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Gene therapy returns to centre stage

Author

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  • Luigi Naldini

    (San Raffaele Telethon Institute for Gene Therapy (TIGET), San Raffaele Scientific Institute
    Vita Salute San Raffaele University)

Abstract

Recent clinical trials of gene therapy have shown remarkable therapeutic benefits and an excellent safety record. They provide evidence for the long-sought promise of gene therapy to deliver 'cures' for some otherwise terminal or severely disabling conditions. Behind these advances lie improved vector designs that enable the safe delivery of therapeutic genes to specific cells. Technologies for editing genes and correcting inherited mutations, the engagement of stem cells to regenerate tissues and the effective exploitation of powerful immune responses to fight cancer are also contributing to the revitalization of gene therapy.

Suggested Citation

  • Luigi Naldini, 2015. "Gene therapy returns to centre stage," Nature, Nature, vol. 526(7573), pages 351-360, October.
    • Handle: RePEc:nat:nature:v:526:y:2015:i:7573:d:10.1038_nature15818
    DOI: 10.1038/nature15818
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    Cited by:

    1. Jifeng Yu & Bangguo Zhou & Shen Zhang & Haohao Yin & Liping Sun & Yinying Pu & Boyang Zhou & Yikang Sun & Xiaolong Li & Yan Fang & Lifan Wang & Chongke Zhao & Dou Du & Yan Zhang & Huixiong Xu, 2022. "Design of a self-driven probiotic-CRISPR/Cas9 nanosystem for sono-immunometabolic cancer therapy," Nature Communications, Nature, vol. 13(1), pages 1-20, December.
    2. Xin Wu & Yuanhuan Yu & Meiyan Wang & Di Dai & Jianli Yin & Wenjing Liu & Deqiang Kong & Shasha Tang & Meiyao Meng & Tian Gao & Yuanjin Zhang & Yang Zhou & Ningzi Guan & Shangang Zhao & Haifeng Ye, 2024. "AAV-delivered muscone-induced transgene system for treating chronic diseases in mice via inhalation," Nature Communications, Nature, vol. 15(1), pages 1-16, December.
    3. Tinglong Dai & Sridhar Tayur, 2020. "OM Forum—Healthcare Operations Management: A Snapshot of Emerging Research," Manufacturing & Service Operations Management, INFORMS, vol. 22(5), pages 869-887, September.
    4. Aaldering, Lukas Jan & Leker, Jens & Song, Chie Hoon, 2019. "Uncovering the dynamics of market convergence through M&A," Technological Forecasting and Social Change, Elsevier, vol. 138(C), pages 95-114.
    5. Haobo Han & Jiakai Xing & Wenqi Chen & Jiaxin Jia & Quanshun Li, 2023. "Fluorinated polyamidoamine dendrimer-mediated miR-23b delivery for the treatment of experimental rheumatoid arthritis in rats," Nature Communications, Nature, vol. 14(1), pages 1-20, December.

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