Borneo Journal of Pharmacy
Vol. 6 No. 1 (2023): Borneo Journal of Pharmacy

Antisense Oligonucleotides: Concepts and Pharmaceutical Applications

Ariana Araya (Universidad de Costa Rica)
David Arias (Universidad de Costa Rica)
Karla Coto (Universidad de Costa Rica)
María Rebeca López (Universidad de Costa Rica)
Joselyn Rivera (Universidad de Costa Rica)
Juan José Mora (Universidad de Costa Rica)



Article Info

Publish Date
28 Feb 2023

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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Journal Info

Abbrev

bjop

Publisher

Subject

Medicine & Pharmacology

Description

Borneo Journal of Pharmacy publishes various scientific articles covering Pharmacy and Pharmaceutical Sciences in the field but not limited to: Pharmacology-Toxicology, including pharmacokinetics, pharmacodynamics, pharmacotherapy, and toxicology. Pharmacognosy-Phytochemistry, including ...