Abstract：
:Genome-editing tools such as Cre recombinase (Cre), zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and most recently the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein system have revolutionized biomedical research, agriculture, microbial engineering, and therapeutic development. Direct delivery of genome editing enzymes, as opposed to their corresponding DNA and mRNA precursors, is advantageous since they do not require transcription and/or translation. In addition, prolonged overexpression is a problem when delivering viral vector or plasmid DNA which is bypassed when delivering whole proteins. This lowers the risk of insertional mutagenesis and makes for relatively easier manufacturing. However, a major limitation of utilizing genome editing proteins in vivo is their low delivery efficiency, and currently the most successful strategy involves using potentially immunogenic viral vectors. This lack of safe and effective non-viral delivery systems is still a big hurdle for the clinical translation of such enzymes. This review discusses the challenges of non-viral delivery strategies of widely used genome editing enzymes, including Cre recombinase, ZFNs and TALENs, CRISPR/Cas9, and Cas12a (Cpf1) in their protein format and highlights recent innovations of non-viral delivery strategies which have the potential to overcome current delivery limitations and advance the clinical translation of genome editing.
journal_name
Adv Drug Deliv Revjournal_title
Advanced drug delivery reviewsauthors
Li J,Røise JJ,He M,Das R,Murthy Ndoi
10.1016/j.addr.2020.09.004subject
Has Abstractpub_date
2021-01-01 00:00:00pages
99-117eissn
0169-409Xissn
1872-8294pii
S0169-409X(20)30128-9journal_volume
168pub_type
杂志文章,评审abstract：:Cells cultured in vitro will tend to retain their differentiated phenotype under conditions that resemble their natural in vivo environment, for example, when cultured on polymer scaffolds in tissue culture bioreactors. In this chapter, we define organized cell communities as three-dimensional in vitro grown cell-poly...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/s0169-409x(98)00017-9
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journal_title：Advanced drug delivery reviews
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journal_title：Advanced drug delivery reviews
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
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journal_title：Advanced drug delivery reviews
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journal_title：Advanced drug delivery reviews
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2018.06.019
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2017.07.016
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2020.04.001
更新日期：2021-01-01 00:00:00
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/s0169-409x(01)00123-5
更新日期：2001-07-02 00:00:00
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2006.08.002
更新日期：2006-11-30 00:00:00
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2004.07.005
更新日期：2004-10-14 00:00:00
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2015.11.002
更新日期：2016-02-01 00:00:00
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2012.05.015
更新日期：2013-04-01 00:00:00
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/s0169-409x(01)00170-3
更新日期：2001-09-23 00:00:00
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2006.09.011
更新日期：2006-12-01 00:00:00
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2018.10.010
更新日期：2019-02-01 00:00:00
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2019.08.004
更新日期：2019-04-01 00:00:00
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/s0169-409x(97)00105-1
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2017.07.012
更新日期：2017-10-01 00:00:00
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2017.10.013
更新日期：2017-11-01 00:00:00
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2012.07.005
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2007.03.017
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/s0169-409x(97)00056-2
更新日期：1997-10-13 00:00:00
abstract：:Theranostics, the fusion of therapy and diagnostics for optimizing efficacy and safety of therapeutic regimes, is a growing field that is paving the way towards the goal of personalized medicine for the benefit of patients. The use of light as a remote-activation mechanism for drug delivery has received increased atte...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2010.09.002
更新日期：2010-08-30 00:00:00
abstract：:Using oligonucleotide-based drugs to modulate gene expression has opened a new avenue for drug discovery. In particular small interfering RNAs (siRNAs) are being rapidly recognized as promising therapeutic tools, but their poor bioavailability limits the full realization of their clinical potential. In recent years, c...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2012.08.008
更新日期：2013-03-01 00:00:00
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journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2015.09.004
更新日期：2015-12-01 00:00:00
abstract：:Vaccines are one of the most powerful technologies supporting public health. The adaptive immune response induced by immunization arises following appropriate activation and differentiation of T and B cells in lymph nodes. Among many parameters impacting the resulting immune response, the presence of antigen and infla...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2020.06.019
更新日期：2020-01-01 00:00:00
