In vivo delivery of CRISPR-Cas9 genome editing components for therapeutic applications

Huang, Kun; Zapata, Daniel; Tang, Yuzhe; Teng, Yong; Li, Yamin

doi:10.1016/j.biomaterials.2022.121876

Cited by 21 publications

(22 citation statements)

References 244 publications

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“…This method is economical as well as convenient [ 12 , 26 ]. However, gRNA and Cas9 protein are further formed by transcription and translation in cells, which increases the working time and the rate of off-target effects [ 27 ]. Meanwhile, this process, which is difficult to control, could have potential risks for DNA sequences integrated into the genome [ 28 ].…”

Section: Delivery Of Crispr/cas9mentioning

confidence: 99%

“…With precise site-specific gene editing, the CRISPR-Cas9 system has gradually become a powerful tool to cure a number of genetic disorders and tumors, especially transthyretin amyloidosis, which has entered phase 1 clinical trials [ 27 ]. As mentioned above, EVs, with good biocompatibility, low immunogenicity, capacity, protection ability, modification and targeting ability, have emerged as favorable tissue-specific vectors for delivering the CRISPR-Cas9 system and other biomacromolecules, which have been discussed.…”

Section: Delivery Of the Crispr/cas9 System By Evsmentioning

confidence: 99%

“…The delivery form of the CRISPR/Cas9 system determines the efficiency of gene editing. Briefly, because they avoid the process of transcription and translation, RNPs make gene editing faster and more efficient and lower the rate of off-target effects than the DNA and RNA forms of the CRISPR/Cas9 system, but are more expensive [ 27 ]. Recent studies have demonstrated that the DNA, RNA and RNP form of the CRISPR/Cas9 system can be carried and delivered by EVs for gene editing in vitro and in vivo.…”

Section: Delivery Of the Crispr/cas9 System By Evsmentioning

confidence: 99%

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The CRISPR/Cas9 System Delivered by Extracellular Vesicles

et al. 2023

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Clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein (Cas) systems can precisely manipulate DNA sequences to change the characteristics of cells and organs, which has potential in the mechanistic research on genes and the treatment of diseases. However, clinical applications are restricted by the lack of safe, targeted and effective delivery vectors. Extracellular vesicles (EVs) are an attractive delivery platform for CRISPR/Cas9. Compared with viral and other vectors, EVs present several advantages, including safety, protection, capacity, penetrating ability, targeting ability and potential for modification. Consequently, EVs are profitably used to deliver the CRISPR/Cas9 in vivo. In this review, the advantages and disadvantages of the delivery form and vectors of the CRISPR/Cas9 are concluded. The favorable traits of EVs as vectors, such as the innate characteristics, physiological and pathological functions, safety and targeting ability of EVs, are summarized. Furthermore, in terms of the delivery of the CRISPR/Cas9 by EVs, EV sources and isolation strategies, the delivery form and loading methods of the CRISPR/Cas9 and applications have been concluded and discussed. Finally, this review provides future directions of EVs as vectors of the CRISPR/Cas9 system in clinical applications, such as the safety, capacity, consistent quality, yield and targeting ability of EVs.

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Section: Delivery Of Crispr/cas9mentioning

confidence: 99%

Section: Delivery Of the Crispr/cas9 System By Evsmentioning

confidence: 99%

Section: Delivery Of the Crispr/cas9 System By Evsmentioning

confidence: 99%

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The CRISPR/Cas9 System Delivered by Extracellular Vesicles

et al. 2023

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“…In 1996, the zinc finger enzyme gene editing technology was invented 5 . Furthermore, the advent of clustered regularly interspaced short palindromic repeats (CRISPR/Cas9) gene editing technology in 2012 revolutionized the field of gene therapy by simplifying the workload while reducing the costs of gene editing 6 . Subsequently, scientists discovered other Cas enzymes with unique properties and have continued to develop novel gene editing methods that can edit multiple loci in the genome simultaneously, such as Cas Hybrid for Multiplexed Editing and screening Applications 7,8 .…”

Section: Introductionmentioning

confidence: 99%

Biomaterial‐based gene therapy

Gao

et al. 2023

MedComm

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Gene therapy, a medical approach that involves the correction or replacement of defective and abnormal genes, plays an essential role in the treatment of complex and refractory diseases, such as hereditary diseases, cancer, and rheumatic immune diseases. Nucleic acids alone do not easily enter the target cells due to their easy degradation in vivo and the structure of the target cell membranes. The introduction of genes into biological cells is often dependent on gene delivery vectors, such as adenoviral vectors, which are commonly used in gene therapy. However, traditional viral vectors have strong immunogenicity while also presenting a potential infection risk. Recently, biomaterials have attracted attention for use as efficient gene delivery vehicles, because they can avoid the drawbacks associated with viral vectors. Biomaterials can improve the biological stability of nucleic acids and the efficiency of intracellular gene delivery. This review is focused on biomaterial‐based delivery systems in gene therapy and disease treatment. Herein, we review the recent developments and modalities of gene therapy. Additionally, we discuss nucleic acid delivery strategies, with a focus on biomaterial‐based gene delivery systems. Furthermore, the current applications of biomaterial‐based gene therapy are summarized.

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“…Therefore, it is required to identify CRISPR/Cas9 delivery vectors with high targeting, low immunogenicity, biocompatibility and efficacy. 12,13 Compared to physical and chemical delivery vectors, biogenic materials are effective delivery vectors with good biocompatibility, targeting and low immunogenicity. 14 Biogenic materials are composed of a complete or a portion of a living structure that accomplishes or enhances or substitutes a natural function.…”

Section: Introductionmentioning

confidence: 99%