Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules.

Abstract:

:This review, one in a series on myotonic dystrophy (DM), is focused on the development and potential use of small molecules as therapeutics for DM. The complex mechanisms and pathogenesis of DM are covered in the associated reviews. Here, we examine the various small molecule approaches taken to target the DNA, RNA, and proteins that contribute to disease onset and progression in myotonic dystrophy type 1 (DM1) and 2 (DM2).

journal_name

Int J Mol Sci

authors

Reddy K,Jenquin JR,Cleary JD,Berglund JA

doi

10.3390/ijms20164017

subject

Has Abstract

pub_date

2019-08-17 00:00:00

issue

16

issn

1422-0067

pii

ijms20164017

journal_volume

20

pub_type

杂志文章,评审
  • The Primary Causes of Muscle Dysfunction Associated with the Point Mutations in Tpm3.12; Conformational Analysis of Mutant Proteins as a Tool for Classification of Myopathies.

    abstract::Point mutations in genes encoding isoforms of skeletal muscle tropomyosin may cause nemaline myopathy, cap myopathy (Cap), congenital fiber-type disproportion (CFTD), and distal arthrogryposis. The molecular mechanisms of muscle dysfunction in these diseases remain unclear. We studied the effect of the E173A, R90P, E1...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms19123975

    authors: Borovikov YS,Karpicheva OE,Simonyan AO,Avrova SV,Rogozovets EA,Sirenko VV,Redwood CS

    更新日期:2018-12-10 00:00:00

  • Prevention of Colorectal Cancer by Targeting Obesity-Related Disorders and Inflammation.

    abstract::Colorectal cancer is a major healthcare concern worldwide. Many experimental and clinical studies have been conducted to date to discover agents that help in the prevention of this disease. Chronic inflammation in colonic mucosa and obesity, and its related metabolic abnormalities, are considered to increase the risk ...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章,评审

    doi:10.3390/ijms18050908

    authors: Shirakami Y,Ohnishi M,Sakai H,Tanaka T,Shimizu M

    更新日期:2017-04-26 00:00:00

  • Detection of Bacterial Pathogens from Broncho-Alveolar Lavage by Next-Generation Sequencing.

    abstract::The applications of whole-metagenome shotgun sequencing (WMGS) in routine clinical analysis are still limited. A combination of a DNA extraction procedure, sequencing, and bioinformatics tools is essential for the removal of human DNA and for improving bacterial species identification in a timely manner. We tackled th...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms18092011

    authors: Leo S,Gaïa N,Ruppé E,Emonet S,Girard M,Lazarevic V,Schrenzel J

    更新日期:2017-09-20 00:00:00

  • Metagenomic Analysis of Upwelling-Affected Brazilian Coastal Seawater Reveals Sequence Domains of Type I PKS and Modular NRPS.

    abstract::Marine environments harbor a wide range of microorganisms from the three domains of life. These microorganisms have great potential to enable discovery of new enzymes and bioactive compounds for industrial use. However, only ~1% of microorganisms from the environment can currently be identified through cultured isolat...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms161226101

    authors: Cuadrat RR,Cury JC,Dávila AM

    更新日期:2015-11-27 00:00:00

  • Circulating microRNA: The Potential Novel Diagnostic Biomarkers to Predict Drug Resistance in Temporal Lobe Epilepsy, a Pilot Study.

    abstract::MicroRNAs (miRNAs) are small noncoding RNAs that have emerged as new potential epigenetic biomarkers. Here, we evaluate the efficacy of six circulating miRNA previously described in the literature as biomarkers for the diagnosis of temporal lobe epilepsy (TLE) and/or as predictive biomarkers to antiepileptic drug resp...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms22020702

    authors: De Benedittis S,Fortunato F,Cava C,Gallivanone F,Iaccino E,Caligiuri ME,Castiglioni I,Bertoli G,Manna I,Labate A,Gambardella A

