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Transcript of Gene Therapy
-"Turn On" or Activate the Gene
-Integrate the Gene
-Avoid Harmful side effects Steps to Take: - Some medical conditions are genetic mutations.
-"Gene Therapy" is when you replace mutated genes with healthy, regular genes. "In-Vivo"
Inject vector into the mutated area. "Ex-Vivo"
Removing cells, fix genes, culture more cells, and replace. How do you know whether a disorder is a good candidate for gene therapy? 1. Does the condition result from mutations in one or more genes? 2. Which genes are involved? 3. What do you or others know about the disorder? 4. Will adding a normal copy of the gene fix the problem? 5. Can you deliver the gene to cells of the affected area? What is your best mode of delivery? -Vector: Something that transfers genetic material from one cell to another. Visit learn.genetics.utah.edu and click on Gene Therapy. How can replacing genes
fix genetic mutations? Pro's and Cons of Gene Therapy * Gene Therapy could save lives
*Some of the most common diseases are genetic disorders.
* Once doctors know more about Gene Therapy they can use it more commonly. *Sometimes the vectors get attacked by the body's immune system, which can be deadly.
* Some doctors may want to use Gene Therapy to alter normal humans. Case Study: Cystic Fibrosis http://learn.genetics.utah.edu/content/tech/genetherapy/cysticfibrosis/