Gene therapy is an advanced biotechnology-based treatment approach that uses genes to treat, prevent, or slow the progression of diseases. The main strategies include replacing a defective gene with a healthy version, silencing a malfunctioning gene, or introducing new genetic material into cells. FDA-approved applications already exist for conditions such as SMA, hemophilia, inherited retinal disorders, and certain cancers. Unlike conventional therapies, gene therapy directly targets the genetic root cause of disease.