Gene therapy comes of age

Abstract

Gene therapy: The power of persistence Nearly 50 years after the concept was first proposed, gene therapy is now considered a promising treatment option for several human diseases. The path to success has been long and tortuous. Serious adverse effects were encountered in early clinical studies, but this fueled basic research that led to safer and more efficient gene transfer vectors. Gene therapy in various forms has produced clinical benefits in patients with blindness, neuromuscular disease, hemophilia, immunodeficiencies, and cancer. Dunbar et al. review the pioneering work that led the gene therapy field to its current state, describe gene-editing technologies that are expected to play a major role in the field's future, and discuss practical challenges in getting these therapies to patients who need them. Science , this issue p. eaan4672

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Affiliated Institutions

• Massachusetts General Hospital US
• National Heart Lung and Blood Institute US
• Harvard University US
• Spark Therapeutics (United States) US
• University of California, Los Angeles US
• The University of Tokyo JP
• Memorial Sloan Kettering Cancer Center US

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