Findings from postmortem studies of the brains of Huntington's Disease (HD) patients suggest that transcriptional dysregulation may be an early step in the pathogenesis of HD before symptoms appear. Other studies report transcriptional ...
Aug 26, 2013
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The initial clinical trial of a novel approach to treating amyotrophic lateral sclerosis (ALS) – blocking production of a mutant protein that causes an inherited form of the progressive neurodegererative disease – may ...
Apr 3, 2013
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The dystrophin protein offers critical support to muscle fibers. Mutations affecting dystrophin's expression cause the muscle-wasting disease muscular dystrophy. In Duchenne muscular dystrophy (DMD), these mutations take ...
Jan 30, 2013
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A phase 1 clinical trial published in the British Journal of Clinical Pharmacology found that radavirsen—an antisense oligomer that inhibits the production of certain influenza proteins—is safe and well-tolerated in healthy ...
Sep 20, 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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An innovative therapeutic strategy for reducing the levels of toxic protein fragments associated with Huntington's disease uses a new approach called exon skipping to remove the disease-causing component of the essential ...
Feb 11, 2014
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New classes of therapeutic antisense oligonucleotides can have toxic effects on the liver. A novel machine learning-based approach used to predict the hepatotoxic potential of an antisense drug based on its chemical sequence ...
Aug 27, 2013
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