1. A Half-Century History of Applications of Antisense Oligonucleotides in Medicine, Agriculture and Forestry: We Should Continue the Journey.
- Author
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Oberemok VV, Laikova KV, Repetskaya AI, Kenyo IM, Gorlov MV, Kasich IN, Krasnodubets AM, Gal'chinsky NV, Fomochkina II, Zaitsev AS, Bekirova VV, Seidosmanova EE, Dydik KI, Meshcheryakova AO, Nazarov SA, Smagliy NN, Chelengerova EL, Kulanova AA, Deri K, Subbotkin MV, Useinov RZ, Shumskykh MN, and Kubyshkin AV
- Subjects
- Agriculture methods, Animals, Biological Control Agents chemical synthesis, Biological Control Agents history, DNA antagonists & inhibitors, DNA genetics, DNA metabolism, Forestry methods, Gene Expression Regulation drug effects, History, 20th Century, History, 21st Century, Humans, Larva drug effects, Larva genetics, Larva metabolism, Moths genetics, Moths growth & development, Moths metabolism, Muscular Atrophy, Spinal genetics, Muscular Atrophy, Spinal metabolism, Muscular Atrophy, Spinal pathology, Muscular Dystrophy, Duchenne genetics, Muscular Dystrophy, Duchenne metabolism, Muscular Dystrophy, Duchenne pathology, Nanoparticles administration & dosage, Nanoparticles chemistry, Neuromuscular Agents chemical synthesis, Neuromuscular Agents history, Oligonucleotides, Antisense chemical synthesis, Oligonucleotides, Antisense metabolism, RNA, Messenger antagonists & inhibitors, RNA, Messenger genetics, RNA, Messenger metabolism, Biological Control Agents pharmacology, Moths drug effects, Muscular Atrophy, Spinal therapy, Muscular Dystrophy, Duchenne therapy, Neuromuscular Agents therapeutic use, Oligonucleotides, Antisense genetics
- Abstract
Antisense oligonucleotides (ASO), short single-stranded polymers based on DNA or RNA chemistries and synthesized in vitro, regulate gene expression by binding in a sequence-specific manner to an RNA target. The functional activity and selectivity in the action of ASOs largely depends on the combination of nitrogenous bases in a target sequence. This simple and natural property of nucleic acids provides an attractive route by which scientists can create different ASO-based techniques. Over the last 50 years, planned and realized applications in the field of antisense and nucleic acid nanotechnologies have produced astonishing results and posed new challenges for further developments, exemplifying the essence of the post-genomic era. Today the majority of ASOs are chemically modified and/or incorporated within nanoparticles to enhance their stability and cellular uptake. This review critically analyzes some successful cases using the antisense approach in medicine to address severe diseases, such as Duchenne muscular dystrophy and spinal muscular atrophy, and suggests some prospective directions for future research. We also examine in detail the elaboration of unmodified insect-specific DNA insecticides and RNA preparations in the areas of agriculture and forestry, a relatively new branch of ASO that allows circumvention of the use of non-selective chemical insecticides. When considering the variety of successful ASO modifications with an efficient signal-to-noise ratio of action, coupled with the affordability of in vitro oligonucleotide synthesis and post-synthesis procedures, we predict that the next half-century will produce a fruitful yield of tools created from effective ASO-based end products.
- Published
- 2018
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