Antisense Molecules: Design, Chemistry, and Therapeutic Innovations

Introduction: Antisense molecules are short synthetic nucleic acid strands designed to bind to complementary sequences of messenger RNA (mRNA) to regulate gene expression. This study focuses on understanding the chemistry, modifications, and therapeutic applications of antisense molecules. Method: A comprehensive literature review was conducted using scientific databases, including PubMed, Scopus, and Google Scholar. Relevant published articles were selected using keywords such as “antisense oligonucleotides,” “gene silencing,” “therapeutic applications,” “neurological disorders,” and “cancer.” Studies included in this review consisted of research focusing on the mechanisms, delivery methods, and therapeutic outcomes of antisense oligonucleotides. Result: The data obtained in this study indicate that third-generation ASOs exhibit greater therapeutic efficacy than first- and second-generation ASOs. Discussion: This review compiles and discusses the mechanisms of action, structural features, chemical modifications, recent advances, applications, challenges, and future directions of ASOs in genetic disorders and other diseases. Conclusion: 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.