Designing new therapeutic DNA aptamers with diverse side chains can improve their ability to interact with targets, and a new study describes characteristics of these side chains that may determine how long the aptamers remain ...
Nov 29, 2017
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A team of leading European clinicians and scientists presents a unique perspective on how to move forward in the development of exon skipping therapies to treat the severe muscle-wasting disease Duchenne Muscular Dystrophy ...
Oct 16, 2017
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Stanley T. Crooke, MD, PhD, CEO of Ionis Pharmaceuticals and recipient of the 2016 Lifetime Achievement Award from the Oligonucleotide Therapeutic Society presents a detailed look at the mechanisms that underlie antisense ...
Feb 2, 2017
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Combining the therapeutic potential and advantages of existing oligonucleotide-based approaches to turn off disease-related genes, a type of single-stranded silencing RNAs (ss-siRNAs) has shown significantly improved potency ...
May 12, 2016
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The complement system, the human body's first line of defense against blood-borne intruders, is blamed for infusion-related reactions to nanomedicines, but the conventional models used to predict the risk of cardiopulmonary ...
Jan 28, 2016
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Researchers from Isis Pharmaceuticals (Carlsbad, CA) and Prysis Biotechnologies (Pudong, Shanghai, China) have demonstrated proof-of-concept for using a sense oligonucleotide to undo the effects of an antisense drug, an antithrombotic ...
Oct 13, 2015
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Researchers have demonstrated the feasibility of delivering an RNA that encodes for the protein alpha-1-antitrypsin (AAT)—which is missing or nonfunctional in the genetic disorder AAT deficiency—into cells in the laboratory, ...
Jul 27, 2015
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Researchers have shown that a novel delivery strategy can efficiently introduce a functional microRNA that has anti-cancer and angiogenic activities into two different types of cells—breast cancer cells to inhibit tumor ...
Jun 3, 2015
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An experimental single-stranded oligonucleotide-based drug, MGN1703, comprised only of natural DNA components, stimulates the human immune system to fight infections and attack cancer cells without causing the harmful side ...
Apr 7, 2015
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The two U.S. FDA approved oligonucleotide-based drugs on the market both have a modified chemical backbone made of phosphorothioates. The therapeutic advantages of the phosphorothioate group and the new types of gene expression-regulation ...
Nov 13, 2014
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