Open Access
Subscription Access
Open Access
Subscription Access
Emergence of Oligonucleotide Drugs
Subscribe/Renew Journal
Identification of DNA as hereditary material together with the revelation of double helical structure of DNA laid the foundation for understanding various diseases of genetic origin. Later on, with the report of non-aqueous chemical synthesis of genetic material, i.e., oligonucleotides, and discovery of solid phase synthesis, possibilities to produce custom design pieces of genetic material in large quantity, in reasonable time frame and in cost-effective manner emerged. This paved the path for nucleic acid based therapeutics. As technology matured, several oligonucleotide drugs got green signals from FDA during last decade. Waylivra® and Givlaari® are the latest additions.
Keywords
DNA, Oligonucleotide, Therapeutics, Antisense, siRNA.
User
Subscription
Login to verify subscription
Font Size
Information
- O T Avery, C M MacLeod and M McCarty, Studies on the Chemical Nature of the Substance Inducing Transformation of Pneumococcal Types, Journal of Experimental Medicine, Vol 79, No 2, page 137 – 158, 1944.
- K E Lundin, O Gissberg and C I E Smith, Oligonucleotide Therapies: The Past and the Present, Human Gene Therapy,Vol 26, No 8, page 475 – 485, 2015.
- J D Watson and F H C Crick, Molecular Structure of Nucleic Acids; A Structure for Deoxyribose Nucleic Acid, Nature, Vol 171, No 4356, page 737 – 738, 1953.
- M H F Wilkins, A R Stokes and H R Wilson, Molecular Structure of Deoxypentose Nucleic Acids, Nature, Vol 171, No 4356, page 738 – 740, 1953.
- MH F Wilkins, W E Seeds, A R Stokes and H R Wilson, Helical Structure of Crystalline Deoxypentose Nucleic Acid, Nature, Vol 172, No 4382, page 759 – 762, 1953.
- N Kresge, R D Simoni and R L Hill, Total Synthesis of Tyrosine Suppressor tRNA: the Work of H. Gobind Khorana, Journal of Biological Chemistry, Vol 284, No 22, page e5 – e6, 2009.
- P C Zamecnik and M L Stephenson, Inhibition of Rous Sarcoma Virus Replication and Cell Transformation by a Specific Oligodeoxynucleotide, Proceedings of the National Academy of Sciences of the USA, Vol 75, No 1, page 280 – 284, 1978.
- M L Stephenson, and P C Zamecnik, Inhibition of Rous Sarcoma Viral RNA Translation by a Specific Oligodeoxyribonucleotide, Proceedings of the National Academy of Sciences of the USA, Vol 75, No 1, page 285 – 288, 1978.
- H Donis-Keller, Site Specific Enzymatic Cleavage of RNA, Nucleic Acids Research, Vol 7, No 1, page 179 – 192, 1979.
- C A Stein and D Castanotto, FDA-Approved Oligonucleotide Therapies in 2017, Molecular Therapy, Vol 25, No 5, page 1069 – 1075, 2017.
- A Fire, S Xu, M K Montgomery, S A Kostas, S E Driver and C C Mello, Potent and Specific Genetic Interference by Double-Stranded RNA in Caenorhabditis Elegans, Nature, Vol 391, No 6669, page 806 – 811, 1998.
- B Hu, L Zhong, Y Weng, LPeng, Y Huang, Y Zhao and X Liang, Therapeutic siRNA: State of the Art, Signal Transductionand Targeted Therapy,Vol 5, No 101, 2020.
- A Akinc et al., The Onpattro Story and the Clinical Translation of Nanomedicines Containing Nucleic Acid-Based Drugs, Nature. Nanotechnology,Vol 14, No 12, page 1084 – 1087, 2019.
- K E Lundin, O Gissberg, and C I E Smith, Oligonucleotide Therapies: the Past and the Present, Human Gene Therapy, Vol 26, No 8, page 475 – 485, 2015.
- https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-vyondys-53, Content Current as of 12/26/2019.
- C I E Smith and R Zain, Therapeutic Oligonucleotides: State of the Art, Annual Review of Pharmcology and Toxicology, Vol 59, page 605 – 630, 2019.
- V V Oberemok et al., A Half-Century History of Applications of Antisense Oligonucleotides in Medicine, Agriculture and Forestry: We Should Continue the Journey, Molecules, Vol 23, No 6, page 1302, 2018.
- C F Bennett, Therapeutic Antisense Oligonucleotides are Coming of Age, Annual Review Medicine,Vol 70, page 307 – 321, 2019.
- L Gales, Tegsedi (Inotersen): An Antisense Oligonucleotide Approved for the Treatment of Adult Patients with Hereditary Transthyretin Amyloidosis, Pharmaceuticals, Vol 12, No 2, page 78, 2019.
- A D Ellington, and J W Szostak, In Vitro Selection of RNA Molecules that Bind Specific Ligands, Nature, Vol 346, page 818 – 822, 1990.
- C Tuerk and L Gold, Systematic Evolution of Ligands by Exponential Enrichment: RNA Ligands to Bacteriophage T4 DNA Polymerase, Science, Vol 249, No 4968, page 505 – 510, 1990.
- L Gold, SELEX: How It Happened and Where It will Go, Journal of Molecular Evolution, Vol 81, No 5-6, page 140 – 143, 2015.
- R RNikam and K R Gore, Journey of siRNA: Clinical Developments and Targeted Delivery, Nucleic Acids Therapeutics,Vol 28, No 4, page 209 – 224, 2018.
- A Wittrup and J Lieberman, Knocking down Disease: a Progress Report on siRNA Therapeutics, Nature Review Genetics, Vol 16, No 9, page 543 – 552, 2015.
- G L Sen and H M Blau, A Brief History of RNAi: the Silence of the Genes, The FASEB Journal, Vol 20, No 9, page 1293 – 1299, 2006.
- S Guo and K J Kemphues, Par-1, a Gene Required for Establishing Polarity in C. Elegans Embryos, Encodes a Putative Ser/Thr Kinase that is Asymmetrically Distributed, Cell, Vol 81, No 4, page 611 – 620, 1996.
- V K Sharma and J K Watts, Oligonucleotide Therapeutics: Chemistry, Delivery and Clinical Progress, Future Medicinal Chemistry, Vol 7, No 16, page 2221 – 2242, 2015.
- K Garber, Alnylam Launches Era of RNAi Drugs, Nature Biotechnology, Vol 36, No 9, page 777 – 778, 2018.
Abstract Views: 359
PDF Views: 0