• Advancing Medical Genetics: The Role of CRISPR-Cas9 in Treating Genetic Disorders
  • Nafiseh Salehi Kakhki,1,*
    1. Department of Biology, Islamic Azad University Mashhad Branch, Iran


  • Introduction: CRISPR-Cas9, a groundbreaking gene-editing technology, has revolutionized the field of medical genetics, offering unprecedented possibilities for treating genetic disorders. This technology allows for precise modifications of the DNA sequence, enabling the correction of mutations that cause various inherited diseases. As research progresses, CRISPR-Cas9 is increasingly being explored as a tool for developing personalized therapies, potentially providing cures for conditions previously considered untreatable.
  • Methods: This review examines recent studies focused on the application of CRISPR-Cas9 in treating human genetic diseases. We conducted an extensive search of literature from the past decade, selecting studies that demonstrate the use of CRISPR-Cas9 in correcting genetic mutations in diseases such as cystic fibrosis, sickle cell anemia, and Duchenne muscular dystrophy. These studies were analyzed to identify key trends, methodological advancements, and challenges in translating gene-editing technology from the laboratory to clinical practice.
  • Results: Our analysis reveals significant progress in the application of CRISPR-Cas9 for gene therapy. For instance, several studies have successfully corrected genetic defects in preclinical models, demonstrating the potential of CRISPR-Cas9 to cure hereditary diseases. Moreover, the development of enhanced delivery systems has improved the efficiency and specificity of gene editing. However, the field still faces challenges, such as off-target effects, ethical concerns regarding germline editing, and the need for robust regulatory frameworks.
  • Conclusion: CRISPR-Cas9 holds immense promise for the future of personalized medicine by providing targeted treatments for genetic disorders. To fully realize its potential, continued research and careful consideration of ethical implications are essential as the technology moves closer to clinical application.
  • Keywords: Medical Genetics , CRISPR-Cas9 , Gene Editing , Genetic Disorders , Personalized Medicine