Editorial: Recent Advancements in RNA-based and targeted Therapeutics

Yiming Meng ¹ Viola Bianca Serio ^2,3 Yuquan Tong ⁴ Tao Huang ⁵ Elisa Frullanti ^2,3* Maria Palmieri ^2,3

• ¹ Liaoning Cancer Hospital, China Medical University, Shenyang, Liaoning Province, China
• ² Cancer Genomics and Systems Biology Lab, Department of Medical Biotechnology, University of Siena, Siena, Tuscany, Italy
• ³ Med Biotech Hub and Competence Center, Department of Medical Biotechnology, University of Siena, Siena, Tuscany, Italy
• ⁴ The Scripps Research Institute, La Jolla, California, United States
• ⁵ Shanghai Institute of Nutrition and Health, Chinese Academy of Sciences (CAS), Shanghai, Shanxi Province, China

In the early 1980s, the development and application of antisense oligonucleotides that could specifically bind to messenger RNA (mRNA) and inhibit protein synthesis represented a groundbreaking advancement in molecular biology. These synthetic, single-stranded nucleic acid sequences were designed to complement target mRNA strands, thereby blocking their translation into proteins. This innovative approach not only provided a powerful tool for studying gene expression and function but also promoted the rapid advances of RNA-based therapeutics (Zhu Y, 2022).RNA epigenetic modifications have been uncovered as not only an intermediary structure between DNA and protein or an effector molecule, but it plays crucial roles in post-transcriptional gene regulation. As happens with targeted therapy in DNA blocking specific molecular signals that promote tumor growth and improve treatment efficacy reducing side effects (Rina A, 2024), the functional and structural diversity of RNA promotes its applications in therapeutics. Several RNA-based medications have been approved for clinical use, while others are still under investigation or preclinical trials.Numerous studies have established the critical role of RNA in a broad range of diseases including COVID-19, neurological diseases, and various cancers.RNA-based gene therapy requires therapeutic RNA to function inside target cells without eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug delivery systems, which circumvent the limitations of viral delivery vectors (Paunovska K, 2022).Therefore, targeting RNA harbors profound potential to expand the "druggability" of the human genome. On the other hand, the development of small interfering RNA (siRNA), RNA interferences (RNAi), and mRNA vaccines have shown RNA as not only disease targets but also therapeutics themselves. Collectively, the complex yet important role of RNA demands further studies from multiple aspects. This Research Topic focused on the recent advances in the field of RNA-related research with a highlight on translational values to accelerate the development of RNA-based and RNA-targeted biomedical applications in the field of epigenomics and epigenetics. In conclusion, these studies demonstrate that RNA represents a novel and promising therapeutic target, with potential applications in a wide range of diseases. Research continues to explore and develop these strategies to improve clinical outcomes and address current therapeutic challenges. RNA studies will continue to be a dynamic and rapidly evolving field. Collaboration between scientists, clinicians and industry will be essential to translate basic discoveries into practical clinical applications, thus improving human health, for this reason it is essential to continue investing in this field to fully exploit its possibilities.