    更新日期:2021-01-12 00:00:00

  • Vitamin D Supplementation in Central Nervous System Demyelinating Disease-Enough Is Enough.

    abstract::The exact cause of multiple sclerosis (MS) remains elusive. Various factors, however, have been identified that increase an individual's risk of developing this central nervous system (CNS) demyelinating disease and are associated with an acceleration in disease severity. Besides genetic determinants, environmental fa...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章,评审

    doi:10.3390/ijms20010218

    authors: Häusler D,Weber MS

    更新日期:2019-01-08 00:00:00

  • Differentially Expressed microRNAs in MIA PaCa-2 and PANC-1 Pancreas Ductal Adenocarcinoma Cell Lines are Involved in Cancer Stem Cell Regulation.

    abstract::Pancreatic ductal adenocarcinoma (PDAC) is one of the most lethal malignancies, and thus better understanding of its molecular pathology is crucial for us to devise more effective treatment of this deadly disease. As cancer cell line remains a convenient starting point for discovery and proof-of-concept studies, here ...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms20184473

    authors: Shen Y,Pu K,Zheng K,Ma X,Qin J,Jiang L,Li J

    更新日期:2019-09-10 00:00:00

  • Omega Fatty Acids and Inflammatory Bowel Diseases: An Overview.

    abstract::Inflammatory bowel diseases (IBD) are chronic, inflammatory processes that affect the gastrointestinal tract and are mainly represented by ulcerative colitis (UC) and Crohn's disease (CD). Omega 3 (ω3) fatty acids (eicosapentanoic acid and docosahexaenoic acid) show an indispensable role in the inflammatory processes ...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章,meta分析,评审

    doi:10.3390/ijms20194851

    authors: Marton LT,Goulart RA,Carvalho ACA,Barbalho SM

    更新日期:2019-09-30 00:00:00

  • Peptide-lipid interactions: experiments and applications.

    abstract::The interactions between peptides and lipids are of fundamental importance in the functioning of numerous membrane-mediated cellular processes including antimicrobial peptide action, hormone-receptor interactions, drug bioavailability across the blood-brain barrier and viral fusion processes. Moreover, a major goal of...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms140918758

    authors: Galdiero S,Falanga A,Cantisani M,Vitiello M,Morelli G,Galdiero M

    更新日期:2013-09-12 00:00:00

  • miRNAmotif-A Tool for the Prediction of Pre-miRNA⁻Protein Interactions.

    abstract::MicroRNAs (miRNAs) are short, non-coding post-transcriptional gene regulators. In mammalian cells, mature miRNAs are produced from primary precursors (pri-miRNAs) using canonical protein machinery, which includes Drosha/DGCR8 and Dicer, or the non-canonical mirtron pathway. In plant cells, mature miRNAs are excised fr...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms19124075

    authors: Urbanek-Trzeciak MO,Jaworska E,Krzyzosiak WJ

    更新日期:2018-12-17 00:00:00

  • Integrated RNA-Seq Analysis and Meta-QTLs Mapping Provide Insights into Cold Stress Response in Rice Seedling Roots.

    abstract::Rice (Oryza sativa L.) is a widely cultivated food crop around the world, especially in Asia. However, rice seedlings often suffer from cold stress, which affects their growth and yield. Here, RNA-seq analysis and Meta-QTLs mapping were performed to understand the molecular mechanisms underlying cold tolerance in the ...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms21134615

    authors: Kong W,Zhang C,Qiang Y,Zhong H,Zhao G,Li Y

    更新日期:2020-06-29 00:00:00

  • Identifying the Pathological Domain of Alpha- Synuclein as a Therapeutic for Parkinson's Disease.

