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Antisense oligonucleotide

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https://www.readbyqxmd.com/read/29473733/%C3%AE-l-threose-nucleic-acids-as-biocompatible-antisense-oligonucleotides-for-suppressing-gene-expression-in-living-cells
#1
Ling Sum Liu, Hoi Man Leung, Dick Yan Tam, Tsz Wan Lo, Sze Wing Wong, Pik Kwan Lo
Due to the chemical simplicity of α- L-Threose nucleic acid (TNA) and its ability to exchange genetic information between itself and RNA, it has been raised significant interests in TNA as RNA ancestor. We herein explore the biological properties and evaluate the potency of sequence-designed TNA polymers to suppress gene expression in living environments. We found that sequence-specific TNA macromolecules exhibit strong affinity and specificity towards the complementary RNA targets, are highly biocompatible and non-toxic in living cell system, and readily enter a number of cell lines without using transfecting agents...
February 23, 2018: ACS Applied Materials & Interfaces
https://www.readbyqxmd.com/read/29467310/whole-body-but-not-hepatic-knockdown-of-chemerin-by-antisense-oligonucleotide-decreases-blood-pressure-in-rats
#2
David J Ferland, Bridget Seitz, Emma S Darios, Janice M Thompson, Steve T Yeh, Adam E Mullick, Stephanie W Watts
Chemerin is an inflammatory adipokine positively associated with hypertension and obesity. The majority of chemerin derives from the liver and adipose tissue, however, their individual contributions to blood pressure are unknown. We began studying chemerin in the normal rat using antisense oligonucleotides (ASO) with whole-body activity (Gen 2.5 chemerin ASO) or liver-restricted activity (GalNAc chemerin ASO). We hypothesized that in normotensive male SD rats, circulating chemerin is predominately liver-derived and regulates blood pressure...
February 21, 2018: Journal of Pharmacology and Experimental Therapeutics
https://www.readbyqxmd.com/read/29466448/a-dystrophic-duchenne-mouse-model-for-testing-human-antisense-oligonucleotides
#3
Marcel Veltrop, Laura van Vliet, Margriet Hulsker, Jill Claassens, Conny Brouwers, Cor Breukel, Jos van der Kaa, Margot M Linssen, Johan T den Dunnen, Sjef Verbeek, Annemieke Aartsma-Rus, Maaike van Putten
Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disease generally caused by reading frame disrupting mutations in the DMD gene resulting in loss of functional dystrophin protein. The reading frame can be restored by antisense oligonucleotide (AON)-mediated exon skipping, allowing production of internally deleted, but partially functional dystrophin proteins as found in the less severe Becker muscular dystrophy. Due to genetic variation between species, mouse models with mutations in the murine genes are of limited use to test and further optimize human specific AONs in vivo...
2018: PloS One
https://www.readbyqxmd.com/read/29463471/il-4-receptor-alpha-signaling-through-macrophages-differentially-regulates-liver-fibrosis-progression-and-reversal
#4
Shih-Yen Weng, Xiaoyu Wang, Santosh Vijayan, Yilang Tang, Yong Ook Kim, Kornelius Padberg, Tommy Regen, Olena Molokanova, Tao Chen, Tobias Bopp, Hansjörg Schild, Frank Brombacher, Jeff R Crosby, Michael L McCaleb, Ari Waisman, Ernesto Bockamp, Detlef Schuppan
Chronic hepatitis leads to liver fibrosis and cirrhosis. Cirrhosis is a major cause of worldwide morbidity and mortality. Macrophages play a key role in fibrosis progression and reversal. However, the signals that determine fibrogenic vs fibrolytic macrophage function remain ill defined. We studied the role of interleukin-4 receptor α (IL-4Rα), a potential central switch of macrophage polarization, in liver fibrosis progression and reversal. We demonstrate that inflammatory monocyte infiltration and liver fibrogenesis were suppressed in general IL-4Rα-/- as well as in macrophage-specific IL-4Rα-/- (IL-4RαΔLysM ) mice...
February 17, 2018: EBioMedicine
https://www.readbyqxmd.com/read/29461008/propofol-exposure-during-early-gestation-impairs-learning-and-memory-in-rat-offspring-by-inhibiting-the-acetylation-of-histone
#5
Jiamei Lin, Shengqiang Wang, Yunlin Feng, Weihong Zhao, Weilu Zhao, Foquan Luo, Namin Feng
Propofol is widely used in clinical practice, including non-obstetric surgery in pregnant women. Previously, we found that propofol anaesthesia in maternal rats during the third trimester (E18) caused learning and memory impairment to the offspring rats, but how about the exposure during early pregnancy and the underlying mechanisms? Histone acetylation plays an important role in synaptic plasticity. In this study, propofol was administered to the pregnant rats in the early pregnancy (E7). The learning and memory function of the offspring were tested by Morris water maze (MWM) test on post-natal day 30...
