ASO5-1 C9ORF72 Manuscript – 05Feb2021 POTENT MIXED BACKBONE ANTISENSE OLIGONUCLEOTIDE SAFELY SUPPRESSES EXPRESSION OF MUTANT C9ORF72 TRANSCRIPTS AND POLYDIPEPTIDES: FIRST IN HUMAN PILOT STUDY

Affiliations: 1Department of Neurology, University of Massachusetts Medical School, Worcester, MA, 01655 USA 2RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, 01655 USA 3Research Pharmacy, University of Massachusetts Medical School, Worcester, MA, 01655 USA 4Department of Radiology, University of Massachusetts Medical School, Worcester, MA, 01655 USA 5Department of Biomedical Engineering, Worcester Polytechnic Institute, Worcester, MA, 01609, USA; 6Department of Chemistry, University of Connecticut, Storrs, CT, 06269, USA 7Departments of Neurology and Neurobiology, Barrow Neurological Institute, Phoenix, AZ, 85013, USA