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Antisense oligonucleotide

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https://www.readbyqxmd.com/read/27907033/normalization-of-patient-identified-plasma-biomarkers-in-smn%C3%AE-7-mice-following-postnatal-smn-restoration
#1
W David Arnold, Sandra Duque, Chitra C Iyer, Phillip Zaworski, Vicki L McGovern, Shannon J Taylor, Katharine M von Herrmann, Dione T Kobayashi, Karen S Chen, Stephen J Kolb, Sergey V Paushkin, Arthur H M Burghes
INTRODUCTION AND OBJECTIVE: Spinal muscular atrophy (SMA) is an autosomal recessive motor neuron disorder. SMA is caused by homozygous loss of the SMN1 gene and retention of the SMN2 gene resulting in reduced levels of full length SMN protein that are insufficient for motor neuron function. Various treatments that restore levels of SMN are currently in clinical trials and biomarkers are needed to determine the response to treatment. Here, we sought to investigate in SMA mice a set of plasma analytes, previously identified in patients with SMA to correlate with motor function...
2016: PloS One
https://www.readbyqxmd.com/read/27904776/microrna-195-suppresses-colorectal-cancer-cells-proliferation-via-targeting-fgf2-and-regulating-wnt-%C3%AE-catenin-pathway
#2
Xianxiang Zhang, Ji Xu, Tao Jiang, Guangwei Liu, Dongsheng Wang, Yun Lu
Colorectal cancer (CRC) is a prevalent cancer with high mortality worldwide. This study was aimed to explore the functional effects of microRNA-195 (miR-195) on CRC cells and the underling mechanism involved. quantitative PCR (qPCR) was performed to monitor the expression of miR-195 in CRC tissues and cell lines. SW480 and SW620 cells were transfected with either miR-195 mimic or antisense oligonucleotides (ASO) of miR-195. Then cell viability, cell cycle and the expressions of CyclinB1, CyclinD1 and Cyclin-Dependent Kinase 2 (CDK2) were respectively detected by 3-(4, 5-dimethyl-2-thiazolyl)-2, 5-diphenyltetrazolium bromide (MTT), flow cytometry, qPCR and Western blot...
2016: American Journal of Cancer Research
https://www.readbyqxmd.com/read/27903030/-novel-therapeutic-approaches-in-inflammatory-bowel-diseases
#3
Raja Atreya, Markus Friedrich Neurath
Increasing knowledge regarding the immunopathogenesis of IBD has led to the development or approval of novel therapeutic agents for the treatment of Crohn's disease or ulcerative colitis. The new substances for example include antibodies against IL-12 / 23, anti-adhesion molecules and antisense oligonucleotides. The increase of therapeutic options will lead to a change of currently used therapeutic algorithms. Biomarkers to ensure a more individualized therapeutic approach are urgently needed to improve an efficient therapy of IBD patients...
November 2016: Deutsche Medizinische Wochenschrift
https://www.readbyqxmd.com/read/27902930/modulating-micrornas-in-cardiac-surgery-patients-novel-therapeutic-opportunities
#4
REVIEW
Giovanni Biglino, Massimo Caputo, Cha Rajakaruna, Gianni Angelini, Eva van Rooij, Costanza Emanueli
This review focuses on microRNAs (miRs) in cardiac surgery, where they are emerging as potential targets for therapeutic intervention as well as novel clinical biomarkers. Identification of the up/down-regulation of specific miRs in defined groups of cardiac surgery patients can lead to the development of novel strategies for targeted treatment in order to maximise therapeutic results and minimise acute, delayed or chronic complications. MiRs could also be involved in determining the outcome independently of complications, for example in relation to myocardial perfusion and fibrosis...
November 27, 2016: Pharmacology & Therapeutics
https://www.readbyqxmd.com/read/27898093/corrigendum-to-from-cryptic-toward-canonical-pre-mrna-splicing-in-pompe-disease-a-pipeline-for-the-development-of-antisense-oligonucleotides
#5
(no author information available yet)
No abstract text is available yet for this article.
