doi：10.3390/ijms11041930

    authors： Patil A,Kinoshita K,Nakamura H

    更新日期：2010-04-26 00:00:00

  • A chitosan induced 9-lipoxygenase in Adelostemma gracillimum seedlings.

    abstract：:Oxylipins generated by the lipoxygenase (LOX) pathway play an important role in plant defense against biotic and abiotic stress. In chitosan-treated Adelostemma gracillimum seedlings, obvious accumulation of 9-LOX-derived oxylipins, namely 9,10,11-trihydroxy-12-octadecenoic acid, was detected. Using degenerate primers...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms13010540

    authors： Li J,Zhao PJ,Ma CL,Zeng Y

    更新日期：2012-01-01 00:00:00

  • Thrombin Receptor-Activating Protein (TRAP)-Activated Akt Is Involved in the Release of Phosphorylated-HSP27 (HSPB1) from Platelets in DM Patients.

    abstract：:It is generally known that heat shock protein 27 (HSP27) is phosphorylated through p38 mitogen-activated protein (MAP) kinase. We have previously reported that HSP27 is released from human platelets associated with collagen-induced phosphorylation. In the present study, we conducted an investigation into the effect of...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms17050737

    authors： Tokuda H,Kuroyanagi G,Tsujimoto M,Matsushima-Nishiwaki R,Akamatsu S,Enomoto Y,Iida H,Otsuka T,Ogura S,Iwama T,Kojima K,Kozawa O

    更新日期：2016-05-14 00:00:00

  • Evaluation of the Genotoxic and Oxidative Damage Potential of Silver Nanoparticles in Human NCM460 and HCT116 Cells.

    abstract：:Nano Ag has excellent antibacterial properties and is widely used in various antibacterial materials, such as antibacterial medicine and medical devices, food packaging materials and antibacterial textiles. Despite the many benefits of nano-Ag, more and more research indicates that it may have potential biotoxic effec...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms21051618

    authors： Jia M,Zhang W,He T,Shu M,Deng J,Wang J,Li W,Bai J,Lin Q,Luo F,Zhou W,Zeng X

    更新日期：2020-02-27 00:00:00

  • Influence of Cardiovascular Risk Factors, Comorbidities, Medication Use and Procedural Variables on Remote Ischemic Conditioning Efficacy in Patients with ST-Segment Elevation Myocardial Infarction.

    abstract：:Remote ischemic conditioning (RIC) confers cardioprotection in patients with ST-segment elevation myocardial infarction (STEMI). Despite intense research, the translation of RIC into clinical practice remains a challenge. This may, at least partly, be due to confounding factors that may modify the efficacy of RIC. The...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章,评审

    doi：10.3390/ijms20133246

    authors： Pryds K,Hjortbak MV,Schmidt MR

    更新日期：2019-07-02 00:00:00

  • Nanoparticle-Mediated Combination Therapy: Two-in-One Approach for Cancer.

    abstract：:Cancer represents a group of heterogeneous diseases characterized by uncontrolledgrowth and spread of abnormal cells, ultimately leading to death. Nanomedicine plays a significantrole in the development of nanodrugs, nanodevices, drug delivery systems and nanocarriers. Someof the major issues in the treatment of cance...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章,评审

    doi：10.3390/ijms19103264

    authors： Gurunathan S,Kang MH,Qasim M,Kim JH

    更新日期：2018-10-20 00:00:00

  • Programming Effects of Prenatal Glucocorticoid Exposure with a Postnatal High-Fat Diet in Diabetes Mellitus.

    abstract：:Increasing evidence has shown that many chronic diseases originate from early life, even before birth, through what are termed as fetal programming effects. Glucocorticoids are frequently used prenatally to accelerate the maturation of the lungs of premature infants. High-fat diets are associated with insulin resistan...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms17040533

    authors： Sheen JM,Hsieh CS,Tain YL,Li SW,Yu HR,Chen CC,Tiao MM,Chen YC,Huang LT

    更新日期：2016-04-08 00:00:00

  • Synergistic Effect of Beauveria bassiana and Trichoderma asperellum to Induce Maize (Zea mays L.) Defense against the Asian Corn Borer, Ostrinia furnacalis (Lepidoptera, Crambidae) and Larval Immune Response.

    abstract：:Ostrinia furnacalis, is the major pest of maize causing significant yield losses. So far, many approaches have been used to increase the virulence of entomopathogenic fungal isolates. The current study is an attempt to estimate synergistic effect of Beauveria bassiana and Trichoderma asperellum in order to explore lar...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms21218215

    authors： Batool R,Umer MJ,Wang Y,He K,Zhang T,Bai S,Zhi Y,Chen J,Wang Z

    更新日期：2020-11-03 00:00:00

  • Dual Role of Bile Acids on the Biliary Epithelium: Friend or Foe?

    abstract：:Bile acids are a family of amphipathic compounds predominantly known for their role in solubilizing and absorbing hydrophobic compounds (including liposoluble vitamins) in the intestine. Bile acids also are key signaling molecules and inflammatory agents that activate transcriptional factors and cell signaling pathway...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章,评审

    doi：10.3390/ijms20081869

    authors： Baiocchi L,Zhou T,Liangpunsakul S,Lenci I,Santopaolo F,Meng F,Kennedy L,Glaser S,Francis H,Alpini G

    更新日期：2019-04-16 00:00:00

  • Biomimetic Membranes with Transmembrane Proteins: State-of-the-Art in Transmembrane Protein Applications.

