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Gene therapy is the delivery of exogenous DNA for the treatment of human disease (Friedmann and Roblin 1972). The advantage of gene therapy over other treatments for IRDs is the potentially curative effect of replacing, repairing, or silencing the responsible gene defect. The European Medicines Agency and the American Food and Drug Administration (FDA) approved the first gene therapy products in 2012 and 2017, respectively. Among them was voretigene neparvovec-rzyl (Luxturna), a revolutionary step in the treatment of IRDs.

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“Seeing Stars”: The Gene Therapy Revolution

  • 章节

  • 发表日期：

    2025年

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  • Gareth D. Mercer,
  • Brian G. Ballios,
  • Peter J. Kertes

摘要

Gene therapy is the delivery of exogenous DNA for the treatment of human disease (Friedmann and Roblin 1972). The advantage of gene therapy over other treatments for IRDs is the potentially curative effect of replacing, repairing, or silencing the responsible gene defect. The European Medicines Agency and the American Food and Drug Administration (FDA) approved the first gene therapy products in 2012 and 2017, respectively. Among them was voretigene neparvovec-rzyl (Luxturna), a revolutionary step in the treatment of IRDs.