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Life sciences · Journal article
Medicine and Health Sciences · November 6, 2026
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Gene therapy and genome editing technologies have revolutionized modern medicine by allowing for the precise correction of genetic defects and novel therapeutic approaches for diseases that were previously considered untreatable. It is a technique that treats or prevents various diseases by replacing, correcting or inactivating faulty genes. Among these technologies, CRISPR-Cas systems have emerged as particularly simple, flexible, and rapidly expanding in their clinical applications. This review aims to synthesize the latest advances in gene therapy and CRISPR technologies, including mechanisms, delivery systems, therapeutic applications, clinical trials, challenges, and future perspectives. Recent developments such as base editing and prime editing have significantly improved the accuracy and safety of genome modification approaches. In addition, advances in viral and non-viral delivery systems have enhanced the efficiency of therapeutic gene transfer into target tissues. Current clinical trials have demonstrated promising outcomes in the treatment of inherited blood disorders, cancers, and metabolic diseases. The integration of artificial intelligence and bioinformatics tools is also improving guided RNA design and minimizing off-target effects in genome editing applications. Ethical considerations and regulatory frameworks remain essential for ensuring the safe and equitable implementation of these technologies in clinical practice. We conclude that despite the hurdles, high costs, long term safety monitoring, and immune system reactions to vectors, CRISPR-based strategies are on the verge of revolutionizing the practice of medicine with innovative, accurate solutions to hard hereditary, oncologic, and infectious diseases.