    abstract：:In biological cells, membrane proteins are the most crucial component for the maintenance of cell physiology and processes, including ion transportation, cell signaling, cell adhesion, and recognition of signal molecules. Therefore, researchers have proposed a number of membrane platforms to mimic the biological cell ...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章,评审

    doi：10.3390/ijms20061437

    authors： Ryu H,Fuwad A,Yoon S,Jang H,Lee JC,Kim SM,Jeon TJ

    更新日期：2019-03-21 00:00:00

  • Nucleated polymerisation in the presence of pre-formed seed filaments.

    abstract：:We revisit the classical problem of nucleated polymerisation and derive a range of exact results describing polymerisation in systems intermediate between the well-known limiting cases of a reaction starting from purely soluble material and for a reaction where no new growth nuclei are formed. ...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms12095844

    authors： Cohen SI,Vendruscolo M,Dobson CM,Knowles TP

    更新日期：2011-01-01 00:00:00

  • Sarcopenia Induced by Chronic Liver Disease in Mice Requires the Expression of the Bile Acids Membrane Receptor TGR5.

    abstract：:Sarcopenia is a condition of muscle dysfunction, commonly associated with chronic liver disease (CLD), characterized by a decline in muscle strength, the activation of the ubiquitin-proteasome system (UPS), and oxidative stress. We recently described a murine model of CLD-induced sarcopenia by intake of hepatotoxin 3,...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms21217922

    authors： Abrigo J,Campos F,Gonzalez F,Aguirre F,Gonzalez A,Huerta-Salgado C,Conejeros S,Simon F,Arrese M,Cabrera D,Elorza AA,Cabello-Verrugio C

    更新日期：2020-10-25 00:00:00

  • Role of Major Endocannabinoid-Binding Receptors during Mouse Oocyte Maturation.

    abstract：:Endocannabinoids are key-players of female fertility and potential biomarkers of reproductive dysfunctions. Here, we investigated localization and expression of cannabinoid receptor type-1 and -2 (CB1R and CB2R), G-protein coupled receptor 55 (GPR55), and transient receptor potential vanilloid type 1 channel (TRPV1) i...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms20122866

    authors： Cecconi S,Rossi G,Oddi S,Di Nisio V,Maccarrone M

    更新日期：2019-06-12 00:00:00

  • Genetic analysis of health-related secondary metabolites in a Brassica rapa recombinant inbred line population.

    abstract：:The genetic basis of the wide variation for nutritional traits in Brassica rapa is largely unknown. A new Recombinant Inbred Line (RIL) population was profiled using High Performance Liquid Chromatography (HPLC) and Nuclear Magnetic Resonance (NMR) analysis to detect quantitative trait loci (QTLs) controlling seed toc...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms140815561

    authors： Bagheri H,El-Soda M,Kim HK,Fritsche S,Jung C,Aarts MG

    更新日期：2013-07-25 00:00:00

  • Bistable switch in let-7 miRNA biogenesis pathway involving Lin28.

    abstract：:miRNAs are small noncoding RNAs capable of regulating gene expression at the post-transcriptional level. A growing body of evidence demonstrated that let-7 family of miRNAs, as one of the highly conserved miRNAs, plays an important role in cell differentiation and development, as well as tumor suppressor function depe...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms151019119

    authors： Shi F,Yu W,Wang X

    更新日期：2014-10-21 00:00:00

  • Learned Immobility Produces Enduring Impairment of the HPA Axis Reactivity in Mice without Replicating the Broad Spectrum of Depressive-Like Phenotype.

    abstract：:The forced swim stress test (FST) is widely used for screening pharmacological or non-pharmacological strategies with potential antidepressant activities. Recent data have suggested that repeated FST for five consecutive days (i.e., 5d-RFSS) could be used to generate a robust depressive-like phenotype in mice. However...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms22020937

    authors： Bullich S,Delcourte S,Haddjeri N,Guiard BP

    更新日期：2021-01-19 00:00:00

  • Esculetin, a Coumarin Derivative, Prevents Thrombosis: Inhibitory Signaling on PLCγ2-PKC-AKT Activation in Human Platelets.

    abstract：:Esculetin, a bioactive 6,7-dihydroxy derivative of coumarin, possesses pharmacological activities against obesity, diabetes, renal failure, and cardiovascular disorders (CVDs). Platelet activation plays a major role in CVDs. Thus, disrupting platelet activation represents an attractive therapeutic target. We examined ...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms20112731

    authors： Hsia CW,Lin KC,Lee TY,Hsia CH,Chou DS,Jayakumar T,Velusamy M,Chang CC,Sheu JR

    更新日期：2019-06-03 00:00:00

  • CX3CR1 Deficiency Attenuates DNFB-Induced Contact Hypersensitivity Through Skewed Polarization Towards M2 Phenotype in Macrophages.

    abstract：:CX3CL1 can function as both an adhesion molecule and a chemokine for CX3CR1+ cells, such as T cells, monocytes, and NK cells. Recent studies have demonstrated that CX3CL1-CX3CR1 interaction is associated with the development of various inflammatory skin diseases. In this study, we examined CX3CR1 involvement in 2,4-di...

    journal_title：International journal of molecular sciences

    pub_type： 杂志文章

    doi：10.3390/ijms21197401

    authors： Otobe S,Hisamoto T,Miyagaki T,Morimura S,Suga H,Sugaya M,Sato S

    更新日期：2020-10-07 00:00:00