Advances in Gene and Nucleic Acid-Based Therapeutics: From Molecular Innovations to Potential Misuse

Dear Colleagues,

Recent advances in gene and nucleic acid-based therapeutics have rapidly expanded the scope of modern biomedical research and clinical practice. Breakthroughs in mRNA lipid nanoparticle (LNP) technologies, genome editing platforms such as CRISPR/Cas systems, and diverse nucleic acid modalities—including antisense oligonucleotides, siRNA, and miRNA—have enabled novel approaches to treating genetic, infectious, and complex diseases. In parallel, improvements in viral and non-viral delivery systems, as well as cell-based therapies such as engineered immune cells, have accelerated translation from bench to bedside. As these technologies mature, their potential applicability is extending beyond traditional therapeutic boundaries, raising important scientific and regulatory questions regarding appropriate use, including distinctions between treatment and performance enhancement, such as gene doping.

This Special Issue aims to provide a comprehensive and balanced overview of the recent progress in gene and nucleic acid-based therapeutics, spanning fundamental, preclinical, and clinical research in both human and animal systems. We welcome contributions addressing molecular design, delivery technologies, pharmacokinetics, safety, and therapeutic applications, including mRNA-based vaccines, genome editing, and regenerative medicine. In addition, this Special Issue encourages studies on analytical and detection methodologies relevant to gene doping and gene transfer, such as PCR- and sequencing-based detection of exogenous genetic elements, biomarker discovery, and data-driven approaches for identifying non-natural genomic alterations. Perspectives on regulatory science and frameworks that support the appropriate and responsible application of these technologies are also within scope.

Dr. Teruaki Tozaki
Guest Editor

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