Gene Therapy in the Treatment of Inherited Genetic Disorders: Current Developments and Challenges

Abstract

Gene therapy has emerged as a groundbreaking approach to address inherited genetic disorders, offering the potential to target and correct genetic mutations at the cellular level. This innovative technique involves altering the genetic material within a patient’s cells to correct mutations responsible for diseases such as cystic fibrosis, muscular dystrophy, and hemophilia. Advancements in technologies like CRISPR-Cas9 have significantly improved the accuracy and efficiency of DNA editing, allowing for more precise interventions. While early clinical trials have shown promising results, numerous challenges remain in the widespread application of gene therapy. Key obstacles include the safe and effective delivery of genetic material to target cells, ensuring long-term therapeutic effects, and minimizing off-target genetic alterations. Additionally, the high cost of gene therapies, regulatory issues, and ethical debates surrounding genetic modifications pose significant barriers. Despite these challenges, ongoing research continues to explore solutions, including advancements in gene delivery methods and personalized therapies, which offer hope for more accessible and effective treatments. This review explores the current state of gene therapy for inherited genetic disorders, highlighting recent advancements, challenges, and ethical considerations, and discusses the future prospects of this transformative therapeutic approach.

Keywords: Gene Therapy; Inherited Genetic Disorders; CRISPR-Cas9; DNA Editing; Genetic Mutations; Clinical Challenges; Gene Delivery; Ethical Issues; Genetic Modification

Citation

Haider R, Ahmed Z, Zameer S. Gene Therapy in the Treatment of Inherited Genetic Disorders: Current Developments and Challenges. WebLog J Oncol. wjo.2025.i2407. https://doi.org/10.5281/zenodo.17443118