In order for gene therapy to be most effective, genes should be inserted in
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Gene therapy is most effective when genes are inserted into stem cells because these cells divide and differentiate to form all types of specialized cells. So, the corrected gene can be passed on to many daughter cells, giving a long-lasting or even permanent therapeutic effect.
• RBCs: have no nucleus, so cannot express new genes
• WBCs: short-lived compared to stem cells
• Stem cells: best target for long-term correction
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