Gene Therapy: Current Trends and Future Prospects in Treating Genetic Disorders

Resumen

Abstract:
Gene therapy has emerged as a transformative approach in the treatment of genetic disorders, leveraging advancements in molecular biology and genetic engineering. This therapy aims to address the root causes of genetic diseases by introducing, removing, or altering genetic material within a patient's cells. Recent innovations have led to the development of various vectors, such as viral and non-viral systems, which enhance the efficiency and specificity of gene delivery. Current trends highlight the use of CRISPR/Cas9 technology, which allows for precise genome editing, offering promising outcomes in treating conditions like sickle cell anemia and cystic fibrosis. Furthermore, advances in personalized medicine have paved the way for tailored gene therapies that consider individual genetic profiles, thus increasing treatment efficacy and reducing adverse effects. Clinical trials are underway to explore the potential of gene therapy for a broader range of disorders, including muscular dystrophies and neurodegenerative diseases. However, challenges such as immune responses, ethical considerations, and high costs remain significant hurdles to widespread implementation. Future prospects indicate a shift toward the integration of gene therapy with other therapeutic modalities, such as gene editing combined with stem cell therapy, which may enhance treatment outcomes. Moreover, regulatory frameworks are evolving to facilitate the approval and accessibility of gene therapies, ensuring that they reach patients in need. This review aims to provide a comprehensive overview of current trends and future directions in gene therapy, highlighting its potential to revolutionize the management of genetic disorders and improve patient outcomes.