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I01 Orally Bioavailable Small Molecule Splicing Modifiers with Systemic and Even Htt-Lowering Activity in Vitro and in Vivo

Anuradha Bhattacharyya,Kerstin Effenberger,Christopher Trotta,Jana Narasimhan,Wencheng Li,Matthew Woll, Minakshi Jani,Nicole Risher,Shirley Yeh,Yaofeng Cheng,Nadiya Sydorenko,Marla Weetall,Amber L. Southwell,Michael R. Hayden,Joseph Colacino,Stuart Peltz

JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY（2021）

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