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Tran H, Moazami MP, Yang H, McKenna-Yasek D, Douthwright C, Pinto C, Metterville J, Shin M, Sanil N, Dooley C, Puri A, Weiss A, Wightman N, Gray-Edwards H, Marosfoi M, King RM, Kenderdine T, Fabris D, Bowser R, Watts JK, Brown RH. Potent Mixed Backbone Antisense Oligonucleotide Safely Suppresses Expression of Mutant C9ORF72 Transcripts and Polydipeptides: First in Human Pilot Study. Research Square preprint, February 21 2021. Research Square.
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