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 & Psychiatry Sep 2021, 92 (Suppl 1) A48; DOI: 10.1136/jnnp-2021-EHDN.115