基因编辑试剂的治疗性体内递送。
Therapeutic in vivo delivery of gene editing agents.
机构信息
Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA.
Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA.
出版信息
Cell. 2022 Jul 21;185(15):2806-2827. doi: 10.1016/j.cell.2022.03.045. Epub 2022 Jul 6.
In vivo gene editing therapies offer the potential to treat the root causes of many genetic diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to safely and efficiently deliver gene editing agents to relevant organs and tissues in vivo. Here, we review current delivery technologies that have been used to enable therapeutic in vivo gene editing, including viral vectors, lipid nanoparticles, and virus-like particles. Since no single delivery modality is likely to be appropriate for every possible application, we compare the benefits and drawbacks of each method and highlight opportunities for future improvements.
体内基因编辑疗法为治疗许多遗传性疾病的根本原因提供了可能。要实现体内基因编辑治疗的潜力，就需要有能力将基因编辑试剂安全有效地递送到体内的相关器官和组织中。在这里，我们回顾了目前已被用于实现体内治疗性基因编辑的各种递药技术，包括病毒载体、脂质纳米粒和类病毒颗粒。由于没有任何单一的递药方式可能适用于每种可能的应用，因此我们比较了每种方法的优缺点，并强调了未来改进的机会。
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