Theoretical and Natural Science
- The Open Access Proceedings Series for Conferences
Theoretical and Natural Science
Vol. 15, 04 December 2023
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The application of antisense oligonucleotides in disease treatment
* Author to whom correspondence should be addressed.
Abstract
Scientific research in recent years has made every endeavor to combat against various untreatable diseases in neurodegenerative or genetic diseases under the guidance of constantly updated underlying gene-associated pathological mechanisms. However, substantial therapeutic challenges still remain and many original causes of diseases are under great debate. Problems of drugs for neurological disorders involve high resistance to drug molecules by the blood-brain barrier (BBB) and only work efficiently in certain populations of patients, which is also a side effect of genetic malady. The introduction of Antisense oligonucleotides (ASOs) brings a new era for these diseases and meets the requirement. The US Food and Drug Administration (FDA) has proven ten oligonucleotide drugs until 2020. This article reviews some of their clinical trials with mechanisms, up-to-date attempts in inventing new ASO therapy, continuous safety and efficacy in preclinical and clinical trials of proven ASO, and great potential usage for RNA virus therapy.
Keywords
Antisense Oligonucleotides, Neurodegenerative Disease, Genetic Diseases
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Data Availability
The datasets used and/or analyzed during the current study will be available from the authors upon reasonable request.
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- Volume Title
- Proceedings of the 2nd International Conference on Modern Medicine and Global Health
- ISBN (Print)
- 978-1-83558-193-3
- ISBN (Online)
- 978-1-83558-194-0
- Published Date
- 04 December 2023
- Series
- Theoretical and Natural Science
- ISSN (Print)
- 2753-8818
- ISSN (Online)
- 2753-8826
- DOI
- 10.54254/2753-8818/15/20240457
- Copyright
- 04 December 2023
- Open Access
- This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited