Journal Article

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

Author(s)

Tania Valencia/Regulus Therapeutics, Inc
Laura Y Yen/Columbia University/Columbia University Irving Medical Center
Cindy Berman/Berman Consulting
Thomas Vincent/Regulus Therapeutics Inc.
Scott Davis/Regulus Therapeutics Inc.
Francesca Varrone/Regulus Therapeutics Inc.
Jianfeng Huang/Regulus Therapeutics Inc.
Jessica Mastroianni/Regulus Therapeutics Inc.
Morgan Carlson/Regulus Therapeutics Inc.
Tate Owen/Regulus Therapeutics Inc.
Amin Kamel/Regulus Therapeutics Inc.
Denis Drygin/Regulus Therapeutics Inc.
Garth A Kinberger/Regulus Therapeutics Inc.
Shanti P Gangwar/Columbia University Irving Medical Center
Maria V Yelshanskaya/Columbia University Irving Medical Center
John Ridley/Metrion Biosciences Ltd.
Robert Kirby/Metrion Biosciences Ltd.
Jesus Alvarez/University of Texas Southwestern Medical Center
Ronak Lakhia/University of Texas Southwestern Medical Center
Vishal Patel/University of Texas Southwestern Medical Center
Alexander I Sobolevsky/Columbia University Irving Medical Center
Edmund C Lee/Regulus Therapeutics Inc.

Publisher

Nature Communications

Publication Date

November 28, 2025

DOI

DOI: 10.1038/s41467-025-65799-5

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