Liposome-Based Carriers for CRISPR Genome Editing

被引:9
作者
Yin, Xing [1 ]
Harmancey, Romain [1 ]
McPherson, David D. [1 ]
Kim, Hyunggun [2 ]
Huang, Shao-Ling [1 ]
机构
[1] Univ Texas Hlth Sci Ctr Houston, Dept Internal Med, Div Cardiovasc Med, Houston, TX 77030 USA
[2] Sungkyunkwan Univ, Dept Biomechatron Engn, Suwon 16419, South Korea
基金
美国国家卫生研究院;
关键词
liposome; CRISPR/Cas9; single-guide RNA; gRNA; gene editing; gene delivery; TARGETED DELIVERY; GENE-TRANSFER; DRUG; CANCER; TRANSFECTION; EFFICIENT; PROGRESS; AGENTS;
D O I
10.3390/ijms241612844
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
The CRISPR-based genome editing technology, known as clustered regularly interspaced short palindromic repeats (CRISPR), has sparked renewed interest in gene therapy. This interest is accompanied by the development of single-guide RNAs (sgRNAs), which enable the introduction of desired genetic modifications at the targeted site when used alongside the CRISPR components. However, the efficient delivery of CRISPR/Cas remains a challenge. Successful gene editing relies on the development of a delivery strategy that can effectively deliver the CRISPR cargo to the target site. To overcome this obstacle, researchers have extensively explored non-viral, viral, and physical methods for targeted delivery of CRISPR/Cas9 and a guide RNA (gRNA) into cells and tissues. Among those methods, liposomes offer a promising approach to enhance the delivery of CRISPR/Cas and gRNA. Liposomes facilitate endosomal escape and leverage various stimuli such as light, pH, ultrasound, and environmental cues to provide both spatial and temporal control of cargo release. Thus, the combination of the CRISPR-based system with liposome delivery technology enables precise and efficient genetic modifications in cells and tissues. This approach has numerous applications in basic research, biotechnology, and therapeutic interventions. For instance, it can be employed to correct genetic mutations associated with inherited diseases and other disorders or to modify immune cells to enhance their disease-fighting capabilities. In summary, liposome-based CRISPR genome editing provides a valuable tool for achieving precise and efficient genetic modifications. This review discusses future directions and opportunities to further advance this rapidly evolving field.
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页数:14
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共 76 条
[41]   Lentiviral gene therapy for X-linked chronic granulomatous disease [J].
Kohn, Donald B. ;
Booth, Claire ;
Kang, Elizabeth M. ;
Pai, Sung-Yun ;
Shaw, Kit L. ;
Santilli, Giorgia ;
Armant, Myriam ;
Buckland, Karen F. ;
Choi, Uimook ;
De Ravin, Suk See ;
Dorsey, Morna J. ;
Kuo, Caroline Y. ;
Leon-Rico, Diego ;
Rivat, Christine ;
Izotova, Natalia ;
Gilmour, Kimberly ;
Snell, Katie ;
Dip, Jinhua Xu-Bayford ;
Darwish, Jinan ;
Morris, Emma C. ;
Terrazas, Dayna ;
Wang, Leo D. ;
Bauser, Christopher A. ;
Paprotka, Tobias ;
Kuhns, Douglas B. ;
Gregg, John ;
Raymond, Hayley E. ;
Everett, John K. ;
Honnet, Geraldine ;
Biasco, Luca ;
Newburger, Peter E. ;
Bushman, Frederic D. ;
Grez, Manuel ;
Gaspar, H. Bobby ;
Williams, David A. ;
Malech, Harry L. ;
Galy, Anne ;
Thrasher, Adrian J. ;
Buckland, Karen F. ;
Bauser, Christopher A. ;
Honnet, Geraldine ;
Grez, Manuel ;
Gaspar, H. Bobby ;
Galy, Anne ;
Thrasher, Adrian J. .
NATURE MEDICINE, 2020, 26 (02) :200-+
[42]   Phase diagram, stability, and overcharging of lamellar cationic lipid-DNA self-assembled complexes [J].
