Diseases treated by Gene Therapy

Replace a defective gene, provide targeted and long-lasting treatments

Repair mutated gene or to modify gene expression.

1.Genetic Disorders:
• Cystic Fibrosis 2. Acquired Disorders:
• Hemophilia • Cancer
• Sever combined immunodeficiency • HIV/AIDS
(SCID) • Cardiovascular
• Chronic Granulomatus Disorder diseases
(CGD)
• Leber congenital amaurosis (LCA)
 Advantages
• Gene therapy can provide hope for patients with incurable
or life-threatening diseases
• Gene therapy can improve the quality of life and survival of
patients
• Gene therapy can reduce the need for other interventions
• Gene therapy can prevent the transmission of genetic
diseases to future generations
 Disadvantages

• Unwanted immune system reaction

• Targeting the wrong cells
• Gene mutations
• Ethical issues