    abstract::Alpha-synuclein is considered the major pathological protein associated with Parkinson's disease, but there is still no effective immunotherapy which targets alpha-synuclein. In order to create a safer and more effective therapy against PD, we are targeting an epitope of alpha-synuclein rather than full-length alpha-s...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms20092338

    authors: Shen N,Song G,Yang H,Lin X,Brown B,Hong Y,Cai J,Cao C

    更新日期:2019-05-11 00:00:00

  • Molecular Dynamics Simulations to Investigate How PZM21 Affects the Conformational State of the μ-Opioid Receptor Upon Activation.

    abstract::Opioid analgesics such as morphine have indispensable roles in analgesia. However, morphine use can elicit side effects such as respiratory depression and constipation. It has been reported that G protein-biased agonists as substitutes for classic opioid agonists can alleviate (or even eliminate) these side effects. T...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms21134699

    authors: Zhao Z,Huang T,Li J

    更新日期:2020-07-01 00:00:00

  • Aminolevulinic Acid-Based Tumor Detection and Therapy: Molecular Mechanisms and Strategies for Enhancement.

    abstract::Aminolevulinic acid (ALA) is the first metabolite in the heme biosynthesis pathway in humans. In addition to the end product heme, this pathway also produces other porphyrin metabolites. Protoporphyrin (PpIX) is one heme precursor porphyrin with good fluorescence and photosensitizing activity. Because tumors and other...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章,评审

    doi:10.3390/ijms161025865

    authors: Yang X,Palasuberniam P,Kraus D,Chen B

    更新日期:2015-10-28 00:00:00

  • Characterization of Fluid Biomarkers Reveals Lysosome Dysfunction and Neurodegeneration in Neuronopathic MPS II Patients.

    abstract::Mucopolysaccharidosis type II is a lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase (IDS) and characterized by the accumulation of the primary storage substrate, glycosaminoglycans (GAGs). Understanding central nervous system (CNS) pathophysiology in neuronopathic MPS II (nMPS II) has been hi...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms21155188

    authors: Bhalla A,Ravi R,Fang M,Arguello A,Davis SS,Chiu CL,Blumenfeld JR,Nguyen HN,Earr TK,Wang J,Astarita G,Zhu Y,Fiore D,Scearce-Levie K,Diaz D,Cahan H,Troyer MD,Harris JM,Escolar ML

    更新日期:2020-07-22 00:00:00

  • New Insights into the Structure-Function Relationship of the Endosomal-Type Na+, K+/H+ Antiporter NHX6 from Mulberry (Morus notabilis).

    abstract::The endosomal-type Na+, K+/H+ antiporters (NHXs) play important roles in K+, vesicle pH homeostasis, and protein trafficking in plant. However, the structure governing ion transport mechanism and the key residues related to the structure-function of the endosomal-type NHXs remain unclear. Here, the structure-function ...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms21020428

    authors: Cao B,Xia Z,Liu C,Fan W,Zhang S,Liu Q,Xiang Z,Zhao A

    更新日期:2020-01-09 00:00:00

  • Theoretical and experimental spectroscopic analysis of cyano-substituted styrylpyridine compounds.

    abstract::A combined theoretical and experimental study on the structure, infrared, UV-Vis and 1H NMR data of trans-2-(m-cyanostyryl)pyridine, trans-2-[3-methyl-(m-cyanostyryl)]pyridine and trans-4-(m-cyanostyryl)pyridine is presented. The synthesis was carried out with an efficient Knoevenagel condensation using green chemist...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms14024005

    authors: Castro ME,Percino MJ,Chapela VM,Ceron M,Soriano-Moro G,Lopez-Cruz J,Melendez FJ

    更新日期:2013-02-18 00:00:00

  • Identification of Proteins Modulated in the Date Palm Stem Infested with Red Palm Weevil (Rhynchophorus ferrugineus Oliv.) Using Two Dimensional Differential Gel Electrophoresis and Mass Spectrometry.

