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3 Cards in this Set
- Front
- Back
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What is the goal of gene therapy?
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To provide a patient with a functional gene to counteract effects of a non-functional version.
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What are the three main methods used to select a disease as a possible target for gene therapy?
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1. Disease must be a result of defect in single gene.
2. The gene identified + purified in functional form. 3. Cells affected must be accessible for gene delivery. |
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What is an example of a disease in which gene therapy is being tested on?
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ADA-SCIDS, which is an immune system deficiency.
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