Gene Therapy

• Gene

  a part of a cell in a living thing that controls what it looks like, how it grows and how it develop
• Genetic Disorder

   a disease that is caused by an abnormality in an individual’s DNA
• Genetic diseases 

  are treated by repairing the damaged gene responsible for the disease. Through the introduction of a normal copy of the gene instead of using drugs or surgery, the cells can produce the normal functional protein.
• Gene therapy

  When a gene is mutated, the gene that codes for the normal protein is altered and results in producing an abnormal or non-functional protein
• Gene therapy

  is the introduction of genes into existing cells to prevent or cure a wide range of diseases.
• Gene therapy

  is a technique for correcting defective genes responsible for disease development.
• Gene therapy

  Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
• Ashanti DeSilva – received the first approved gene therapy experiment due to ADA-SCID on September 14, 1990 in USA
• ADA-SCID – (Adenosine Deaminase Deficiency) an inherited disorder that damages the immune system causing severe combined immunodeficiency (SCID) leading to repeated and persistent infections that can be very serious or life-threatening and is characterized by pneumonia, chronic