Search Results - "Wancewicz, Edward"
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Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis
Published in Neuron (Cambridge, Mass.) (21-06-2012)“…The primary cause of Huntington's disease (HD) is expression of huntingtin with a polyglutamine expansion. Despite an absence of consensus on the mechanism(s)…”
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Long pre-mRNA depletion and RNA missplicing contribute to neuronal vulnerability from loss of TDP-43
Published in Nature neuroscience (01-04-2011)“…The authors identify brain RNA targets for TDP-43, a RNA binding protein linked to ALS. RNAs derived from genes with very long introns were more affected by…”
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A modular analysis of microglia gene expression, insights into the aged phenotype
Published in BMC genomics (28-02-2019)“…Microglia are multifunctional cells that are key players in brain development and homeostasis. Recent years have seen tremendous growth in our understanding of…”
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Short Antisense Oligonucleotides with Novel 2′−4′ Conformationaly Restricted Nucleoside Analogues Show Improved Potency without Increased Toxicity in Animals
Published in Journal of medicinal chemistry (08-01-2009)“…The potency of second generation antisense oligonucleotides (ASOs) in animals was increased 3- to 5 -fold (ED50 ≈ 2−5 mg/kg) without producing hepatotoxicity,…”
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Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals
Published in Nucleic acids research (01-01-2007)“…A series of antisense oligonucleotides (ASOs) containing either 2'-O-methoxyethylribose (MOE) or locked nucleic acid (LNA) modifications were designed to…”
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Co-Administration of an Excipient Oligonucleotide Helps Delineate Pathways of Productive and Nonproductive Uptake of Phosphorothioate Antisense Oligonucleotides in the Liver
Published in Nucleic acid therapeutics (01-08-2017)“…Phosphorothioate (PS) modified antisense oligonucleotides (ASOs) have progressed rapidly in the clinic for treating a variety of disease indications. We…”
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Comparing In Vitro and In Vivo Activity of 2′-O-[2-(Methylamino)-2-oxoethyl]- and 2′-O-Methoxyethyl-Modified Antisense Oligonucleotides
Published in Journal of medicinal chemistry (08-05-2008)“…A number of 2′-O-modified antisense oligonucleotides have been reported for their potential use in oligonucleotide-based therapeutics. To date, most of the in…”
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Peptide Nucleic Acids Conjugated to Short Basic Peptides Show Improved Pharmacokinetics and Antisense Activity in Adipose Tissue
Published in Journal of medicinal chemistry (27-05-2010)“…A peptide nucleic acid (PNA) targeting a splice junction of the murine PTEN primary transcript was covalently conjugated to various basic peptides. When…”
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Inhibition of hepatic endothelial E-selectin expression by C-raf antisense oligonucleotides blocks colorectal carcinoma liver metastasis
Published in Cancer research (Chicago, Ill.) (01-10-2002)“…Cytokine-dependent induction of E-selectin expression is mediated through cooperative signaling involving the Ras/Raf/mitogen-activated protein kinase pathway…”
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10
Identification and functional validation of PNAs that inhibit murine CD40 expression by redirection of splicing
Published in Nucleic acids research (2004)“…Cognate recognition between the CD40 receptor and its ligand, CD154, is thought to play a central role in the initiation and propagation of immune responses…”
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Disruption of WT1 gene expression and exon 5 splicing following cytotoxic drug treatment: Antisense down-regulation of exon 5 alters target gene expression and inhibits cell survival
Published in Molecular cancer therapeutics (01-11-2004)“…Deregulated expression of the Wilms' tumor gene ( WT1 ) has been implicated in the maintenance of a malignant phenotype in leukemias and a wide range of solid…”
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Structure−Activity Relationship Study on a Simple Cationic Peptide Motif for Cellular Delivery of Antisense Peptide Nucleic Acid
Published in Journal of medicinal chemistry (20-10-2005)“…Improving cellular uptake and biodistribution remains one of the major obstacles for a successful and broad application of peptide nucleic acids (PNAs) as…”
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Evaluation of Basic Amphipathic Peptides for Cellular Delivery of Antisense Peptide Nucleic Acids
Published in Journal of medicinal chemistry (20-04-2006)“…Cellular permeation peptides have been used successfully for the delivery of a variety of cargoes across cellular membranes, including large hydrophilic…”
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Inhibition of telomerase by 2'-O-(2-methoxyethyl) RNA oligomers: effect of length, phosphorothioate substitution and time inside cells
Published in Nucleic acids research (15-04-2001)“…2'-O-(2-methoxyethyl) (2'-MOE) RNA possesses favorable pharmocokinetic properties that make it a promising option for the design of oligonucleotide drugs…”
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15
Oncogenic Homeodomain Transcription Factor E2A-Pbx1 Activates a Novel WNT Gene in Pre-B Acute Lymphoblastoid Leukemia
Published in Proceedings of the National Academy of Sciences - PNAS (28-09-1999)“…A large fraction of pediatric pre-B acute lymphoblastoid leukemias (ALL) consistently contain a t(1;19) chromosomal translocation. The t(1;19) translocation…”
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Intravitreous anti-raf-1 kinase antisense oligonucleotide as an angioinhibitory agent in porcine preretinal neovascularization
Published in Current eye research (2003)“…Purpose. To test the efficacy of a synthetic antisense oligonucleotide inhibitor of an intracellular signal transduction protein, C-raf-1 kinase, as an…”
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Divergent roles of ALS-linked proteins FUS/TLS and TDP-43 intersect in processing long pre-mRNAs
Published in Nature neuroscience (01-11-2012)“…In this study, the authors compare the RNA binding patterns of FUS/TLS and TDP-43. Although these proteins regulate the processing of mostly distinct gene…”
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P3‐327: Therapeutic suppression of tau kinases in the CNS with antisense oligonucleotides
Published in Alzheimer's & dementia (01-07-2012)Get full text
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Therapeutic suppression of tau kinases in the CNS with antisense oligonucleotides
Published in Alzheimer's & dementia (01-07-2012)Get full text
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Protein kinase C inhibitors block the enhanced expression of intercellular adhesion molecule-1 on endothelial cells activated by interleukin-1, lipopolysaccharide and tumor necrosis factor
Published in Biochemical and biophysical research communications (15-11-1990)“…Interleukin 1 (IL-1), bacterial lipopolysaccharide (LPS) and tumor necrosis factor (TNF alpha) enhance the adherence properties of endothelial cells (EC) for…”
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