Koltover, I ;
Salditt, T ;
Safinya, CR .
BIOPHYSICAL JOURNAL, 1999, 77 (02) :915-924
[43]   GLABRA2-based selection efficiently enriches Cas9-generated nonchimeric mutants in the T1 generation [J].
Kong, Xiangjiu ;
Pan, Wenbo ;
Sun, Nengxu ;
Zhang, Tingyu ;
Liu, Lijing ;
Zhang, Huawei .
PLANT PHYSIOLOGY, 2021, 187 (02) :758-768
[44]   Liposome composition in drug delivery design, synthesis, characterization, and clinical application [J].
Large, Danielle E. ;
Abdelmessih, Rudolf G. ;
Fink, Elizabeth A. ;
Auguste, Debra T. .
ADVANCED DRUG DELIVERY REVIEWS, 2021, 176
[45]   Tumor cell-targeted delivery of CRISPR/Cas9 by aptamer-functionalized lipopolymer for therapeutic genome editing of VEGFA in osteosarcoma [J].
Liang, Chao ;
Li, Fangfei ;
Wang, Luyao ;
Zhang, Zong-Kang ;
Wang, Chao ;
He, Bing ;
Li, Jie ;
Chen, Zhihao ;
Shaikh, Atik Badshah ;
Liu, Jin ;
Wu, Xiaohao ;
Peng, Songlin ;
Dang, Lei ;
Guo, Baosheng ;
He, Xiaojuan ;
Au, D. W. T. ;
Lu, Cheng ;
Zhu, Hailong ;
Zhang, Bao-Ting ;
Lu, Aiping ;
Zhang, Ge .
BIOMATERIALS, 2017, 147 :68-85
[46]   CATIONIC LIPOSOME-MEDIATED RNA TRANSFECTION [J].
MALONE, RW ;
FELGNER, PL ;
VERMA, IM .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1989, 86 (16) :6077-6081
[47]   PEGylation of Proteins and Liposomes: a Powerful and Flexible Strategy to Improve the Drug Delivery [J].
Milla, Paola ;
Dosio, Franco ;
Cattel, Luigi .
CURRENT DRUG METABOLISM, 2012, 13 (01) :105-119
[48]   Progress in Cationic Lipid-Mediated Gene Transfection: A Series of Bio-Inspired Lipids as an Example [J].
Montier, Tristan ;
Benvegnu, Thierry ;
Jaffres, Paul-Alain ;
Yaouanc, Jean-Jacques ;
Lehn, Pierre .
CURRENT GENE THERAPY, 2008, 8 (05) :296-312
[49]   Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine [J].
Polack, Fernando P. ;
Thomas, Stephen J. ;
Kitchin, Nicholas ;
Absalon, Judith ;
Gurtman, Alejandra ;
Lockhart, Stephen ;
Perez, John L. ;
Perez Marc, Gonzalo ;
Moreira, Edson D. ;
Zerbini, Cristiano ;
Bailey, Ruth ;
Swanson, Kena A. ;
Roychoudhury, Satrajit ;
Koury, Kenneth ;
Li, Ping ;
Kalina, Warren V. ;
Cooper, David ;
Frenck, Robert W., Jr. ;
Hammitt, Laura L. ;
Tureci, Ozlem ;
Nell, Haylene ;
Schaefer, Axel ;
Unal, Serhat ;
Tresnan, Dina B. ;
Mather, Susan ;
Dormitzer, Philip R. ;
Sahin, Ugur ;
Jansen, Kathrin U. ;
Gruber, William C. .
NEW ENGLAND JOURNAL OF MEDICINE, 2020, 383 (27) :2603-2615
[50]   Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3 [J].
Qiu, Min ;
Glass, Zachary ;
Chen, Jinjin ;
Haas, Mary ;
Jin, Xin ;
Zhao, Xuewei ;
Rui, Xuehui ;
Ye, Zhongfeng ;
Li, Yamin ;
Zhang, Feng ;
Xu, Qiaobing .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2021, 118 (10)