2020
DOI: 10.3390/pharmaceutics12121233
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Nanovesicle-Mediated Delivery Systems for CRISPR/Cas Genome Editing

Abstract: Genome-editing technology has emerged as a potential tool for treating incurable diseases for which few therapeutic modalities are available. In particular, discovery of the clustered regularly interspaced short palindromic repeats (CRISPR)/Cas system together with the design of single-guide RNAs (sgRNAs) has sparked medical applications of genome editing. Despite the great promise of the CRISPR/Cas system, its clinical application is limited, in large part, by the lack of adequate delivery technology. To over… Show more

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Cited by 26 publications
(21 citation statements)
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References 183 publications
(217 reference statements)
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“…This demonstrates the possibility of using tube particles to transport specialized proteins to nanoparticles that could influence exosome development. In addition, a previous study reported direct engineering of a fluorescent substance into exosomes to confirm trafficking in human cells (Figure 1d(i)) [48,49].…”
Section: Strategies Of Exosome Applications 41 Development Of Exosome Trafficking Systemmentioning
confidence: 93%
“…This demonstrates the possibility of using tube particles to transport specialized proteins to nanoparticles that could influence exosome development. In addition, a previous study reported direct engineering of a fluorescent substance into exosomes to confirm trafficking in human cells (Figure 1d(i)) [48,49].…”
Section: Strategies Of Exosome Applications 41 Development Of Exosome Trafficking Systemmentioning
confidence: 93%
“…This was demonstrated by evaluating the cancer-targeting ability of targeted exosomes in tumor transplant animal models and evaluating the anticancer efficacy of romidepsin [110]. CRISPR-Cas9 technology, which is widely used in cell and animal experiments, presents obstacles to its clinical application [113]. The researchers confirmed that the CRISPR-Cas9-sgRNA and Cas9 protein can be packaged into EVs that exist as sgRNA and Cas9 protein complexes of sgRNA: Cas9 ribonucleoprotein [111,112].…”
Section: Ev Engineering To Develop a Targeted Drug Delivery Systemmentioning
confidence: 96%
“…To target destinations, studies reported through engineering modifications of proteins such as CRISPR/CAS9 or aptamers have been introduced [112][113][114]. To verify the efficiency of targeted drug delivery, the histone deacetylase inhibitor lomidepsin was loaded into mAb-exosomes, which showed increased anticancer toxicity in vitro.…”
Section: Ev Engineering To Develop a Targeted Drug Delivery Systemmentioning
confidence: 99%
“…Thus, it is essential to improve various aspects of nanovesicles [ 148 ]. For such nanomaterials, techniques and gene delivery approaches continue to be developed, and the key challenge is to balance transfection efficiency, targeting specificity, particle size, biodegradability, and cytotoxicity, as well as their short- and long-term fates in the environment [ 149 ].…”
Section: Future Prospectsmentioning
confidence: 99%