Antisense Oligonucleotides as Precision Therapies for Neurodegenerative Diseases: A Narrative Review

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AuthorSohameh Mohebbien
AuthorMarayam Ghanbarien
AuthorMaryam Hassanlouen
OrcidSohameh Mohebbi [0000-0002-4512-175X]en
OrcidMarayam Ghanbari [0000-0002-7183-3769]en
OrcidMaryam Hassanlou [0000-0002-0856-4236]en
Issued Date2026-03-31en
AbstractContext: Antisense oligonucleotides (antisense oligonucleotides (ASOs)) are emerging precision therapies that modulate RNA expression and have shown promise for neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis, and Huntington’s disease. Evidence Acquisition: This narrative review summarizes key ASO mechanisms of action, major disease-related targets, delivery approaches, clinical progress, safety concerns, emerging technologies, approved ASO therapies, and ongoing clinical development relevant to neurodegeneration. Results: ASO-based therapies have shown increasing therapeutic potential across multiple neurodegenerative disorders, driven by advances in molecular targeting, delivery strategies, and clinical development. However, key safety concerns and translational barriers continue to limit their broader clinical application. Conclusions: ASO-based therapy represents a promising precision-medicine approach for neurodegenerative diseases. Further research should focus on overcoming translational barriers, improving delivery and safety, and advancing effective ASO therapies toward wider clinical adoption.en
DOIhttps://doi.org/10.5812/jjcmb-169275en
URIhttps://brieflands.com/journals/jjcmb/articles/169275en
KeywordAntisense Oligonucleotidesen
KeywordNeurodegenerative Diseasesen
KeywordRNase H Mechanismen
KeywordGene Silencingen
KeywordNanoplatform Deliveryen
PublisherBrieflandsen
TitleAntisense Oligonucleotides as Precision Therapies for Neurodegenerative Diseases: A Narrative Reviewen
TypeReview Articleen

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