期刊
MOLECULAR PHARMACEUTICS
卷 19, 期 6, 页码 1669-1686出版社
AMER CHEMICAL SOC
DOI: 10.1021/acs.molpharmaceut.1c00916
关键词
lipid nanoparticles; CRISPR; Cas9; genome editing; nanomedicine; gene therapy
资金
- Canadian Institutes of Health Research (CIHR)
- Huntington Society of Canada (HSC)
- Nanomedicines Innovation Network (NMIN) [2019T2-05]
- Michael Smith Foundation for Health Research (MSFHR) Scholar Award [16458]
Gene editing mediated by CRISPR/Cas9 systems is a promising therapeutic option for genetic diseases and cancers. Lipid nanoparticles (LNPs), due to their low immunogenicity and flexibility, have emerged as an attractive nonviral delivery platform for CRISPR-mediated genome editing. The development of new, safe, and optimized LNP formulations has addressed the challenges in CRISPR delivery.
Gene editing mediated by CRISPR/Cas9 systems is due to become a beneficial therapeutic option for treating genetic diseases and some cancers. However, there are challenges in delivering CRISPR components which necessitate sophisticated delivery systems for safe and effective genome editing. Lipid nanoparticles (LNPs) have become an attractive nonviral delivery platform for CRISPR-mediated genome editing due to their low immunogenicity and application flexibility. In this review, we provide a background of CRISPR-mediated gene therapy, as well as LNPs and their applicable characteristics for delivering CRISPR components. We then highlight the challenges of CRISPR delivery, which have driven the significant development of new, safe, and optimized LNP formulations in the past decade. Finally, we discuss considerations for using LNPs to deliver CRISPR and future perspectives on clinical translation of LNP-CRISPR gene editing
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