Potent Mixed Backbone Antisense Oligonucleotide Safety Suppressed Expression of Mutant C9ORF72 Transcripts and Polypeptides: First in Human Pilot Study
Robert C. Brown(University of Massachusetts Chan Medical School), Jonathan K. Watts(University of Massachusetts Chan Medical School), Ajit S Puri(University of Massachusetts Chan Medical School), Catherine Douthwright, Craig Dooley(University of Massachusetts Chan Medical School), Jake Metterville(University of Massachusetts Chan Medical School), Thomas Kenderdine(University of Connecticut), Hélène Tran(University of Massachusetts Chan Medical School), Minwook Shin(University of Massachusetts Chan Medical School), Robert Bowser(St. Joseph's Hospital and Medical Center), R King(University of Massachusetts Chan Medical School), Huiya Yang(University of Massachusetts Chan Medical School), Michael P. Moazami(University of Massachusetts Chan Medical School), Daniele Fabris(University of Connecticut), Nitasha Sanil(University of Massachusetts Chan Medical School), Courtney Pinto(University of Massachusetts Chan Medical School), Heather Gray‐Edwards(UMass Memorial Health Care), Alexandra Weiss(University of Massachusetts Chan Medical School), Nichols Wightman(University of Massachusetts Chan Medical School), Diane McKenna‐Yasek(University of Massachusetts Chan Medical School), Miklós Marosföi
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