Gene Therapy That Works

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Science  23 Aug 2013:
Vol. 341, Issue 6148, pp. 853-855
DOI: 10.1126/science.1242551

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The concept of gene therapy is disarmingly simple: Introduce a healthy gene in a patient and its product should alleviate the defect caused by a faulty gene or slow the progression of disease (1). Why then, over the past three decades, have there been so few clinical successes in treating patients with this approach? A major obstacle has been the delivery of genes to the appropriate cell, tissue, and organ. How does one introduce a gene into the brain with trillions of cells, or the liver with billions of cells, or the rare hematopoietic adult stem cell that has the potential to populate all lineages of lymphoid and myeloid cells? Much effort has been devoted to finding ways to efficiently deliver a therapeutic gene to the desired cell type, resulting in sustained production of the gene product, ideally through the entire life of the recipient, without unwanted side effects like genotoxicity or unsettling the immune balance (2). On pages 864 and 865 in this issue, Biffi et al. (3) and Aiuti et al. (4) report encouraging results using lentivirus-mediated gene therapy to treat children with rare genetic defects.