1. Gene Therapy Biology 11: Section 6.5

2. Learning Goals Students will describe gene therapy Students will describe the process of gene insertion

3. What is it? Process by which defective genes in a genome are corrected with a normal copy of the gene Still in the experimental stages No gene therapy product is for sale

4. Example of Genetic Diseases Diabetes (type 1) Haemophilia Cystic fibrosis Sickle cell anemia

5. Gene Therapy This technology has the potential for correcting defective genes 3 possible strategies: 1.Gene insertion 2.Gene modification 3.Gene surgery

6. Gene Insertion A normal gene is inserted into position on a chromosome to replace a defective gene Accomplished using a virus or bacterial agent to “deliver” the DNA

7. Process of Gene Insertion 1.Virus DNA altered so they are harmless 2.Normal human gene placed inside virus and incorporated into viral DNA 3.Viruses used to infect target cells – hopefully normal human DNA will be inserted into cells’ genomes

8. Gene Modification Expose the defective gene to chemicals to attempt to recode the genetic message

9. Gene Surgery The defective gene is physically removed A healthy gene is put in as a replacement

10. Successfully Gene Therapy 1.Inherited blindness in dogs 2.Duchenne Muscular Dystrophy in mice 3.Deafness in guinea pigs

11. To Do: Read section 6.5 Questions 1-3, 5 on page 251 Write a paragraph considering the ethical issues involved in gene therapy