February 20, 2018: Journal of Cellular and Molecular Medicine
https://www.readbyqxmd.com/read/29459241/the-atherogenic-dyslipidemia-complex-and-novel-approaches-to-cardiovascular-disease-prevention-in-diabetes
#6
REVIEW
Priska Stahel, Changting Xiao, Robert A Hegele, Gary F Lewis
Despite the effectiveness of low-density lipoprotein (LDL)-lowering strategies for the treatment of diabetic dyslipidemia, significant residual risk of atherosclerotic cardiovascular disease remains. Residual risk might in part be explained by lipid abnormalities that go beyond LDL cholesterol elevation, collectively termed the "atherogenic dyslipidemia complex (ADC)," consisting of hypertriglyceridemia, elevated small dense LDL particles, reduced high-density lipoprotein cholesterol, and high-density lipoprotein particle numbers, increased remnant lipoproteins, and postprandial hyperlipidemia...
December 15, 2017: Canadian Journal of Cardiology
https://www.readbyqxmd.com/read/29455156/antisense-oligonucleotides-and-other-genetic-therapies-made-simple
#7
EDITORIAL
Alexander M Rossor, Mary M Reilly, James M Sleigh
Many genetic neurological diseases result from the dysfunction of single proteins. Genetic therapies aim to modify these disease-associated proteins by targeting the RNA and DNA precursors. This review provides a brief overview of the main types of genetic therapies, with a focus on antisense oligonucleotides (ASOs) and RNA interference (RNAi). We use examples of new genetic therapies for spinal muscular atrophy, Duchenne muscular dystrophy and familial amyloid polyneuropathy to highlight the different mechanisms of action of ASOs and RNAi...
February 17, 2018: Practical Neurology
https://www.readbyqxmd.com/read/29452686/technological-advances-and-changing-indications-for-lumbar-puncture-in-neurological-disorders
#8
REVIEW
Joost M Costerus, Matthijs C Brouwer, Diederik van de Beek
Technological advances have changed the indications for and the way in which lumbar puncture is done. Suspected CNS infection remains the most common indication for lumbar puncture, but new molecular techniques have broadened CSF analysis indications, such as the determination of neuronal autoantibodies in autoimmune encephalitis. New screening techniques have increased sensitvity for pathogen detection and can be used to identify pathogens that were previously unknown to cause CNS infections. Evidence suggests that potential treatments for neurodegenerative diseases, such as Alzheimer's disease, will rely on early detection of the disease with the use of CSF biomarkers...
March 2018: Lancet Neurology
https://www.readbyqxmd.com/read/29446733/current-understanding-of-inflammatory-responses-in-acute-kidney-injury
#9
Chao Hu, Yue Sheng, Zhijian Qian
Acute kidney injury has been a tough complex with increased mortality and morbidity. Inflammatory responses, including innate and adaptive immune responses, involve in the initiation and development of acute kidney injury, especially under the ischemic circumstances. Tubular cells and distinct immune cell subgroups play a critical role in the pathogenesis of inflammation. Current gene therapies show their benefits in renal repair. Here, we reviewed the renal inflammatory infiltration, inflammatory mediators, oxidative stress and potential signaling pathways, which give rise to the kidney diseases, in the mechanism of acute kidney injury...
February 13, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29443664/nusinersen-versus-sham-control-in-later-onset-spinal-muscular-atrophy
#10
Eugenio Mercuri, Basil T Darras, Claudia A Chiriboga, John W Day, Craig Campbell, Anne M Connolly, Susan T Iannaccone, Janbernd Kirschner, Nancy L Kuntz, Kayoko Saito, Perry B Shieh, Már Tulinius, Elena S Mazzone, Jacqueline Montes, Kathie M Bishop, Qingqing Yang, Richard Foster, Sarah Gheuens, C Frank Bennett, Wildon Farwell, Eugene Schneider, Darryl C De Vivo, Richard S Finkel
BACKGROUND: Nusinersen is an antisense oligonucleotide drug that modulates pre-messenger RNA splicing of the survival motor neuron 2 ( SMN2) gene. It has been developed for the treatment of spinal muscular atrophy (SMA). METHODS: We conducted a multicenter, double-blind, sham-controlled, phase 3 trial of nusinersen in 126 children with SMA who had symptom onset after 6 months of age. The children were randomly assigned, in a 2:1 ratio, to undergo intrathecal administration of nusinersen at a dose of 12 mg (nusinersen group) or a sham procedure (control group) on days 1, 29, 85, and 274...
