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Gene Therapy: “Mr. Fix-it” for Cells What is a Gene? • The “blueprint” for our bodies comes in the form of chromosomes, made out of DNA Question: What does DNA stand for? • DNA encodes lots and lots of different genes (pronounced “jeans”), which specify hair and eye colour, height, size of feet, etc. Genes and Diseases • “faulty” or missing genes cause disease • Genetic conditions used to be considered a “life sentence” Is this still the case?? Gene Therapy • Gene Therapy: Treating a genetic disease by inserting a healthy version of the missing or defective gene into a patient’s cells Could it be True? True or False: 1. Scientists have used gene therapy to successfully restore sight to blind dogs 2. Scientists used gene therapy to make mice “smarter” What do you think? How Can You Get Genes into Cells? • Viruses (including perhaps HIV) Viral genes inserted into target cell http://www.tulane.edu/~dmsander/WWW/224/Classification224.html How Can You Get Genes into Cells? • Other weird and wacky ways of putting healthy genes into cells….. – Liposomes (“mini cells”) Liposome – Electroporation (making cell membrane more “holey” to allow genetic material to enter cytoplasm) Does Gene Therapy Work? • Success in treatment of SCID in two infants – “bubble boy” syndrome • Viruses used to deliver functional gene Movie: Bubble Boy (2001) Another Question…… Which of the following diseases are current or potential targets of gene therapy? a. Cancer b. AIDS c. Diabetes d. Parkinson’s Disease Ethical Considerations (William Bates, 2001) What Lies Ahead? • Formation of groups with 4 or 5 students/group to participate in: – Gene therapy scavenger hunt – Gene therapy debate