Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Components

被引：112
|
作者：
Kazemian, Pardis [1 ,2 ]
Yu, Si-Yue [3 ]
Thomson, Sarah B. [1 ,2 ]
Birkenshaw, Alexandra [3 ]
Leavitt, Blair R. [1 ,2 ]
Ross, Colin J. D. [3 ]
机构：
[1] Univ British Columbia, Fac Med, Dept Med Genet, Vancouver, BC V6T 1Z3, Canada
[2] BC Childrens Hosp Res Inst, Ctr Mol Med & Therapeut, Vancouver, BC V5Z 4H4, Canada
[3] Univ British Columbia, Fac Pharmaceut Sci, Vancouver, BC V6T 1Z3, Canada
来源：
MOLECULAR PHARMACEUTICS | 2022年 / 19卷 / 06期
基金：
加拿大健康研究院;
关键词：
lipid nanoparticles; CRISPR; Cas9; genome editing; nanomedicine; gene therapy; IN-VIVO DELIVERY; MESSENGER-RNA; INTRACELLULAR DELIVERY; SYSTEMIC DELIVERY; CATIONIC LIPIDS; BLOOD PROTEINS; HOMING PEPTIDE; GENE-THERAPY; LIPOSOMES; EFFICIENT;
D O I：
10.1021/acs.molpharmaceut.1c00916
中图分类号：
R-3 [医学研究方法]; R3 [基础医学];
学科分类号：
1001 ;
摘要：
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
引用
收藏
页码：1669 / 1686
页数：18
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