November 29, 2016: Molecular Therapy. Nucleic Acids
https://www.readbyqxmd.com/read/27898020/multivalent-aptamers-versatile-tools-for-diagnostic-and-therapeutic-applications
#6
REVIEW
Mariya Vorobyeva, Pavel Vorobjev, Alya Venyaminova
Nucleic acid aptamers generated through an in vitro selection are currently extensively applied as very valuable biomolecular tools thanks to their prominent advantages. Diversity of spatial structures, ease of production through chemical synthesis and a large variety of chemical modifications make aptamers convenient building blocks for the generation of multifunctional constructs. An opportunity to combine different aptamer functionalities with other molecules of interest such as reporter groups, nanoparticles, chemotherapeutic agents, siRNA or antisense oligonucleotides provides a widest range of applications of multivalent aptamers...
November 25, 2016: Molecules: a Journal of Synthetic Chemistry and Natural Product Chemistry
https://www.readbyqxmd.com/read/27896813/transcriptional-and-post-transcriptional-regulation-of-histone-variant-h2a-z-during-sea-urchin-development
#7
Mihai Hajdu, Jasmine Calle, Andrea Puno, Aminat Haruna, César Arenas-Mena
Histone variant H2A.Z promotes chromatin accessibility at transcriptional regulatory elements and is developmentally regulated in metazoans. We characterize the transcriptional and post-transcriptional regulation of H2A.Z in the purple sea urchin Strongylocentrotus purpuratus. H2A.Z depletion by antisense translation-blocking morpholino oligonucleotides during early development causes developmental collapse, in agreement with its previously demonstrated general role in transcriptional multipotency. During H2A...
November 29, 2016: Development, Growth & Differentiation
https://www.readbyqxmd.com/read/27896589/lipid-nanoparticles-loaded-with-an-antisense-oligonucleotide-gapmer-against-bcl-2-for-treatment-of-lung-cancer
#8
Xinwei Cheng, Qibing Liu, Hong Li, Chen Kang, Yang Liu, Tianqi Guo, Ke Shang, Chengyun Yan, Guang Cheng, Robert J Lee
PURPOSE: Bcl-2 is an anti-apoptotic gene that is frequently overexpressed in human cancers. G3139 is an antisense oligonucleotide against bcl-2 that has shown limited efficacy in clinical trials. Here, we report the synthesis of a new antisense oligonucleotide containing additional chemical modifications and its delivery using nanoparticles. METHODS: An oligonucleotide G3139-GAP was synthesized, which has 2'-O-methyl nucleotides at the 5' and 3' ends based on a "gapmer" design...
November 28, 2016: Pharmaceutical Research
https://www.readbyqxmd.com/read/27892730/delivery-of-therapeutic-rna-cleaving-oligodeoxyribonucleotides-deoxyribozymes-from-cell-culture-studies-to-clinical-trials
#9
Alesya A Fokina, Boris P Chelobanov, Masayuki Fujii, Dmitry A Stetsenko
Development of efficient in vivo delivery systems remains a major challenge en route to clinical application of antisense technology, including RNA-cleaving molecules such as deoxyribozymes (DNAzymes). The mechanisms of oligonucleotide uptake and trafficking are clearly dependent on cell type and the type of oligonucleotide analogue. It appears likely that each particular disease target would pose its own specific requirements for a delivery method. Areas covered. In this review we will discuss the available options for DNAzyme delivery in vitro and in vivo, outline various exogenous and endogenous strategies that have been, or are still being, developed and ascertain their applicability with emphasis on those methods that are currently being used in clinical trials...
November 28, 2016: Expert Opinion on Drug Delivery
https://www.readbyqxmd.com/read/27890901/can-colorectal-delivery-technology-provide-a-platform-for-enteral-oligonucleotide-based-therapeutics
#10
Masahiro Murakami, Chie Watanabe
Nucleic acid-based therapeutics including antisense and siRNA oligonucleotides has been expected as an innovative treatment for intractable diseases. Oral drug delivery is the most patient-friendly route of administration but developing an effective delivery system for oligonucleotides remains a major challenge. In this commentary, we discuss the potential benefits of the colorectal route as another platform for the development of oral oligonucleotide therapeutics. The importance of the targeting or the availability of oligonucleotides in targeted tissue is highlighted in contrast to systemic availability, while the liver-targeted enteral siRNA delivery technology that we recently developed is introduced...
