The nucleobase guanine at the 3’-terminus of oligonucleotide RGLS4326 drives off-target AMPAR inhibition and CNS toxicity

Tania Valencia(Regulus Therapeutics (United States)), Edmund C. Lee(Regulus Therapeutics (United States)), Francesca Varrone(Regulus Therapeutics (United States)), Laura Y. Yen(Columbia University), Denis Drygin(Regulus Therapeutics (United States)), Jianfeng Huang(Regulus Therapeutics (United States)), Ronak Lakhia(Southwestern Medical Center), John W. Ridley(Granta Design (United Kingdom)), Tate Owen(Regulus Therapeutics (United States)), Scott Davis(Unknown), Thomas M. Vincent(Regulus Therapeutics (United States)), Maria V. Yelshanskaya(Columbia University), Shanti Pal Gangwar(Columbia University), Vishal Patel(Southwestern Medical Center), Amin Kamel(Regulus Therapeutics (United States)), Jesus Alvarez(The University of Texas Southwestern Medical Center), Morgan E. Carlson(Regulus Therapeutics (United States)), Robert W. Kirby(Granta Design (United Kingdom)), Garth A. Kinberger(Ionis Pharmaceuticals (United States)), Jessica Mastroianni(Regulus Therapeutics (United States)), Alexander I. Sobolevsky(Columbia University Irving Medical Center)
Nature Communications
November 28, 2025
Cited by 1


Related Papers