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Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide.

Autor: Tran H; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA., Moazami MP; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA., Yang H; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA., McKenna-Yasek D; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA., Douthwright CL; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA., Pinto C; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA., Metterville J; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA., Shin M; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA., Sanil N; Research Pharmacy, University of Massachusetts Medical School, Worcester, MA, USA., Dooley C; Research Pharmacy, University of Massachusetts Medical School, Worcester, MA, USA., Puri A; Department of Radiology, University of Massachusetts Medical School, Worcester, MA, USA., Weiss A; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA., Wightman N; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA., Gray-Edwards H; Department of Radiology, University of Massachusetts Medical School, Worcester, MA, USA., Marosfoi M; Department of Radiology, University of Massachusetts Medical School, Worcester, MA, USA., King RM; Department of Radiology, University of Massachusetts Medical School, Worcester, MA, USA.; Department of Biomedical Engineering, Worcester Polytechnic Institute, Worcester, MA, USA., Kenderdine T; Department of Chemistry, University of Connecticut, Storrs, CT, USA., Fabris D; Department of Chemistry, University of Connecticut, Storrs, CT, USA., Bowser R; Departments of Neurology and Neurobiology, Barrow Neurological Institute, Phoenix, AZ, USA., Watts JK; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA. jonathan.watts@umassmed.edu., Brown RH Jr; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA. robert.brown@umassmed.edu.

Publikováno v: Nature medicine [Nat Med] 2022 Jan; Vol. 28 (1), pp. 117-124. Date of Electronic Publication: 2021 Dec 23.