THE IMPACT OF GENE THERAPY ON TREATING GENETIC DISORDERS

Abstract

Gene therapy represents a groundbreaking approach to the treatment of genetic disorders by directly addressing the root causes embedded in an individual's DNA. This article explores the scientific principles, methodologies, and advancements in gene therapy, highlighting its transformative impact on diseases such as cystic fibrosis, sickle cell anemia, and Duchenne muscular dystrophy. By employing techniques such as gene editing, viral vector delivery, and CRISPR-Cas9 technology, gene therapy has shown remarkable potential in correcting genetic mutations and restoring normal cellular function. Despite its promise, the field faces challenges, including ethical considerations, delivery system limitations, and potential off-target effects. This study examines the current landscape of gene therapy, analyzing its clinical applications, ongoing trials, and future prospects in combating genetic disorders