Gene therapy holds potential for a wide range of genetic mutations including:
Single-Gene Disorders: Diseases caused by mutations in a single gene, such as Cystic Fibrosis, Sickle Cell Anemia, and Hemophilia. Multifactorial Disorders: Conditions resulting from a combination of multiple genetic and environmental factors, like some forms of cancer and heart disease. Mitochondrial Disorders: Genetic diseases caused by mutations in the mitochondrial DNA, such as Leber's Hereditary Optic Neuropathy.
