Antisense Oligonucleotides: Concepts and Pharmaceutical Applications
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Submitted
3 February 2021
Published
28 February 2023
Keywords:
-
Antisense oligonucleotides, Production, Validation, Clinical Trial, Disease treatment
Abstract
Antisense oligonucleotides are drugs whose mechanism is based on binding to RNA target sequences. For this purpose, they modify the protein expression through steric hindrance and exon omission. Its production involves several steps: synthesis, purification, and lyophilization. Usually, the most complicated procedure is synthesis due to the chemical reactions necessary to add the required oligonucleotide bases. BP1001, inotersen, nusinersen, eteplirsen, and golodirsen are a few antisense drugs developed for treating neurodegenerative and neuromuscular diseases. Although antisense oligonucleotides present off-target reactions, multiple studies are being performed. The following review shows information regarding the pharmaceutical characteristics for industrial production and the current state of applicability in clinical practice. In conclusion, some molecules have already been approved for commercialization (inotersen, nusinersen, ataluren, eteplirsen, and golodirsen), showing them as promising therapeutic solutions in the short and medium term for disorders developed by specific genetic factors.
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Araya A, Arias D, Coto K, López MR, Rivera J, Mora JJ. Antisense Oligonucleotides: Concepts and Pharmaceutical Applications. Borneo J Pharm [Internet]. 2023Feb.28 [cited 2024Apr.25];6(1):41-57. Available from: https://journal.umpr.ac.id/index.php/bjop/article/view/2092
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