Disease modeling (genome editing) is a research methodology that employs genome editing technologies to generate or modify biological models for studying human diseases. This approach involves making targeted genetic alterations in cell lines, induced pluripotent stem cells, organoids, or animal models to accurately recapitulate specific genetic mutations, pathways, or cellular dysfunctions associated with particular diseases, such as inherited metabolic disorders or neurodegenerative diseases. The generated models serve as critical tools for investigating disease mechanisms, identifying potential therapeutic targets, and evaluating the efficacy and safety of novel treatments, thereby accelerating translational research.