    abstract::A state of the art proteomic methodology using Matrix Assisted Laser Desorption/Ionization-Time of Flight (MALDI TOF) has been employed to characterize peptides modulated in the date palm stem subsequent to infestation with red palm weevil (RPW). Our analyses revealed 32 differentially expressed peptides associated wi...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms160819326

    authors: Rasool KG,Khan MA,Aldawood AS,Tufail M,Mukhtar M,Takeda M

    更新日期:2015-08-17 00:00:00

  • Optogenetic Stimulation of Prelimbic Pyramidal Neurons Maintains Fear Memories and Modulates Amygdala Pyramidal Neuron Transcriptome.

    abstract::Fear extinction requires coordinated neural activity within the amygdala and medial prefrontal cortex (mPFC). Any behavior has a transcriptomic signature that is modified by environmental experiences, and specific genes are involved in functional plasticity and synaptic wiring during fear extinction. Here, we investig...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms22020810

    authors: Laricchiuta D,Sciamanna G,Gimenez J,Termine A,Fabrizio C,Caioli S,Balsamo F,Panuccio A,De Bardi M,Saba L,Passarello N,Cutuli D,Mattioni A,Zona C,Orlando V,Petrosini L

    更新日期:2021-01-15 00:00:00

  • Cell Death Conversion under Hypoxic Condition in Tumor Development and Therapy.

    abstract::Hypoxia, which is common during tumor progression, plays important roles in tumor biology. Failure in cell death in response to hypoxia contributes to progression and metastasis of tumors. On the one hand, the metabolic and oxidative stress following hypoxia could lead to cell death by triggering signal cascades, like...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章,评审

    doi:10.3390/ijms161025536

    authors: Qiu Y,Li P,Ji C

    更新日期:2015-10-23 00:00:00

  • LH-induced Transcriptional Regulation of Klf4 Expression in Granulosa Cells Occurs via the cAMP/PKA Pathway and Requires a Putative Sp1 Binding Site.

    abstract::Krüppel-like factor 4 (Klf4) plays an important role in the transition from proliferation to differentiation in a wide variety of cells. Previous studies demonstrated its critical role in the luteal transition of preovulatory granulosa cells (GCs). This study used cultured rat preovulatory GCs to investigate the mecha...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms21197385

    authors: Choi H,Roh J

    更新日期:2020-10-06 00:00:00

  • Dietary Supplementation with Lactobacillus casei Alleviates Lipopolysaccharide-Induced Liver Injury in a Porcine Model.

    abstract::This study aims to determine whether Lactobacillus casei (L. casei) could relieve liver injury in piglets challenged with lipopolysaccharide (LPS). Piglets were randomly allocated into one of the three groups: control, LPS, and L. casei. The control and LPS groups were fed a corn- and soybean meal-based diet, whereas ...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms18122535

    authors: Zhao D,Wu T,Yi D,Wang L,Li P,Zhang J,Hou Y,Wu G

    更新日期:2017-11-26 00:00:00

  • A Novel HPLC-Based Method to Investigate on RNA after Fixation.

    abstract::RNA isolated from fixed and paraffin-embedded tissues is widely used in biomedical research and molecular pathology for diagnosis. In the present study, we have set-up a method based on high performance liquid chromatography (HPLC) to investigate the effects of different fixatives on RNA. By the application of the pre...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms21207540

    authors: Fattorini P,Forzato C,Tierno D,De Martino E,Azzalini E,Canzonieri V,Stanta G,Bonin S

    更新日期:2020-10-13 00:00:00

  • A comparative proteomic analysis of Pinellia ternata leaves exposed to heat stress.

    abstract::Pinellia ternata is an important traditional Chinese medicinal plant. The growth of P. ternata is sensitive to high temperatures. To gain a better understanding of heat stress responses in P. ternata, we performed a comparative proteomic analysis. P. ternata seedlings were subjected to a temperature of 38 °C and sampl...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms141020614

    authors: Zhu Y,Zhu G,Guo Q,Zhu Z,Wang C,Liu Z

    更新日期:2013-10-15 00:00:00

  • Dissection of Molecular Processes and Genetic Architecture Underlying Iron and Zinc Homeostasis for Biofortification: From Model Plants to Common Wheat.

