Current Advances in RNA Therapeutics for Human Diseases
Following the discovery of nucleic acids by Friedrich Miescher in 1868, DNA and RNA were recognized as the genetic code containing the necessary information for proper cell functioning. In the years following these discoveries, vast knowledge of the seemingly endless roles of RNA have become better...
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| Format: | Article |
| Language: | English |
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MDPI AG
2022-03-01
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| Series: | International Journal of Molecular Sciences |
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| Online Access: | https://www.mdpi.com/1422-0067/23/5/2736 |
| _version_ | 1797474866644910080 |
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| author | Hannah Zogg Rajan Singh Seungil Ro |
| author_facet | Hannah Zogg Rajan Singh Seungil Ro |
| author_sort | Hannah Zogg |
| collection | DOAJ |
| description | Following the discovery of nucleic acids by Friedrich Miescher in 1868, DNA and RNA were recognized as the genetic code containing the necessary information for proper cell functioning. In the years following these discoveries, vast knowledge of the seemingly endless roles of RNA have become better understood. Additionally, many new types of RNAs were discovered that seemed to have no coding properties (non-coding RNAs), such as microRNAs (miRNAs). The discovery of these new RNAs created a new avenue for treating various human diseases. However, RNA is relatively unstable and is degraded fairly rapidly once administered; this has led to the development of novel delivery mechanisms, such as nanoparticles to increase stability as well as to prevent off-target effects of these molecules. Current advances in RNA-based therapies have substantial promise in treating and preventing many human diseases and disorders through fixing the pathology instead of merely treating the symptomology similarly to traditional therapeutics. Although many RNA therapeutics have made it to clinical trials, only a few have been FDA approved thus far. Additionally, the results of clinical trials for RNA therapeutics have been ambivalent to date, with some studies demonstrating potent efficacy, whereas others have limited effectiveness and/or toxicity. Momentum is building in the clinic for RNA therapeutics; future clinical care of human diseases will likely comprise promising RNA therapeutics. This review focuses on the current advances of RNA therapeutics and addresses current challenges with their development. |
| first_indexed | 2024-03-09T20:37:09Z |
| format | Article |
| id | doaj.art-9b1d17d7642f466bb08b3c5c64b2b015 |
| institution | Directory Open Access Journal |
| issn | 1661-6596 1422-0067 |
| language | English |
| last_indexed | 2024-03-09T20:37:09Z |
| publishDate | 2022-03-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | International Journal of Molecular Sciences |
| spelling | doaj.art-9b1d17d7642f466bb08b3c5c64b2b0152023-11-23T23:08:50ZengMDPI AGInternational Journal of Molecular Sciences1661-65961422-00672022-03-01235273610.3390/ijms23052736Current Advances in RNA Therapeutics for Human DiseasesHannah Zogg0Rajan Singh1Seungil Ro2Department of Physiology and Cell Biology, Reno School of Medicine, University of Nevada, 1664 North Virginia Street, Reno, NV 89557, USADepartment of Physiology and Cell Biology, Reno School of Medicine, University of Nevada, 1664 North Virginia Street, Reno, NV 89557, USADepartment of Physiology and Cell Biology, Reno School of Medicine, University of Nevada, 1664 North Virginia Street, Reno, NV 89557, USAFollowing the discovery of nucleic acids by Friedrich Miescher in 1868, DNA and RNA were recognized as the genetic code containing the necessary information for proper cell functioning. In the years following these discoveries, vast knowledge of the seemingly endless roles of RNA have become better understood. Additionally, many new types of RNAs were discovered that seemed to have no coding properties (non-coding RNAs), such as microRNAs (miRNAs). The discovery of these new RNAs created a new avenue for treating various human diseases. However, RNA is relatively unstable and is degraded fairly rapidly once administered; this has led to the development of novel delivery mechanisms, such as nanoparticles to increase stability as well as to prevent off-target effects of these molecules. Current advances in RNA-based therapies have substantial promise in treating and preventing many human diseases and disorders through fixing the pathology instead of merely treating the symptomology similarly to traditional therapeutics. Although many RNA therapeutics have made it to clinical trials, only a few have been FDA approved thus far. Additionally, the results of clinical trials for RNA therapeutics have been ambivalent to date, with some studies demonstrating potent efficacy, whereas others have limited effectiveness and/or toxicity. Momentum is building in the clinic for RNA therapeutics; future clinical care of human diseases will likely comprise promising RNA therapeutics. This review focuses on the current advances of RNA therapeutics and addresses current challenges with their development.https://www.mdpi.com/1422-0067/23/5/2736RNA therapeuticsnon-coding RNAmiRNAASOsiRNAaptamer |
| spellingShingle | Hannah Zogg Rajan Singh Seungil Ro Current Advances in RNA Therapeutics for Human Diseases International Journal of Molecular Sciences RNA therapeutics non-coding RNA miRNA ASO siRNA aptamer |
| title | Current Advances in RNA Therapeutics for Human Diseases |
| title_full | Current Advances in RNA Therapeutics for Human Diseases |
| title_fullStr | Current Advances in RNA Therapeutics for Human Diseases |
| title_full_unstemmed | Current Advances in RNA Therapeutics for Human Diseases |
| title_short | Current Advances in RNA Therapeutics for Human Diseases |
| title_sort | current advances in rna therapeutics for human diseases |
| topic | RNA therapeutics non-coding RNA miRNA ASO siRNA aptamer |
| url | https://www.mdpi.com/1422-0067/23/5/2736 |
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