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<p>Rapid progress in the development of genome editing has expanded the method spectrum for correcting disease-causing mutations in gene therapy. While allowing clinically relevant functional restoration of defective genes, each gene therapy method requires extensive efficacy and safety assessment prior to adoption in clinical practice. Here, we illustrate these issues with the example of chronic granulomatous disease, which has been the object of gene therapy development over three decades.</p>

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Sichere Gentherapie für Septische Granulomatose

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2025年6月18日

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  • Kah Mun Siow,
  • Ute Modlich,
  • Janine Reichenbach

摘要

Rapid progress in the development of genome editing has expanded the method spectrum for correcting disease-causing mutations in gene therapy. While allowing clinically relevant functional restoration of defective genes, each gene therapy method requires extensive efficacy and safety assessment prior to adoption in clinical practice. Here, we illustrate these issues with the example of chronic granulomatous disease, which has been the object of gene therapy development over three decades.