February 15, 2018: New England Journal of Medicine
https://www.readbyqxmd.com/read/29441982/microrna-17-3p-promotes-keratinocyte-cells-growth-and-metastasis-via-targeting-myot-and-regulating-notch1-nf-%C3%AE%C2%BAb-pathways
#11
Hongshan Yan, Kunxiu Song, Guohui Zhang
Wound healing is a fundamental biological process to restore skin integrity. The role of microRNAs (miRNAs) during this process remains elusive. Thus, our study aimed to investigate the biological functions and its molecular mechanisms of miR-17-3p in cutaneous wound healing. Human keratinocyte cell line HaCaT was transfected with miR-17-3p mimic, antisense oligonucleotides (ASO)-miR-17-3p and corresponding controls respectively. After transfection, MTT, flow cytometry, qRT-PCR and western blot were performed to analyze cell viability, colony-formation and cell cycle...
September 1, 2017: Die Pharmazie
https://www.readbyqxmd.com/read/29438061/inhibiting-mirna-function-by-antisense-oligonucleotides-in-cultured-mammalian-cells
#12
Chengjian Li, Phillip D Zamore
Methods for transfecting antisense oligonucleotides (ASOs) into mammalian cells for suppression of miRNA function are described in this protocol. The effects of ASOs on the miRNA are evaluated by measuring the protein level of the putative miRNA target or the activity of a reporter bearing a 3' untranslated region (3' UTR) derived from the putative miRNA target.
February 1, 2018: Cold Spring Harbor Protocols
https://www.readbyqxmd.com/read/29438060/preparation-of-antisense-oligonucleotides-to-inhibit-mirna-function
#13
Chengjian Li, Phillip D Zamore
This protocol is used to design antisense oligonucleotides (ASOs) for specific inhibition of miRNA function in cultured cells. The incorporation of 2'- O -methyl-modifications in ASOs enhances their potency and resistance to degradation, whereas 3'-terminal cholesterol-conjugation facilitates delivery of ASOs into cells.
February 1, 2018: Cold Spring Harbor Protocols
https://www.readbyqxmd.com/read/29437530/endosomal-escape-of-asos-internalized-by-stabilin-receptors-is-regulated-by-rab5c-and-eea1-during-endosomal-maturation
#14
Colton M Miller, W Brad Wan, Punit P Seth, Edward N Harris
Second-generation (Gen 2) Antisense oligonucleotides (ASOs) show increased nuclease stability and affinity for their RNA targets, which has translated to improved potency and therapeutic index in the clinic. Gen 2 ASOs are typically modified using the phosphorothioate (PS) backbone modification, which enhances ASO interactions with plasma, cell surface, and intracellular proteins. This facilitates ASO distribution to peripheral tissues and also promotes cellular uptake after injection into animals. Previous work identified that Stabilin receptors specifically internalize PS-ASOs in the sinusoidal endothelial cells of the liver and the spleen...
February 13, 2018: Nucleic Acid Therapeutics
https://www.readbyqxmd.com/read/29434670/advances-in-spinal-muscular-atrophy-therapeutics
#15
REVIEW
Valeria Parente, Stefania Corti
Spinal muscular atrophy (SMA) is a progressive, recessively inherited neuromuscular disease, characterized by the degeneration of lower motor neurons in the spinal cord and brainstem, which leads to weakness and muscle atrophy. SMA currently represents the most common genetic cause of infant death. SMA is caused by the lack of survival motor neuron (SMN) protein due to mutations, which are often deletions, in the SMN1 gene. In the absence of treatments able to modify the disease course, a considerable burden falls on patients and their families...
