International Journal of
MediPharm Research

The last decade has witnessed a paradigm shift in genetic medicine, transitioning the field of gene editing and gene therapy from a conceptual framework to a robust clinical reality. Innovations spanning genome editing precision (e.g., base and prime editing), enhanced delivery mechanisms (e.g., next-generation AAV vectors and lipid nanoparticles), and expansion of therapeutic targets have accelerated this progress. This review critically examines the technological advancements underpinning this transition and highlights their transformative clinical applications across inherited genetic disorders, oncology, and infectious and immune diseases. Specifically, we discuss the evolution of CRISPR-Cas systems and their competitors, the optimization of in vivo and ex vivo delivery platforms, and the emerging success of treatments like CAR T-cell therapy and liver editing. While the potential of gene therapy as a pillar of precision medicine is undeniable, its equitable and safe deployment hinges on continued technological refinement, robust clinical trial design, and the parallel development of comprehensive ethical and regulatory frameworks to manage accessibility and long-term safety. Keywords: Gene editing; CRISPR-Cas systems; base editing; prime editing; CAR-T therapy; gene delivery; off target effects; epigenetic editing; Wiskott-Aldrich syndrome; sickle cell disease; rare genetic disorders; immune therapy; precision medicine; clinical translation.