Gene therapy aims to treat genetic diseases by introducing genetic material into cells to correct medical conditions or enhance health. This involves using nucleic acids, viruses, or genetically modified microorganisms to integrate therapeutic genetic material into the recipient’s genome. Recent advancements have significantly progressed gene therapy, focusing on treating inherited and acquired diseases like cancer and cardiovascular ailments through clinical trials. Overcoming delivery efficiency and immune reaction challenges is crucial for widespread clinical use. Gene therapy is categorized into germline and somatic gene transplantation, with somatic alterations not being passed to future generations. Rapid developments in molecular biology since the 1990s have expanded gene therapy’s potential to address genetic defects and various diseases, with promising results from animal experiments leading to human trials.