The nucleobase guanine at the 3'-terminus of oligonucleotide RGLS4326 drives off-target AMPAR inhibition and CNS toxicity.
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| Title: | The nucleobase guanine at the 3'-terminus of oligonucleotide RGLS4326 drives off-target AMPAR inhibition and CNS toxicity. |
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| Authors: | Valencia T; Regulus Therapeutics Inc., San Diego, CA, USA., Yen LY; Department of Biochemistry and Molecular Biophysics, Columbia University, New York, NY, USA.; Cellular and Molecular Physiology and Biophysics Graduate Program, Columbia University Irving Medical Center, New York, NY, USA., Berman C; Berman Consulting, Wayland, MA, USA., Vincent T; Regulus Therapeutics Inc., San Diego, CA, USA., Davis S; Regulus Therapeutics Inc., San Diego, CA, USA., Varrone F; Regulus Therapeutics Inc., San Diego, CA, USA., Huang J; Regulus Therapeutics Inc., San Diego, CA, USA., Mastroianni J; Regulus Therapeutics Inc., San Diego, CA, USA., Carlson M; Regulus Therapeutics Inc., San Diego, CA, USA., Owen T; Regulus Therapeutics Inc., San Diego, CA, USA., Kamel A; Regulus Therapeutics Inc., San Diego, CA, USA., Drygin D; Regulus Therapeutics Inc., San Diego, CA, USA., Kinberger GA; Regulus Therapeutics Inc., San Diego, CA, USA., Gangwar SP; Cellular and Molecular Physiology and Biophysics Graduate Program, Columbia University Irving Medical Center, New York, NY, USA., Yelshanskaya MV; Cellular and Molecular Physiology and Biophysics Graduate Program, Columbia University Irving Medical Center, New York, NY, USA., Ridley J; Metrion Biosciences Ltd, Granta Park, Cambridge, UK., Kirby R; Metrion Biosciences Ltd, Granta Park, Cambridge, UK., Alvarez J; Department of Internal Medicine and Division of Nephrology, University of Texas Southwestern Medical Center, Dallas, TX, USA., Lakhia R; Department of Internal Medicine and Division of Nephrology, University of Texas Southwestern Medical Center, Dallas, TX, USA., Patel V; Department of Internal Medicine and Division of Nephrology, University of Texas Southwestern Medical Center, Dallas, TX, USA., Sobolevsky AI; Cellular and Molecular Physiology and Biophysics Graduate Program, Columbia University Irving Medical Center, New York, NY, USA. as4005@cumc.columbia.edu., Lee EC; Regulus Therapeutics Inc., San Diego, CA, USA. elee@regulusrx.com. |
| Source: | Nature communications [Nat Commun] 2025 Nov 28; Vol. 16 (1), pp. 10762. Date of Electronic Publication: 2025 Nov 28. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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