Biotechnology

CRISPR & Gene Therapy Applications

CRISPR and gene therapy applications are redefining the treatment landscape by enabling precise correction or modulation of disease-causing genes. CRISPR-Cas systems allow targeted gene editing with high specificity, offering solutions for genetic disorders, cancer, and infectious diseases. Gene therapy employs viral or non-viral vectors to deliver functional genes to patients, addressing underlying genetic deficiencies rather than only managing symptoms. Integration with stem cells and ex vivo editing expands therapeutic potential and safety. Preclinical and clinical advancements have demonstrated efficacy in treating hemophilia, muscular dystrophy, and immune disorders. Ethical and regulatory considerations remain critical to ensure responsible implementation. Innovations in delivery systems, off-target mitigation, and base editing are enhancing therapeutic precision. Combining CRISPR with personalized medicine approaches allows patient-specific interventions, reducing adverse effects. These technologies collectively open transformative pathways for durable, curative therapies, offering unprecedented potential to alter disease trajectories and improve global health outcomes.