Antisense oligonucleotides: a novel Frontier in pharmacological strategy
Antisense oligonucleotides (ASOs) are short single stranded synthetic RNA or DNA molecules, whereas double-stranded RNA nucleotide sequences are called small interfering RNA (siRNA). ASOs bind to complementary nucleic acid sequences impacting the associated functions of the targeted nucleic acids. T...
| Main Authors: | , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Frontiers Media S.A.
2023-11-01
|
| Series: | Frontiers in Pharmacology |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fphar.2023.1304342/full |
| _version_ | 1797612172658868224 |
|---|---|
| author | D. Collotta I. Bertocchi I. Bertocchi E. Chiapello M. Collino |
| author_facet | D. Collotta I. Bertocchi I. Bertocchi E. Chiapello M. Collino |
| author_sort | D. Collotta |
| collection | DOAJ |
| description | Antisense oligonucleotides (ASOs) are short single stranded synthetic RNA or DNA molecules, whereas double-stranded RNA nucleotide sequences are called small interfering RNA (siRNA). ASOs bind to complementary nucleic acid sequences impacting the associated functions of the targeted nucleic acids. They represent an emerging class of drugs that, through a revolutionary mechanism of action, aim to directly regulate disease-causing genes and their variants, providing an alternative tool to traditional “protein-specific” therapies. The majority of the ASOs are designed to treat orphan genetic disorders that in most of the cases are seriously disabling and still lacking an adequate therapy. In order to translate ASOs into clinical success, constant technological advances have been instrumental in overcoming several pharmacological, toxicological and formulation limitations. Accordingly, chemical structures have been recently implemented and new bio-conjugation and nanocarriers formulation strategies explored. The aim of this work is to offer an overview of the antisense technology with a comparative analysis of the oligonucleotides approved by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA). |
| first_indexed | 2024-03-11T06:38:33Z |
| format | Article |
| id | doaj.art-1b48e71169504bf8a3307a977ff51ccd |
| institution | Directory Open Access Journal |
| issn | 1663-9812 |
| language | English |
| last_indexed | 2024-03-11T06:38:33Z |
| publishDate | 2023-11-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Pharmacology |
| spelling | doaj.art-1b48e71169504bf8a3307a977ff51ccd2023-11-17T10:44:08ZengFrontiers Media S.A.Frontiers in Pharmacology1663-98122023-11-011410.3389/fphar.2023.13043421304342Antisense oligonucleotides: a novel Frontier in pharmacological strategyD. Collotta0I. Bertocchi1I. Bertocchi2E. Chiapello3M. Collino4Department of Neuroscience Rita Levi Montalcini, University of Turin, Turin, ItalyDepartment of Neuroscience Rita Levi Montalcini, University of Turin, Turin, ItalyNeuroscience Institute Cavalieri Ottolenghi (NICO), University of Turin, Turin, ItalyDepartment of Neuroscience Rita Levi Montalcini, University of Turin, Turin, ItalyDepartment of Neuroscience Rita Levi Montalcini, University of Turin, Turin, ItalyAntisense oligonucleotides (ASOs) are short single stranded synthetic RNA or DNA molecules, whereas double-stranded RNA nucleotide sequences are called small interfering RNA (siRNA). ASOs bind to complementary nucleic acid sequences impacting the associated functions of the targeted nucleic acids. They represent an emerging class of drugs that, through a revolutionary mechanism of action, aim to directly regulate disease-causing genes and their variants, providing an alternative tool to traditional “protein-specific” therapies. The majority of the ASOs are designed to treat orphan genetic disorders that in most of the cases are seriously disabling and still lacking an adequate therapy. In order to translate ASOs into clinical success, constant technological advances have been instrumental in overcoming several pharmacological, toxicological and formulation limitations. Accordingly, chemical structures have been recently implemented and new bio-conjugation and nanocarriers formulation strategies explored. The aim of this work is to offer an overview of the antisense technology with a comparative analysis of the oligonucleotides approved by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA).https://www.frontiersin.org/articles/10.3389/fphar.2023.1304342/fullantisense oligonucleotidesiRNAgenetic disordergene silencingpharmacology |
| spellingShingle | D. Collotta I. Bertocchi I. Bertocchi E. Chiapello M. Collino Antisense oligonucleotides: a novel Frontier in pharmacological strategy Frontiers in Pharmacology antisense oligonucleotide siRNA genetic disorder gene silencing pharmacology |
| title | Antisense oligonucleotides: a novel Frontier in pharmacological strategy |
| title_full | Antisense oligonucleotides: a novel Frontier in pharmacological strategy |
| title_fullStr | Antisense oligonucleotides: a novel Frontier in pharmacological strategy |
| title_full_unstemmed | Antisense oligonucleotides: a novel Frontier in pharmacological strategy |
| title_short | Antisense oligonucleotides: a novel Frontier in pharmacological strategy |
| title_sort | antisense oligonucleotides a novel frontier in pharmacological strategy |
| topic | antisense oligonucleotide siRNA genetic disorder gene silencing pharmacology |
| url | https://www.frontiersin.org/articles/10.3389/fphar.2023.1304342/full |
| work_keys_str_mv | AT dcollotta antisenseoligonucleotidesanovelfrontierinpharmacologicalstrategy AT ibertocchi antisenseoligonucleotidesanovelfrontierinpharmacologicalstrategy AT ibertocchi antisenseoligonucleotidesanovelfrontierinpharmacologicalstrategy AT echiapello antisenseoligonucleotidesanovelfrontierinpharmacologicalstrategy AT mcollino antisenseoligonucleotidesanovelfrontierinpharmacologicalstrategy |