Potent Mixed Backbone Antisense Oligonucleotide Safety Suppressed Expression of Mutant C9ORF72 Transcripts and Polypeptides: First in Human Pilot Study
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Robert H. Brown | R. Bowser | D. Fabris | J. Watts | M. Marosfoi | A. Puri | D. McKenna-Yasek | H. Tran | Jake P. Metterville | R. King | Catherine L Douthwright | T. Kenderdine | H. Gray-Edwards | Min-Kyu Shin | Huiya Yang | Michael P. Moazami | Courtney Pinto | N. Sanil | C. Dooley | Alexandra Weiss | Nichols Wightman | Robert H. Brown | Thomas Kenderdine | A. Puri | R. King