Viral and nonviral nanocarriers for in vivo CRISPR-based gene editing

被引:1
作者
Guo, Zhongyuan [1 ]
Zhu, Audrey T. [1 ]
Fang, Ronnie H. [1 ,2 ]
Zhang, Liangfang [1 ]
机构
[1] Univ Calif San Diego, Aiiso Yufeng Li Family Dept Chem & Nano Engn, La Jolla, CA 92093 USA
[2] Univ Calif San Diego, Dept Pediat, Div Host Microbe Syst & Therapeut, La Jolla, CA 92093 USA
基金
美国国家卫生研究院;
关键词
CRISPR; in vivo gene editing; nanocarriers; recombinant adeno-associated viruses (rAAVs); virus-like particle (VLP); lipid nanoparticle (LNP); ADENOASSOCIATED VIRUS VECTOR; MESSENGER-RNA DELIVERY; LIPID NANOPARTICLES; GUIDE-RNA; ADENOVIRUS VECTORS; CATIONIC LIPIDS; CHEMICAL-MODIFICATIONS; GENERATION ADENOVIRUS; LENTIVIRAL VECTOR; SKELETAL-MUSCLE;
D O I
10.1007/s12274-024-6748-5
中图分类号
O64 [物理化学(理论化学)、化学物理学];
学科分类号
070304 ; 081704 ;
摘要
The continued development of clustered regularly interspaced short palindromic repeats (CRISPR) technology has the potential to greatly impact clinical medicine, particularly for disease diagnosis and treatment. Despite high demand for the in vivo delivery of CRISPR-based therapies, significant challenges persist. These include rapid degradation by enzymes, inefficient disease site targeting, and the risk of undesired off-target outcomes. Nanoparticulate platforms, with their tailorable properties, have been engineered to efficiently package CRISPR payloads in various formats, including as plasmid DNA, mRNA, and ribonucleoprotein complexes, for in vivo delivery. Among them, recombinant adeno-associated viruses, virus-like particles, and lipid nanoparticles have displayed exceptional promise. This review will discuss the development of these and other nanocarriers for in vivo CRISPR-based genome editing.
引用
收藏
页码:8904 / 8925
页数:22
相关论文
共 296 条
  • [1] RNA targeting with CRISPR-Cas13
    Abudayyeh, Omar O.
    Gootenberg, Jonathan S.
    Essletzbichler, Patrick
    Han, Shuo
    Joung, Julia
    Belanto, Joseph J.
    Verdine, Vanessa
    Cox, David B. T.
    Kellner, Max J.
    Regev, Aviv
    Lander, Eric S.
    Voytas, Daniel F.
    Ting, Alice Y.
    Zhang, Feng
    [J]. NATURE, 2017, 550 (7675) : 280 - +
  • [2] Optimized nickase- and nuclease-based prime editing in human and mouse cells
    Adikusuma, Fatwa
    Lushington, Caleb
    Arudkumar, Jayshen
    Godahewa, Gelshan, I
    Chey, Yu C. J.
    Gierus, Luke
    Piltz, Sandra
    Geiger, Ashleigh
    Jain, Yatish
    Reti, Daniel
    Wilson, Laurence O. W.
    Bauer, Denis C.
    Thomas, Paul Q.
    [J]. NUCLEIC ACIDS RESEARCH, 2021, 49 (18) : 10785 - 10795
  • [3] The CRISPR tool kit for genome editing and beyond
    Adli, Mazhar
    [J]. NATURE COMMUNICATIONS, 2018, 9
  • [4] Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms
    Akinc, Akin
    Querbes, William
    De, Soma
    Qin, June
    Frank-Kamenetsky, Maria
    Jayaprakash, K. Narayanannair
    Jayaraman, Muthusamy
    Rajeev, Kallanthottathil G.
    Cantley, William L.
    Dorkin, J. Robert
    Butler, James S.
    Qin, LiuLiang
    Racie, Timothy
    Sprague, Andrew
    Fava, Eugenio
    Zeigerer, Anja
    Hope, Michael J.
    Zerial, Marino
    Sah, Dinah W. Y.
    Fitzgerald, Kevin
    Tracy, Mark A.
    Manoharan, Muthiah
    Koteliansky, Victor
    de Fougerolles, Antonin
    Maier, Martin A.
    [J]. MOLECULAR THERAPY, 2010, 18 (07) : 1357 - 1364
  • [5] Gutless adenovirus: last-generation adenovirus for gene therapy
    Alba, R
    Bosch, A
    Chillon, M
    [J]. GENE THERAPY, 2005, 12 (Suppl 1) : S18 - S27
  • [6] The role of lipid components in lipid nanoparticles for vaccines and gene therapy
    Albertsen, Camilla Hald
    Kulkarni, Jayesh A.
    Witzigmann, Dominik
    Lind, Marianne
    Petersson, Karsten
    Simonsen, Jens B.
    [J]. ADVANCED DRUG DELIVERY REVIEWS, 2022, 188
  • [7] Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo
    An, Meirui
    Raguram, Aditya
    Du, Samuel W.
    Banskota, Samagya
    Davis, Jessie R.
    Newby, Gregory A.
    Chen, Paul Z.
    Palczewski, Krzysztof
    Liu, David R.
    [J]. NATURE BIOTECHNOLOGY, 2024, 42 (10) : 1526 - +
  • [8] Search-and-replace genome editing without double-strand breaks or donor DNA
    Anzalone, Andrew V.
    Randolph, Peyton B.
    Davis, Jessie R.
    Sousa, Alexander A.
    Koblan, Luke W.
    Levy, Jonathan M.
    Chen, Peter J.
    Wilson, Christopher
    Newby, Gregory A.
    Raguram, Aditya
    Liu, David R.
    [J]. NATURE, 2019, 576 (7785) : 149 - +
  • [9] Nature's recipe for splitting inteins
    Aranko, A. Sesilja
    Wlodawer, Alexander
    Iwai, Hideo
    [J]. PROTEIN ENGINEERING DESIGN & SELECTION, 2014, 27 (08) : 263 - 271
  • [10] Designed arginine-rich RNA-binding peptides with picomolar affinity
    Austin, RJ
    Xia, TB
    Ren, JS
    Takahashi, TT
    Roberts, RW
    [J]. JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, 2002, 124 (37) : 10966 - 10967