Abstract

Gene therapy represents a transformative leap in medical science, offering potential cures for previously untreatable genetic disorders by directly modifying the genetic  material of patients. This article explores the scientific principles underpinning gene therapy, including the mechanisms of gene delivery, gene editing techniques, and the ethical considerations involved. Recent advancements in technologies such as CRISPR/Cas9 have dramatically expanded the possibilities of gene therapy, enabling precise modifications to the genome with unprecedented accuracy. The article also examines ongoing clinical trials and the future directions of gene therapy, emphasizing the promise and challenges associated with this revolutionary approach.