2016: Drug Discoveries & Therapeutics
https://www.readbyqxmd.com/read/27890900/in-vitro-and-in-vivo-biophysical-properties-of-oligonucleotides-containing-5-thio-nucleosides
#11
Md Ariful Islam, Reiko Waki, Aki Fujisaka, Kosuke Ramon Ito, Satoshi Obika
Phosphorothioate modification is one of the most widely investigated and promising chemical modifications in oligonucleotide (ON) based therapeutics. Structurally similar 5'-thio or phosphorothiolate-modified nucleotides, in which a 5'-bridging oxygen is replaced with a sulfur atom, are gaining importance for ON-based research. Several reports have been published describing the synthesis of 5'-thio-modified ONs but no detailed in vitro and in vivo data are available. Here, we report the synthesis of 5'-thio-modified 2'-deoxy-5-methylcytidine...
2016: Drug Discoveries & Therapeutics
https://www.readbyqxmd.com/read/27890899/drug-delivery-system-of-therapeutic-oligonucleotides
#12
Yutaro Asami, Kotaro Yoshioka, Kazutaka Nishina, Tetsuya Nagata, Takanori Yokota
Therapeutic oligonucleotides are promising technologies. Nevertheless, improvement of their efficacy is an important issue. Introducing this drug delivery system (DDS) makes for a great enhancement for delivery of oligonucleotides to targeted tissue or cells. The strategy of DDS for therapeutic oligonucleotides is divided into four categories, A) single piece of oligonucleotide, B) oligonucleotide-ligand conjugate, C) oligonucleotide-polymer conjugate, and D) nanoparticle. In this review we will describe those basic concepts, especially for the technology of conjugating ligand...
2016: Drug Discoveries & Therapeutics
https://www.readbyqxmd.com/read/27890825/the-expression-of-chemorepulsive-guidance-receptors-and-the-regenerative-abilities-of-spinal-projecting-neurons-after-spinal-cord-injury
#13
Jie Chen, Cindy Laramore, Michael I Shifman
Spinal cord injury (SCI) in mammals leads to permanent loss of function because axons do not regenerate in the central nervous system (CNS). To date, treatments based on neutralizing inhibitory environmental cues, such as the myelin-associated growth inhibitors and chondroitin sulfate proteoglycans, or on adding neurotrophic factors, have had limited success in enhancing regeneration. Published studies suggested that multiple axon guidance cues (repulsive guidance molecule (RGM) family, semaphorins, ephrins, and netrins) persist in adult animals, and that their expression is upregulated after CNS injury...
November 24, 2016: Neuroscience
https://www.readbyqxmd.com/read/27890673/functional-validation-of-abhd12-mutations-in-the-neurodegenerative-disease-pharc
#14
Angèle Tingaud-Sequeira, Demetrio Raldúa, Julie Lavie, Guilaine Mathieu, Magali Bordier, Anja Knoll-Gellida, Pierre Rambeau, Isabelle Coupry, Michèle André, Eva Malm, Claes Möller, Sten Andreasson, Nanna D Rendtorff, Lisbeth Tranebjærg, Michel Koenig, Didier Lacombe, Cyril Goizet, Patrick J Babin
ABHD12 mutations have been linked to neurodegenerative PHARC (polyneuropathy, hearing loss, ataxia, retinitis pigmentosa, and early-onset cataract), a rare, progressive, autosomal, recessive disease. Although ABHD12 is suspected to play a role in the lysophosphatidylserine and/or endocannabinoid pathways, its precise functional role(s) leading to PHARC disease had not previously been characterized. Cell and zebrafish models were designed to demonstrate the causal link between an identified new missense mutation p...
November 23, 2016: Neurobiology of Disease
https://www.readbyqxmd.com/read/27888137/the-role-of-rhoa-in-retrograde-neuronal-death-and-axon-regeneration-after-spinal-cord-injury
#15
Jianli Hu, Guixin Zhang, William Rodemer, Li-Qing Jin, Michael Shifman, Michael E Selzer
Paralysis following spinal cord injury (SCI) is due to interruption of axons and their failure to regenerate. It has been suggested that the small GTPase RhoA may be an intracellular signaling convergence point for several types of growth-inhibiting extracellular molecules. Even if this is true in vitro, it is not clear from studies in mammalian SCI, whether the effects of RhoA manipulations on axon growth in vivo are due to a RhoA-mediated inhibition of true regeneration or only of collateral sprouting from spared axons, since work on SCI generally is performed with partial injury models...
