OLIGONUCLEOTIDES: THE TINY MOLECULES, RESHAPING MODERN MEDICINE
AbstractOligonucleotides (ONs) have become the groundbreaking category of therapeutics capable of specific control of gene expression and treatment of pathogenic mutations. ONs are transforming the future of modern medicine, with the use of antisense oligonucleotides and small interfering RNAs, aptamers, and CRISPR guide RNAs. Multiple ON-based drugs have already been approved by FDA and EMA, and these drugs show therapeutic effectiveness in genetic, metabolic, neurodegenerative, and infectious diseases. Nevertheless, other obstacles as delivery barriers, off-target effects, and high cost are a major concern. In this review, the oligonucleotide therapeutics, its nature, mode of action, chemo-modified and /or delivery strategy/plan, therapeutic use, clinical outcomes, limitations, and forecasts have been described in detail.
Article Information
8
858-871
553 KB
1
English
IJPSR
Deepali K. Kadam *, Rutuja R. Salvi and Laxmikant B. Borse
Department of Pharmaceutical Chemistry, Sandip Foundation’s, Sandip Institute of Pharmaceutical Sciences, Mahiravani, Nashik, Maharashtra, India.
drdeepalikadam777@gmail.com
29 September 2025
28 October 2025
02 November 2025
10.13040/IJPSR.0975-8232.17(3).858-71
01 March 2026
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