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What are the five criteria for a gene therapy candidate disease?
Wiki User
∙ 11y ago
1. Does the condition result from mutations in one or more genes?
2. Which genes are involved?
3. What do you know about the biology of the disorder?
4. Will adding a normal copy of the gene fix the problem in the affected tissue?
5. Can you deliver the gene to cells of the affected tissue?
There ya go(:
Wiki User
∙ 11y ago
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