Nucleic Acid-Based Therapeutics: Translating Genetic Code into Clinical Applications

Nucleic Acid-Based Therapeutics: Translating Genetic Code into Clinical Applications
Mousarreza Shiri,¹ Mohammadreza Sharifi,^2,*
1. Department of Genetics and Molecular Biology, School of Medicine, Isfahan University of Medical Sciences, Isfahan, Iran
2. Department of Genetics and Molecular Biology, School of Medicine, Isfahan University of Medical Sciences, Isfahan, Iran
Introduction: Nucleic acids, being the fundamental components of genetic information, have an essential role in regulating biological functions. In recent decades, they have garnered increased attention in the domain of advanced therapies, particularly as nucleic acid-based drugs (NADs).
Methods: These pharmaceuticals employ precise gene engineering, facilitating targeted molecular treatments, rapid development, and the potential to address cancer, viral infections, and genetic abnormalities. A quick design process, high adaptability, and the potential to treat diseases that were previously incurable are some of the primary advantages.
Results: The immune system being stimulated, intracellular dispersion limitations, and instability in biological environments are some of the remaining barriers to the widespread clinical use of these medications. Novel methodologies, encompassing chemical alterations, efficient nanocarriers, and precise drug delivery systems, have been employed to tackle these issues. The FDA approved several RNA-based therapeutics, including Patisiran, Onpattro, and mRNA vaccines for COVID-19, indicating substantial advancement in this field.
Conclusion: This comprehensive review examines the roles of NADs, categorizes RNA-based therapies, and determines their importance in the therapy of diseases.
Keywords: Nucleic acids, Nucleic acid-based drugs, RNA therapeutics, Targeted therapy, Gene engineering