Therapeutic Oligonucleotides (RSC Biomolecular Sciences)

Protein-Nucleic Acid Interactions: Structural Biology (RSC Biomolecular Sciences)
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Molecular Medicine : An Introduction.

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Jens Kurreck , Cy Aaron Stein. Easy to read, yet comprehensive, this is the perfect introduction into the molecular basis of disease and the novel treatment options that have become available. The authors, Jens Kurreck and Cy Stein, have both long-standing teaching experience on the subject, one from a biologist's angle, the other with a medical background. Together, they have produced a modern textbook for courses in Molecular Medicine that incorporates modules from immunology to signaling, from virology to gene therapy, and the latest development in personalized medicine.

Genetic Disorders. Dmytro Honcharenko 1 ,. Malgorzata Honcharenko 1 ,. Full text PDF Related articles.

Abstract In vivo bioavailability and delivery of nucleic acids to the site of action is a severe limitation in oligonucleotide ON therapeutics. Related articles Based on techniques. Sinebe et al. Development of therapeutic-grade small interfering RNAs by chemical engineering. Front Genet ; Improved silencing properties using small internally segmented interfering RNAs. Efficient regulation of VEGF expression by promoter-targeted lentiviral shRNAs based on epigenetic mechanism: a novel example of epigenetherapy.

Circ Res ;— RNA duplexes with abasic substitutions are potent and allele-selective inhibitors of huntingtin and ataxin-3 expression. Allele-selective inhibition of mutant atrophin-1 expression by duplex and single-stranded RNAs. Ligands of the asialoglycoprotein receptor for targeted gene delivery, part 1: Synthesis of and binding studies with biotinylated cluster glycosides containing N-acetylgalactosamine.

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Beal, and Cynthia J. Molecules , 21 7 , Analytical Chemistry , 81 9 , Berkovitch, Webster L. The ability of locked nucleic acid oligonucleotides to pre-structure the double helix: A molecular simulation and binding study.

Glycoconj J ;— Solid-phase synthesis of oligonucleotide conjugates useful for delivery and targeting of potential nucleic acid therapeutics. Bioconjug Chem ;— Hepatocyte-specific delivery of siRNAs conjugated to novel non-nucleosidic trivalent N-acetylgalactosamine elicits robust gene silencing in vivo. Chembiochem ;— Cellular uptake and intracellular trafficking of oligonucleotides: implications for oligonucleotide pharmacology. Nucleic Acid Ther ;— Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes. Exosomes for targeted siRNA delivery across biological barriers.

Adv Drug Deliv Rev ;— Delivery of siRNA to the brain using a combination of nose-to-brain delivery and cell-penetrating peptide-modified nano-micelles. Biomaterials ;— Smart functional nucleic acid chimeras: enabling tissue specific RNA targeting therapy. RNA Biol ;— Complexity in the therapeutic delivery of RNAi medicines: an analytical challenge. Expert Opin Drug Deliv ;— Going beyond the liver: progress and challenges of targeted delivery of siRNA therapeutics. J Control Release ;— Fomiversen sodium approved to treat CMV retinitis. J Am Pharm Assoc Wash ;— Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial.

Lancet ;— Reciprocal effects of micro-RNA on expression of heme oxygenase-1 and hepatitis C virus genes in human hepatocytes. Gastroenterology ;— Int J Oncol ;— Potential of anti-cancer therapy based on anti-miR oligonucleotides in glioma and brain tumours. Chem Biol Drug Des ;— Alzheimers Res Ther ; Curr Mol Med ;— Nat Rev Drug Discov ;— Molecular basis of muscular dystrophies.

Muscle Nerve ;— Antisense oligonucleotides for the treatment of spinal muscular atrophy.

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Curr Opin Mol Ther ;— Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model. J Clin Invest ;— Splice-correction strategies for treatment of x-linked agammaglobulinemia. Curr Allergy Asthma Rep ; The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion. Am J Hum Genet ;— Clinical trials using antisense oligonucleotides in Duchenne muscular dystrophy. Antisense oligonucleotide therapeutics for iron-sulphur cluster deficiency myopathy.

Neuromuscul Disord ;— Antisense oligonucleotide corrects splice abnormality in hereditary myopathy with lactic acidosis. Gene ;— Nucleic Acids Res Induction of splice correction by cell-penetrating peptide nucleic acids. J Gene Med ;— Correlating in vitro splice switching activity with systemic in vivo delivery using novel ZEN-modified oligonucleotides.

Mol Ther Nucleic Acids ;3:e Antitumorigenic potential of STAT3 alternative splicing modulation. Br J Cancer ;— J Mol Cell Cardiol ;— Splicing of a critical exon of human survival motor neuron is regulated by a unique silencer element located in the last intron. Mol Cell Biol ;— Bifunctional antisense oligonucleotides provide a trans-acting splicing enhancer that stimulates SMN2 gene expression in patient fibroblasts.

Correction of disease-associated exon skipping by synthetic exon-specific activators. Nat Struct Biol ;— Redirecting splicing with bifunctional oligonucleotides. Nucleic Acids Res ;e Skipping multiple exons of dystrophin transcripts using cocktail antisense oligonucleotides. Bispecific antisense oligonucleotides having binding sites directed against an autocrine regulated growth pathway and bcl-2 for the treatment of prostate tumors. Med Oncol ;— Design and application of bispecific splice-switching oligonucleotides. Cell-type-specific, aptamer-functionalized agents for targeted disease therapy.

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Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease. Induction of tumour immunity by targeted inhibition of nonsense-mediated mRNA decay. Functional in vivo delivery of multiplexed anti-HIV-1 siRNAs via a chemically synthesized aptamer with a sticky bridge. Mol Ther ;— Prostate-targeted radiosensitization via aptamer-shRNA chimeras in human tumor xenografts.

Aptamer-mediated delivery of splice-switching oligonucleotides to the nuclei of cancer cells. Multifunctional nucleic acids for tumor cell treatment. Nucleic Acid Therapeutics ;— Helix formation by guanylic acid. Effects of sequence of thioated oligonucleotides on cultured human mammary epithelial cells.

Therapeutic Oligonucleotides (RSC Biomolecular Sciences)

Antiproliferative activity of G-quartet-forming oligonucleotides with backbone and sugar modifications. Triplex-forming oligonucleotide-orthophenanthroline conjugates for efficient targeted genome modification. Gene regulation by antisense transcription. Nat Rev Genet ;— Sequence-selective targeting of duplex DNA by peptide nucleic acids.

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Purine twisted-intercalating nucleic acids: a new class of anti-gene molecules resistant to potassium-induced aggregation. Triplex-forming oligonucleotides targeting c-MYC potentiate the anti-tumor activity of gemcitabine in a mouse model of human cancer.

RSC Biomolecular Sciences

Mol Carcinog ;— Biological activity and biotechnological aspects of peptide nucleic acid. Superior duplex DNA strand invasion by acridine conjugated peptide nucleic acids. Biomol Eng ;— Biochim Biophys Acta ;— Recognition of chromosomal DNA inside cells by locked nucleic acids. Targeted correction of a thalassemia-associated beta-globin mutation induced by pseudo-complementary peptide nucleic acids.

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Cellular uptake and intracellular trafficking of oligonucleotides. Google Scholar. RNA G-quadruplex as supramolecular carrier for cancer-selective delivery. The study of genes and signal transduction pathways involved in mustard lung injury: A gene therapy approach. Sara C. Humphreys , Mai B. Thayer , Iain D. Campuzano , Chawita Netirojjanakul , and Brooke M. Arylation Chemistry for Bioconjugation. The ability of locked nucleic acid oligonucleotides to pre-structure the double helix: A molecular simulation and binding study.

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Michael J. Gait , Andrey A. Application of ion pair chromatography coupled with mass spectrometry to assess antisense oligonucleotides concentrations in living cells. Therapeutic Oligonucleotides: State of the Art. Synthesis of Nucleobase-Functionalized Morpholino Monomers. Chemical Development of Therapeutic Oligonucleotides.

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Non-coding RNA in C9orfrelated amyotrophic lateral sclerosis and frontotemporal dementia: A perfect storm of dysfunction.