2018: Therapeutic Advances in Neurological Disorders
https://www.readbyqxmd.com/read/29425080/receptor-mediated-uptake-of-phosphorothioate-antisense-oligonucleotides-in-different-cell-types-of-the-liver
#16
Colton M Miller, Michael Tanowitz, Aaron J Donner, Thazha P Prakash, Eric E Swayze, Edward N Harris, Punit P Seth
Oligonucleotide therapeutics have emerged as a third distinct platform for drug discovery within the pharmaceutical industry. Five oligonucleotide-based drugs have been approved by the US FDA and over 100 oligonucleotides drugs are currently at different stages of human trials. Several of these oligonucleotide drugs are modified using the phosphorothioate (PS) backbone modification where one of the nonbridging oxygen atoms of the phosphodiester linkage is replaced with sulfur. In this review, we summarize our knowledge on receptor-mediated uptake of PS antisense oligonucleotides (ASOs) within different cell types of the liver-a privileged organ for the discovery of oligonucleotide-based therapeutics...
February 9, 2018: Nucleic Acid Therapeutics
https://www.readbyqxmd.com/read/29423057/interleukin-34-sustains-pro-tumorigenic-signals-in-colon-cancer-tissue
#17
Eleonora Franzè, Vicenzo Dinallo, Angela Rizzo, Martina Di Giovangiulio, Gerolamo Bevivino, Carmine Stolfi, Flavio Caprioli, Alfredo Colantoni, Angela Ortenzi, Antonio Di Grazia, Giuseppe Sica, Pier Paolo Sileri, Piero Rossi, Giovanni Monteleone
Interleukin-34 (IL-34), a cytokine produced by a wide range of cells, binds to the macrophage colony-stimulating factor receptor (M-CSFR-1) and receptor-type protein-tyrosine phosphatase zeta (PTP-z) and controls myeloid cell differentiation, proliferation and survival. various types of cancers over-express IL-34 but the role of the cytokine in colorectal cancer (CRC) remains unknown. We here investigated the expression and functional role of IL-34 in CRC. A more pronounced expression of IL-34 was seen in CRC samples as compared to matched normal/benign colonic samples and this occurred at both RNA and protein level...
January 9, 2018: Oncotarget
https://www.readbyqxmd.com/read/29422644/advances-in-therapy-for-spinal-muscular-atrophy-promises-and-challenges
#18
REVIEW
Ewout J N Groen, Kevin Talbot, Thomas H Gillingwater
Spinal muscular atrophy (SMA) is a devastating motor neuron disease that predominantly affects children and represents the most common cause of hereditary infant mortality. The condition results from deleterious variants in SMN1, which lead to depletion of the survival motor neuron protein (SMN). Now, 20 years after the discovery of this genetic defect, a major milestone in SMA and motor neuron disease research has been reached with the approval of the first disease-modifying therapy for SMA by US and European authorities - the antisense oligonucleotide nusinersen...
February 9, 2018: Nature Reviews. Neurology
https://www.readbyqxmd.com/read/29417937/advances-in-lipid-lowering-therapy-through-gene-silencing-technologies
#19
REVIEW
Børge G Nordestgaard, Stephen J Nicholls, Anne Langsted, Kausik K Ray, Anne Tybjærg-Hansen
New treatment opportunities are emerging in the field of lipid-lowering therapy through gene-silencing approaches. Both antisense oligonucleotide inhibition and small interfering RNA technology aim to degrade gene mRNA transcripts to reduce protein production and plasma lipoprotein levels. Elevated levels of LDL, remnant lipoproteins, and lipoprotein(a) all cause cardiovascular disease, whereas elevated levels of triglyceride-rich lipoproteins in some patients can cause acute pancreatitis. The levels of each of these lipoproteins can be reduced using gene-silencing therapies by targeting proteins that have an important role in lipoprotein production or removal (for example, the protein products of ANGPTL3, APOB, APOC3, LPA, and PCSK9)...
February 8, 2018: Nature Reviews. Cardiology
https://www.readbyqxmd.com/read/29415514/covalent-strategies-for-targeting-messenger-and-non-coding-rnas-an-updated-review-on-sirna-mirna-and-antimir-conjugates
#20
REVIEW
Santiago Grijalvo, Adele Alagia, Andreia F Jorge, Ramon Eritja
Oligonucleotide-based therapy has become an alternative to classical approaches in the search of novel therapeutics involving gene-related diseases. Several mechanisms have been described in which demonstrate the pivotal role of oligonucleotide for modulating gene expression. Antisense oligonucleotides (ASOs) and more recently siRNAs and miRNAs have made important contributions either in reducing aberrant protein levels by sequence-specific targeting messenger RNAs (mRNAs) or restoring the anomalous levels of non-coding RNAs (ncRNAs) that are involved in a good number of diseases including cancer...
February 6, 2018: Genes
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