The Progress and Evolving Trends in Nucleic-Acid-Based Therapies
Nucleic-acid-based therapies have emerged as a pivotal domain within contemporary biomedical science, marked by significant advancements in recent years. These innovative treatments primarily operate through the precise binding of DNA or RNA molecules to discrete target genes, subsequently suppressi...
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MDPI AG
2025-03-01
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| Series: | Biomolecules |
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| Online Access: | https://www.mdpi.com/2218-273X/15/3/376 |
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| author | Yunlong Liu Chunmiao Wang Xiuping Fu Mengtian Ren |
| author_facet | Yunlong Liu Chunmiao Wang Xiuping Fu Mengtian Ren |
| author_sort | Yunlong Liu |
| collection | DOAJ |
| description | Nucleic-acid-based therapies have emerged as a pivotal domain within contemporary biomedical science, marked by significant advancements in recent years. These innovative treatments primarily operate through the precise binding of DNA or RNA molecules to discrete target genes, subsequently suppressing the expression of the target proteins. The spectrum of nucleic-acid-based therapies encompasses antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), microRNAs (miRNAs), and messenger RNAs (mRNAs), etc. Compared to more traditional medicinal approaches, nucleic-acid-based therapies stand out for their highly targeted action on specific genes, as well as their potential for chemical modification to improve resistance to nucleases, ensuring sustained therapeutic activity and mitigating immunogenicity concerns. Nevertheless, these molecules’ limited cellular permeability necessitates the deployment of delivery vectors to enhance their intracellular uptake and stability. As nucleic-acid-based therapies progressively display promising pharmacodynamic profiles, there has been a burgeoning interest in these treatments for applications in clinical research. This review aims to summarize the variety of nucleic acid drugs and their mechanisms, evaluate the present status in research and application, discourse on prospective trends, and potential challenges ahead. These innovative therapeutics are anticipated to assume a pivotal role in the management of a wide array of diseases. |
| format | Article |
| id | doaj-art-c9ecfd15b13a4e9ea02431296cf90c1d |
| institution | OA Journals |
| issn | 2218-273X |
| language | English |
| publishDate | 2025-03-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Biomolecules |
| spelling | doaj-art-c9ecfd15b13a4e9ea02431296cf90c1d2025-08-20T02:11:22ZengMDPI AGBiomolecules2218-273X2025-03-0115337610.3390/biom15030376The Progress and Evolving Trends in Nucleic-Acid-Based TherapiesYunlong Liu0Chunmiao Wang1Xiuping Fu2Mengtian Ren3School of Chemistry and School of Life Sciences, Tiangong University, Tianjin 300387, ChinaSchool of Chemistry and School of Life Sciences, Tiangong University, Tianjin 300387, ChinaSchool of Chemistry and School of Life Sciences, Tiangong University, Tianjin 300387, ChinaSchool of Chemistry and School of Life Sciences, Tiangong University, Tianjin 300387, ChinaNucleic-acid-based therapies have emerged as a pivotal domain within contemporary biomedical science, marked by significant advancements in recent years. These innovative treatments primarily operate through the precise binding of DNA or RNA molecules to discrete target genes, subsequently suppressing the expression of the target proteins. The spectrum of nucleic-acid-based therapies encompasses antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), microRNAs (miRNAs), and messenger RNAs (mRNAs), etc. Compared to more traditional medicinal approaches, nucleic-acid-based therapies stand out for their highly targeted action on specific genes, as well as their potential for chemical modification to improve resistance to nucleases, ensuring sustained therapeutic activity and mitigating immunogenicity concerns. Nevertheless, these molecules’ limited cellular permeability necessitates the deployment of delivery vectors to enhance their intracellular uptake and stability. As nucleic-acid-based therapies progressively display promising pharmacodynamic profiles, there has been a burgeoning interest in these treatments for applications in clinical research. This review aims to summarize the variety of nucleic acid drugs and their mechanisms, evaluate the present status in research and application, discourse on prospective trends, and potential challenges ahead. These innovative therapeutics are anticipated to assume a pivotal role in the management of a wide array of diseases.https://www.mdpi.com/2218-273X/15/3/376nucleic-acid-based therapeuticsnucleic acid drugs’ mechanismschemical modificationdelivery systemsclinical usetrends and challenges |
| spellingShingle | Yunlong Liu Chunmiao Wang Xiuping Fu Mengtian Ren The Progress and Evolving Trends in Nucleic-Acid-Based Therapies Biomolecules nucleic-acid-based therapeutics nucleic acid drugs’ mechanisms chemical modification delivery systems clinical use trends and challenges |
| title | The Progress and Evolving Trends in Nucleic-Acid-Based Therapies |
| title_full | The Progress and Evolving Trends in Nucleic-Acid-Based Therapies |
| title_fullStr | The Progress and Evolving Trends in Nucleic-Acid-Based Therapies |
| title_full_unstemmed | The Progress and Evolving Trends in Nucleic-Acid-Based Therapies |
| title_short | The Progress and Evolving Trends in Nucleic-Acid-Based Therapies |
| title_sort | progress and evolving trends in nucleic acid based therapies |
| topic | nucleic-acid-based therapeutics nucleic acid drugs’ mechanisms chemical modification delivery systems clinical use trends and challenges |
| url | https://www.mdpi.com/2218-273X/15/3/376 |
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