Identification and Optimization of RNA-Splicing Modulators as Huntingtin Protein-Lowering Agents for the Treatment of Huntington’s Disease
作者:Longbin Liu、Karine Malagu、Alan F. Haughan、Vinod Khetarpal、Andrew J. Stott、William Esmieu、Huw D. Vater、Stephen J. Webster、Amanda J. Van de Poël、Cole Clissold、Brett Cosgrove、Benjamin Sutton、Jonathan A. Spencer、Perla Breccia、Emanuela Gancia、Silvia Bonomo、Tammy Ladduwahetty、Ovadia Lazari、Hiral Patel、Helen C. Atton、Steve Clifton、Daniel M. Mota、Dario Magnani、Amy O’Neill、Marta Stebbeds、Natsuko Macabuag、Daniel Todd、Maria E. Herva、Philip Mitchell、Mijke Visser、Sara Compte Sancerni、Laure Grand Moursel、Marta da Silva、Eva Kritikou、Taneli T. Heikkinen、Tamuna Bolkvadze、Valentina Fodale、Debora Spadafora、Manuel Daldin、Alberto Bresciani、John E. Mangette、Elizabeth M. Doherty、Matthew R. Lee、Todd Herbst、Edith Monteagudo、Douglas Macdonald、Nikolay V. Plotnikov、Mark Chambers、George McAllister、Ignacio Muňoz-Sanjuan、Celia Dominguez
DOI:10.1021/acs.jmedchem.3c01173
日期:2023.9.28
HTT MODULATORS FOR TREATING HUNTINGTON'S DISEASE
申请人:CHDI Foundation, Inc.
公开号:US20220409615A1
公开(公告)日:2022-12-29
Provided herein are certain compounds useful as HTT modulators. Such compound are useful in the treatment of Huntington's disease.