Antisense Molecules: Design, Chemistry, and Therapeutic Innovations

Antisense oligonucleotides (ASOs) have been successfully approved by the FDA for the development of drugs such as Nusinersen (used for the treatment of spinal muscular atrophy) and Fomivirsen (used for treating Cytomegalovirus retinitis). ASOs offer a unique ability to target specific RNA sequences and help overcome limitations associated with traditional therapies.

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Publication Details

Journal
Mini-Reviews in Medicinal Chemistry
Published
2026-03-13
DOI
https://doi.org/10.2174/0113895575428832251206055438
Primary Topic
Neurogenetic and Muscular Disorders Research
Type
article
Field-Weighted Citation Impact
0.00
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Antisense Molecules: Design, Chemistry, and Therapeutic Innovations

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Mini-Reviews in Medicinal Chemistry
Neurogenetic and Muscular Disorders Research
article

Antisense Molecules: Design, Chemistry, and Therapeutic Innovations

Abitha Sri Ganapathy, Swetha Aravamudhan, Suryalakshmi Guhanathan, Jocelyn Olivia Prabhakar David, Prema Vediappan
article en

Abstract

Antisense oligonucleotides (ASOs) have been successfully approved by the FDA for the development of drugs such as Nusinersen (used for the treatment of spinal muscular atrophy) and Fomivirsen (used for treating Cytomegalovirus retinitis). ASOs offer a unique ability to target specific RNA sequences and help overcome limitations associated with traditional therapies.

Mini-Reviews in Medicinal ChemistryVol. 26
Tamil Nadu Dr. M.G.R. Medical University (IN)
Openalex Percentile: Top 23%
Neurogenetic and Muscular Disorders Research
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