Abstract

Dr. C.D. Patil, Ms. Komal Digambar Gangurde*

ABSTRACT

1.1 Overview of Gene Therapy Definition and Principles Gene therapy is a therapeutic approach that involves the introduction, removal, or modification of genetic material within a patient’s cells to treat or prevent disease. The fundamental principle is to correct or regulate defective genes responsible for disease pathogenesis rather than merely treating symptoms. The concept was first formally proposed in the 1970s and later advanced through recombinant DNA technology and molecular biology innovations Gene therapy works by delivering nucleic acids—such as DNA or RNA—into target cells using viral or non-viral vectors. The therapeutic gene must reach the appropriate intracellular compartment (nucleus or cytoplasm) to exert its function. Efficient gene delivery requires overcoming multiple biological barriers, including enzymatic degradation, immune recognition, cellular uptake, and endosomal escape.