Gene Therapy: Advancements, Challenges, and Future Directions

Gene therapy represents a groundbreaking approach in modern medicine that aims to treat or prevent diseases by modifying genetic material within a patient’s cells. Over the past decades, remarkable advancements have emerged, including viral and non viral vectors, CRISPR based gene editing, and successful treatments for inherited disorders such as spinal muscular atrophy and certain immunodeficiencies. Despite its great promise, challenges such as immune responses, off target effects, delivery efficiency, and ethical concerns remain significant. This article provides a concise overview of gene therapy, highlighting its principles, therapeutic mechanisms, major applications, and the current limitations that shape future research directions.