In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges

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 in vivo delivery must be enhanced before its therapeutic potential can be fully realized. Here we discuss the most recent progress of in vivo delivery of CRISPR-Cas9 systems, highlight innovative viral and non-viral delivery technologies, emphasize outstanding delivery challenges, and provide the most updated perspectives.

Keywords: AAV; CRISPR; Cas9; Genome editing therapy; In vivo; LNP; Lentivirus; Nanoparticle.

Publication types

  • Review