Journal
ACTA PHARMACEUTICA SINICA B
Volume 11, Issue 8, Pages 2150-2171Publisher
INST MATERIA MEDICA, CHINESE ACAD MEDICAL SCIENCES
DOI: 10.1016/j.apsb.2021.05.020
Keywords
CRISPR; Cas9; Genome editing therapy; LNP; Nanoparticle; AAV; Lentivirus; In vivo
Categories
Funding
- Summer Undergraduate Research Fellowship (SURF) Program at MCPHS University (USA)
- School of Pharmacy e Boston, MCPHS University
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CRISPR-Cas9-based genome editing has advanced to human clinical trials within less than a decade, but the low efficiency of in vivo delivery needs to be enhanced in order to fully realize its therapeutic potential.
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. (C) 2021 Chinese Pharmaceutical Association and Institute of Materia Medica, Chinese Academy of Medical Sciences. Production and hosting by Elsevier B.V.
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