[SAGE] Targeting a Pathogenic Variant Creating an Upstream AUG in theENG5∩ Untranslated Region with Antisense Oligonucleotides Fails to Restore Protein Expression

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journalㄩNucleic Acid Therapeutics

AuthorsㄩMathilde Doisy; Aris Gaci; Omar Soukarieh; Carole Proust; David-Alexandre Tr谷gout; Aur谷lie Goyenvalle

Published dateㄩ2026-2-1

DOIㄩ10.1177/21593337251396711

PDF linkㄩhttps://journals.sagepub.com/doi ... 7/21593337251396711

Article linkㄩhttps://doi.org/10.1177/21593337251396711

Article SourceㄩSAGE Publications﹝


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