Gene-based medicine represents a groundbreaking shift in modern healthcare, offering precise and personalized therapeutic approaches by targeting the genetic foundations of diseases. This field has seen remarkable advancements, from gene editing technologies such as CRISPR-Cas9 to novel gene therapies for inherited and acquired conditions. The approval of multiple gene-based treatments, including those for rare genetic disorders, cancers, and neurological diseases, has opened new doors for clinical applications. Despite these advancements, challenges remain, including delivery mechanisms, immune responses, ethical considerations, and large-scale manufacturing. Understanding the latest innovations, their applications, and the hurdles faced in translating research into clinical practice is crucial for the continued success of gene-based medicine.

This special issue aims to highlight cutting-edge research and clinical advancements in gene-based medicine. We welcome contributions in the following areas:   

Gene Therapy Development

Genetic and Genomic Approaches

Targeted Gene Delivery Systems

Personalized Medicine & Pharmacogenomics

Clinical Applications and Case Studies

Ethical, Regulatory, and Safety Considerations

Keywords: genetics; genomics; biochemistry; molecular biology; personalized medicine