CRISPR-Cas9疗法的递送：进展与挑战。

Behr Matthew, Zhou Jing, Xu Bing, Zhang Hongwei

Department of Pharmaceutical Sciences, School of Pharmacy-Boston, Massachusetts College of Pharmacy and Health Sciences, Boston, MA 02115, USA.

Department of Endocrinology, Tangshan Hospital of Traditional Chinese Medicine, Tangshan 063000, China.

Acta Pharm Sin B. 2021 Aug;11(8):2150-2171. doi: 10.1016/j.apsb.2021.05.020. Epub 2021 May 26.

Within less than a decade since its inception, CRISPR-Cas9-based genome editing has been rapidly advanced to human clinical trials in multiple disease areas. Although it is highly anticipated that this revolutionary technology will bring novel therapeutic modalities to many diseases by precisely manipulating cellular DNA sequences, the low efficiency of delivery must be enhanced before its therapeutic potential can be fully realized. Here we discuss the most recent progress of delivery of CRISPR-Cas9 systems, highlight innovative viral and non-viral delivery technologies, emphasize outstanding delivery challenges, and provide the most updated perspectives.

基于CRISPR-Cas9的基因组编辑技术自诞生以来不到十年的时间里，已迅速推进到多个疾病领域的人体临床试验。尽管人们高度期待这项革命性技术能够通过精确操纵细胞DNA序列为多种疾病带来新的治疗方式，但在其治疗潜力得以充分实现之前，必须提高其低递送效率。在此，我们讨论CRISPR-Cas9系统递送的最新进展，突出创新的病毒和非病毒递送技术，强调突出的递送挑战，并提供最新的观点。