“Breakthrough Article” in Nucleic Acids Research Presents Data Supporting Full-Scale Clinical Development of Antisense Oligonucleotides (ASOs) for Possible Treatment of Prion Diseases Such As Fatal Familial Insomnia & Creutzfeldt-Jakob Disease

Research teams led by Sonia Vallabh (photo), PhD, whose mother died of the hereditary prion disease fatal familial insomnia, and Eric Minikel, PhD, at Harvard Medical School and The Broad Institute, Holly Kordasiewicz, PhD, at Ionis Pharmaceuticals, and Deborah Cabin, PhD, at McLaughlin Research Institute, have reported the results of preclinical studies of an antisense therapy against different strains of prion disorders, including mechanistic experiments and validation in animal model systems, in an article titled “Prion Protein Lowering is a Disease-modifying Therapy Across Prion Strains, Stages, and Endpoints,” and published online on August 10, 2020 in Nucleic Acids Research. This article was designated by NAR as a “Breakthrough Article,” that is, an article that “describes studies that provide exceptional new insight and understanding into an area of research that will clearly motivate and guide new research opportunities and directions.” “Breakthrough Articles” represent the top papers that NAR receives for publication, and are selected by the Editors based on nominations and subsequent recommendations by the reviewers and editorial board members. Prion diseases are rapidly fatal and currently untreatable neurodegenerative diseases. They include Creutzfeldt-Jakob disease, fatal familial insomnia, and kuru in humans, bovine spongiform encephalitis (BSE) (Mad Cow Disease) in cattle, and scrapie in sheep. These diseases are caused by disruption of the structure of a normal prion protein (PrP). The disrupted PrP is characterized by a beta-sheet structure rather than the alpha-helix structure that characterizes the normal PrP.
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