    abstract::The micronutrients iron (Fe) and zinc (Zn) are not only essential for plant survival and proliferation but are crucial for human health. Increasing Fe and Zn levels in edible parts of plants, known as biofortification, is seen a sustainable approach to alleviate micronutrient deficiency in humans. Wheat, as one of the...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章,评审

    doi:10.3390/ijms21239280

    authors: Tong J,Sun M,Wang Y,Zhang Y,Rasheed A,Li M,Xia X,He Z,Hao Y

    更新日期:2020-12-05 00:00:00

  • Experimental and Kinetic Study on Lignin Depolymerization in Water/Formic Acid System.

    abstract::Microwave-assisted depolymerization of black-liquor lignin in formic acid was studied, concentrating on the yield of liquid fractions as bio-oil 1 (mainly aromatic monomers) and bio-oil 2 (mainly aromatic oligomers) and the distribution of the specific compositions. Bio-oil 1 (9.69%) and bio-oil 2 (54.39%) achieved th...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms18102082

    authors: Wang Q,Guan S,Shen D

    更新日期:2017-10-01 00:00:00

  • Chromatin structure following UV-induced DNA damage-repair or death?

    abstract::In eukaryotes, DNA is compacted into a complex structure known as chromatin. The unravelling of DNA is a crucial step in DNA repair, replication, transcription and recombination as this allows access to DNA for these processes. Failure to package DNA into the nucleosome, the individual unit of chromatin, can lead to g...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章,评审

    doi:10.3390/ijms12118063

    authors: Farrell AW,Halliday GM,Lyons JG

    更新日期:2011-01-01 00:00:00

  • Photo-Responsive Supramolecular Micelles for Controlled Drug Release and Improved Chemotherapy.

    abstract::Development of stimuli-responsive supramolecular micelles that enable high levels of well-controlled drug release in cancer cells remains a grand challenge. Here, we encapsulated the antitumor drug doxorubicin (DOX) and pro-photosensitizer 5-aminolevulinic acid (5-ALA) within adenine-functionalized supramolecular mice...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms22010154

    authors: Ilhami FB,Peng KC,Chang YS,Alemayehu YA,Tsai HC,Lai JY,Chiao YH,Kao CY,Cheng CC

    更新日期:2020-12-25 00:00:00

  • Radioprotective Effects of Dermatan Sulfate in a Preclinical Model of Oral Mucositis-Targeting Inflammation, Hypoxia and Junction Proteins without Stimulating Proliferation.

    abstract::Oral mucositis is the most frequently occurring early side effect of head-and-neck cancer radiotherapy. Systemic dermatan sulfate (DS) treatment revealed a significant radioprotective potential in a preclinical model of oral mucositis. This study was initiated to elucidate the mechanistic effects of DS in the same mod...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms19061684

    authors: Gruber S,Arnold M,Cini N,Gernedl V,Hetzendorfer S,Kowald LM,Kuess P,Mayer J,Morava S,Pfaffinger S,Rohorzka A,Dörr W

    更新日期:2018-06-06 00:00:00

  • Melatonin Deficiency Confers Tolerance to Multiple Abiotic Stresses in Rice via Decreased Brassinosteroid Levels.

    abstract::Melatonin has long been recognized as a positive signaling molecule and potent antioxidant in plants, which alleviates damage caused by adverse conditions such as salt, cold, and heat stress. In this study, we found a paradoxical role for melatonin in abiotic stress responses. Suppression of the serotonin N-acetyltran...

    journal_title:International journal of molecular sciences

    pub_type: 杂志文章

    doi:10.3390/ijms20205173

    authors: Hwang OJ,Back K

    更新日期:2019-10-18 00:00:00