Short antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats

Magdalena Derbis; Emre Kul; Daria Niewiadomska; Michał Sekrecki; Agnieszka Piasecka; Katarzyna Taylor; Renate K. Hukema; Oliver Stork; Krzysztof Sobczak

doi:10.1038/s41467-021-21021-w

Nature Communications (Feb 2021)

Short antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats

Magdalena Derbis,
Emre Kul,
Daria Niewiadomska,
Michał Sekrecki,
Agnieszka Piasecka,
Katarzyna Taylor,
Renate K. Hukema,
Oliver Stork,
Krzysztof Sobczak

Affiliations

Magdalena Derbis: Department of Gene Expression, Institute of Molecular Biology and Biotechnology, Adam Mickiewicz University, Uniwersytetu Poznanskiego 6
Emre Kul: Department of Genetics and Molecular Neurobiology, Institute of Biology, Otto von Guericke University
Daria Niewiadomska: Department of Gene Expression, Institute of Molecular Biology and Biotechnology, Adam Mickiewicz University, Uniwersytetu Poznanskiego 6
Michał Sekrecki: Department of Gene Expression, Institute of Molecular Biology and Biotechnology, Adam Mickiewicz University, Uniwersytetu Poznanskiego 6
Agnieszka Piasecka: Department of Gene Expression, Institute of Molecular Biology and Biotechnology, Adam Mickiewicz University, Uniwersytetu Poznanskiego 6
Katarzyna Taylor: Department of Gene Expression, Institute of Molecular Biology and Biotechnology, Adam Mickiewicz University, Uniwersytetu Poznanskiego 6
Renate K. Hukema: Department of Clinical Genetics, Erasmus MC
Oliver Stork: Department of Genetics and Molecular Neurobiology, Institute of Biology, Otto von Guericke University
Krzysztof Sobczak: Department of Gene Expression, Institute of Molecular Biology and Biotechnology, Adam Mickiewicz University, Uniwersytetu Poznanskiego 6

DOI: https://doi.org/10.1038/s41467-021-21021-w
Journal volume & issue: Vol. 12, no. 1
pp. 1 – 17

Abstract

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Fragile X-associated tremor/ataxia syndrome is a neurodegenerative disease caused by toxic RNA containing expanded CGG repeats. Here, the authors show that synthetic oligonucleotides targeting the RNA repeats decrease the pleiotropic effect of this toxic molecule in cellular and animal models of the disease.

Published in Nature Communications

ISSN: 2041-1723 (Online)
Publisher: Nature Portfolio
Country of publisher: United Kingdom
LCC subjects: Science
Website: https://www.nature.com/ncomms/

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