Restricted accessResearch articleFirst published 2026-02-01pp. 12-22Targeting a Pathogenic Variant Creating an Upstream AUG in the ENG 5′ Untranslated Region with Antisense Oligonucleotides Fails to Restore Protein Expression Mathilde Doisy, Aris Gaci, Omar Soukarieh , [...]View all
Restricted accessResearch articleFirst published 2026-02-01pp. 23-36Class-Specific Adverse Events of Patients Treated with Small Interfering RNA Therapeutics: A Disproportionality Analysis of the United States Food and Drug Administration Adverse Event Reporting System Database Based on the MY FAERS Platform Ze Li, Xiaozhen Wang, Dandan Li , [...]View all
Restricted accessResearch articleFirst published 2026-02-01pp. 47-55Unveiling Liver Micro-Distribution: NanoSIMS Imaging Reveals Critical Intracellular Distribution of Chemically Modified Antisense Oligonucleotides Hidenori Yasuhara, Kenta Kadotsuji, Kenichi Watanabe , [...]View all