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

页码：1669 / 1686

页数：18

共 50 条

[1] Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics
Givens, Brittany E.
Naguib, Youssef W.
Geary, Sean M.
Devor, Eric J.
Salem, Aliasger K.
AAPS JOURNAL, 2018, 20 (06):
[2] Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics
Brittany E. Givens
Youssef W. Naguib
Sean M. Geary
Eric J. Devor
Aliasger K. Salem
The AAPS Journal, 20
[3] Nanoparticle Delivery of CRISPR/Cas9 for Genome Editing
Duan, Li
Ouyang, Kan
Xu, Xiao
Xu, Limei
Wen, Caining
Zhou, Xiaoying
Qin, Zhuan
Xu, Zhiyi
Sun, Wei
Liang, Yujie
FRONTIERS IN GENETICS, 2021, 12
[4] Nanoparticle-mediated delivery of the CRISPR/Cas9 system components into plant cell for genome editing
Makhotenko, A.
Makarov, V.
Snigir, E.
Khromov, A.
Makarova, S.
Kalinina, N.
Taliansky, M.
FEBS JOURNAL, 2017, 284 : 168 - 168
[5] Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing
Zhang, Song
Shen, Jiangtao
Li, Dali
Cheng, Yiyun
THERANOSTICS, 2021, 11 (02): : 614 - 648
[6] Modulating CRISPR/Cas9 genome-editing activity by small molecules
Chen, Siwei
Chen, Deng
Liu, Bin
Haisma, Hidde J.
DRUG DISCOVERY TODAY, 2022, 27 (04) : 951 - 966
[7] Delivery of CRISPR/Cas9 for therapeutic genome editing
Xu, Xiaojie
Wan, Tao
Xin, Huhu
Li, Da
Pan, Hongming
Wu, Jun
Ping, Yuan
JOURNAL OF GENE MEDICINE, 2019, 21 (07):
[8] Robust in vivo gene editing in mouse hepatocytes with systemic lipid nanoparticle delivery of CRISPR/Cas9 components
Finn, J. D.
Patel, M.
Smith, A. Rhoden
Shaw, L.
Youniss, M.
Ciullo, C.
van Heteren, J.
Lescarbeau, R.
Seitzer, J.
Growe, J.
Dombrowski, C.
Strapps, W. R.
Barnes, T. M.
Morrissey, D. V.
HUMAN GENE THERAPY, 2016, 27 (11) : A131 - A131
[9] Application progress of CRISPR/Cas9 genome-editing technology in edible fungi
Zhang, Yan
Chen, Shutong
Yang, Long
Zhang, Qiang
FRONTIERS IN MICROBIOLOGY, 2023, 14
[10] Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine
Khoshandam, Mohadeseh
Soltaninejad, Hossein
Mousazadeh, Marziyeh
Hamidieh, Amir Ali
Hosseinkhani, Saman
GENES & DISEASES, 2024, 11 (01) : 268 - 282

← 1 2 3 4 5 →