Technology and Applications of Genome Editing in Biomedicine

A special issue of Biomedicines (ISSN 2227-9059). This special issue belongs to the section "Molecular Genetics and Genetic Diseases".

Deadline for manuscript submissions: 30 June 2026 | Viewed by 9

Special Issue Editor


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Guest Editor
Department of Pediatrics, Stanford University School of Medicine, Palo Alto, CA 94305, USA
Interests: gene editing; RNA therapeutics; nucleic acid modifications; precision medicine

Special Issue Information

Dear Colleagues,

RNA-based therapies have rapidly evolved into a transformative class of medicines, offering new strategies for treating genetic disorders, infectious diseases, and cancer. Recent breakthroughs in messenger RNA (mRNA) vaccines, antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and microRNA modulators have highlighted the potential of RNA molecules as highly versatile therapeutic agents. In parallel, RNA plays a central role in genome editing technologies, with guide RNAs (gRNAs) enabling CRISPR/Cas systems to achieve precise and programmable genetic modifications. These developments illustrate the expanding landscape of RNA as both a therapeutic molecule and a tool for editing and regulation.

In this Special Issue, we will bring together cutting-edge research on RNA-based therapies and RNA-enabled genome editing, with a particular emphasis on the role of chemical and structural modifications in expanding their utility. Topics of interest include design principles for therapeutic RNA molecules, advances in nucleic acid backbone and base modifications, approaches for improving stability and pharmacokinetics, innovative delivery technologies, CRISPR-based applications, and disease-specific studies. We also welcome contributions addressing regulatory mechanisms, safety considerations, and translational insights.

By collecting these contributions, this Special Issue will provide a comprehensive overview of current progress and future directions in RNA therapeutics, nucleic acid modifications, and genome editing, fostering dialogue between basic researchers, clinicians, and industry innovators.

Dr. Guanhua Xun
Guest Editor

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Keywords

  • RNA therapeutics
  • mRNA vaccines
  • antisense oligonucleotides (ASOs)
  • siRNA and miRNA therapeutics
  • nucleic acid modifications
  • chemical stability and delivery
  • gene regulation therapy
  • precision medicine
  • CRISPR/Cas systems
  • guide RNA (gRNA)
  • genome editing

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Published Papers

This special issue is now open for submission.
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