The revolution of clustered regularly interspaced short palindromic repeats -based therapies: From genetic diseases to cancer investigating how clustered regularly interspaced short palindromic repeats technology is being applied to treat genetic disorder
Keywords:
Cancer treatment, clustered regularly interspaced short palindromic repeats technology, gene therapy, genetic disorders, genome editingAbstract
The advent of clustered regularly interspaced short palindromic repeats (CRISPR) technology has revolutionized the field of genetic engineering and molecular biology, offering unprecedented opportunities for precise genome editing. Initially discovered as a natural defense mechanism in bacteria, CRISPR-Cas9 technology enables targeted modifications of DNA, paving the way for significant advancements in gene therapy. This review article explores the multifaceted applications of CRISPR, focusing on its transformative potential in treating genetic diseases and various forms of cancer. We delve into the mechanisms by which CRISPR operates, emphasizing its dual-step process of targeting and cutting DNA, and highlighting its versatility in investigating gene functions and developing therapeutic strategies. Specific applications in genetic disorders, including sickle cell disease and cystic fibrosis, showcase the promising outcomes of ongoing pre-clinical and clinical trials. In the realm of oncology, CRISPR’s ability to edit oncogenes and enhance immune cell functions exemplifies its role in developing personalized cancer therapies. However, the rapid advancement of CRISPR technology also necessitates critical discussions around ethical considerations and regulatory challenges, particularly concerning off-target effects and the implications of germline editing. Looking ahead, innovations such as CRISPR 2.0, base editing, and prime editing are set to further expand the therapeutic landscape, enhancing the precision and safety of gene editing. This article underscores the importance of collaborative efforts across scientific, ethical, and regulatory domains to navigate the complexities of CRISPR technology while maximizing its benefits for patients and society.