Affiliation:
1. Universidad de Costa Rica
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.
Publisher
Universitas Muhammadiyah Palangkaraya
Subject
General Earth and Planetary Sciences,General Environmental Science
Reference107 articles.
1. 1. Bennett CF. Therapeutic Antisense Oligonucleotides Are Coming of Age. Annu Rev Med. 2019;70:307-21. doi:10.1146/annurev-med-041217-010829
2. 2. Oberemok VV, Laikova KV, Repetskaya AI, Kenyo IM, Gorlov MV, Kasich IG, et al. A Half-Century History of Applications of Antisense Oligonucleotides in Medicine, Agriculture and Forestry: We Should Continue the Journey. Molecules. 2018;23(6):1302. doi:10.3390/molecules23061302
3. 3. Svoboda J. Cell Association in Rous Sarcoma Virus (RSV) Rescue and Cell Infection. Folia Biol. 2015;61(5):161-7.
4. 4. Potaczek DP, Garn H, Unger SD, Renz H. Antisense molecules: A new class of drugs. J Allergy Clin Immunol. 2016;137(5):1334-46. doi:10.1016/j.jaci.2015.12.1344
5. 5. Gandhi H, Pathan A. Drug Discovery and Development: An Overview from the Regulatory Prospective. Pharmaceut Reg Affairs. 2019;8(1):1000215.