By correcting the exact causal genetic defect present in the patient, gene therapy represents perhaps the most direct form of precision medicine. Examples include delivery of genes to replace mutated genes, antisense RNA technology, and most recently genome editing (e.g., zinc finger nucleases, TALENs, and CRISPRs). Will and should gene therapy be a focus of the Precision Medicine Initiative? What are some opportunities on the horizon for gene therapy?
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