November 22, 2016: Neurobiology of Disease
https://www.readbyqxmd.com/read/27887908/the-yin-and-yang-of-nucleic-acid-based-therapy-in-the-brain
#16
REVIEW
Stefano Gustincich, Silvia Zucchelli, Antonello Mallamaci
The post-genomic era has unveiled the existence of a large repertory of non-coding RNAs and repetitive elements that play a fundamental role in cellular homeostasis and dysfunction. These may represent unprecedented opportunities to modify gene expression at the right time in the correct space in vivo, providing an almost unlimited reservoir of new potential pharmacological agents. Hijacking their mode of actions, the druggable genome can be extended to regulatory RNAs and DNA elements in a scalable fashion...
November 22, 2016: Progress in Neurobiology
https://www.readbyqxmd.com/read/27885597/method-of-peptide-nucleic-acid-pna-mediated-antisense-inhibition-of-gene-expression-in-campylobacter-jejuni
#17
Euna Oh, Byeonghwa Jeon
Peptide nucleic acid (PNA) is an oligonucleotide mimic that recognizes and binds to nucleic acids. The strong binding affinity of PNA to mRNA coupled with its high sequence specificity enable antisense PNA to selectively inhibit (i.e., knockdown) the protein synthesis of a target gene. This novel technology provides a powerful tool for Campylobacter studies because molecular techniques have been relatively less well-developed for this bacterium as compared to other pathogens, such as Escherichia coli and Salmonella...
2017: Methods in Molecular Biology
https://www.readbyqxmd.com/read/27884961/therapeutic-silencing-of-fat-specific-protein-27-improves-glycemic-control-in-mouse-models-of-obesity-and-insulin-resistance
#18
Cédric Langhi, Noemí Arias, Ananthi Rajamoorthi, Jeannine Basta, Richard G Lee, Ángel Baldán
Obesity is a component of the metabolic syndrome, mechanistically linked to diabetes, fatty liver disease, and cardiovascular disease. Proteins that regulate the metabolic fate of intracellular lipid droplets are potential therapeutic candidates to treat obesity and its related consequences. CIDEC (cell death-inducing DFFA-like effector C), also known in mice as Fsp27 (fat-specific protein 27), is a lipid droplet-associated protein that prevents lipid mobilization and promotes intracellular lipid storage. The consequences of complete loss of FSP27 on hepatic metabolism and on insulin resistance are controversial, as both healthy and deleterious lipodystrophic phenotypes have been reported in Fsp27-/- mice...
November 24, 2016: Journal of Lipid Research
https://www.readbyqxmd.com/read/27883055/gapmer-cellular-internalization-by-macropinocytosis-induces-sequence-specific-gene-silencing-in-human-primary-t-cells
#19
Mobashar Hussain Urf Turabe Fazil, Seow Theng Ong, Madhavi Latha Somaraju Chalasani, Jian Hui Low, Atish Kizhakeyil, Akshay Mamidi, Carey Fang Hui Lim, Graham D Wright, Rajamani Lakshminarayanan, Dermot Kelleher, Navin Kumar Verma
Post-transcriptional gene silencing holds great promise in discovery research for addressing intricate biological questions and as therapeutics. While various gene silencing approaches, such as siRNA and CRISPR-Cas9 techniques, are available, these cannot be effectively applied to "hard-to-transfect" primary T-lymphocytes. The locked nucleic acid-conjugated chimeric antisense oligonucleotide, called "GapmeR", is an emerging new class of gene silencing molecule. Here, we show that GapmeR internalizes into human primary T-cells through macropinocytosis...
November 24, 2016: Scientific Reports
https://www.readbyqxmd.com/read/27879669/synthesis-of-a-morpholino-nucleic-acid-mna-uridine-phosphoramidite-and-exon-skipping-using-mna-2-o-methyl-mixmer-antisense-oligonucleotide
#20
Suxiang Chen, Bao T Le, Kamal Rahimizadeh, Khalil Shaikh, Narinder Mohal, Rakesh N Veedu
In this study, we synthesised a morpholino nucleoside-uridine (MNA-U) phosphoramidite and evaluated the potential of a MNA-modified antisense oligonucleotide (AO) sequences to induce exon 23 skipping in mdx mouse myotubes in vitro towards extending the applicability of morpholino chemistry with other nucleotide monomers. We designed, synthesised, and compared exon skipping efficiencies of 20 mer MNA-modified 2'-O-methyl RNA mixmer AO on a phosphorothioate backbone (MNA/2'-OMePS) to the corresponding fully modified 2'-O-methyl RNA AO (2'-OMePS) as a control...
November 22, 2016: Molecules: a Journal of Synthetic Chemistry and Natural Product Chemistry
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