Synthetic nanoparticles for the delivery of CRISPR/Cas9 gene editing system: classification and biomedical applications

Zheng, Qi; Wang, Weitao; Zhou, Yuhang; Mo, Jiayin; Chang, Xinyue; Zha, Zhengbao; Zha, Lisha

doi:10.1039/d3bm00788j

Cited by 3 publications

(3 citation statements)

References 167 publications

(266 reference statements)

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“…Several unique NPs have been reported for CRISPR-Cas delivery, including polymeric, lipid, gold, and silicon nanoparticles. 75,86,89–95 These NPs exhibit chemical design flexibility, high loading capacity, high safety and stability, biocompatibility, and low immunogenicity. 71,94,96,97 In addition, NPs can be easily modified to target specific cells, making genome editing possible in a broad spectrum of cells in the complicated tumor microenvironment (TME) and immune systems.…”

Section: Introductionmentioning

confidence: 99%

Non-viral vector-based genome editing for cancer immunotherapy

Fang,

Chen

2024

Biomater. Sci.

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Section: Introductionmentioning

confidence: 99%

Non-viral vector-based genome editing for cancer immunotherapy

Fang,

Chen

2024

Biomater. Sci.

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“…21−23 Despite the exciting possibilities offered by CRISPR-Cas systems, the delivery of a solitary CRISPR system often appears insufficient to realize its promising therapeutic potential within the intricate internal environment of an organism. 24 Furthermore, the current limitations in temporal and spatial precision during the editing process significantly restrict the application of complex and diverse genome editing scenarios with CRISPR-Cas9 systems. Therefore, there has been significant interest in stimulation-responsive CRISPR-Cas systems, which can effectively transport gene editing systems to the target site and respond to specific stimuli, thereby maximizing the efficiency of gene therapy.…”

mentioning

confidence: 99%

“…The CRISPR-Cas9-based genome editing technology renders a robust and reliable approach for precisely perturbing almost any genomic sequence in living cells. − Despite the exciting possibilities offered by CRISPR-Cas systems, the delivery of a solitary CRISPR system often appears insufficient to realize its promising therapeutic potential within the intricate internal environment of an organism . Furthermore, the current limitations in temporal and spatial precision during the editing process significantly restrict the application of complex and diverse genome editing scenarios with CRISPR-Cas9 systems.…”

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confidence: 99%

A Nanozyme-Boosted MOF-CRISPR Platform for Treatment of Alzheimer’s Disease

Yang,

Qin,

Liu

et al. 2024

Nano Lett.

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Rectifying the aberrant microenvironment of a disease through maintenance of redox homeostasis has emerged as a promising perspective with significant therapeutic potential for Alzheimer's disease (AD). Herein, we design and construct a novel nanozyme-boosted MOF-CRISPR platform (CMOPKP), which can maintain redox homeostasis and rescue the impaired microenvironment of AD. By modifying the targeted peptides KLVFFAED, CMOPKP can traverse the blood−brain barrier and deliver the CRISPR activation system for precise activation of the Nrf2 signaling pathway and downstream redox proteins in regions characterized by oxidative stress, thereby reinstating neuronal antioxidant capacity and preserving redox homeostasis. Furthermore, cerium dioxide possessing catalase enzyme-like activity can synergistically alleviate oxidative stress. Further in vivo studies demonstrate that CMOPKP can effectively alleviate cognitive impairment in 3xTg-AD mouse models. Therefore, our design presents an effective way for regulating redox homeostasis in AD, which shows promise as a therapeutic strategy for mitigating oxidative stress in AD.

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Harnessing the evolving CRISPR/Cas9 for precision oncology

Li,

Kang

et al. 2024

J Transl Med

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The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)/Cas9 system, a groundbreaking innovation in genetic engineering, has revolutionized our approach to surmounting complex diseases, culminating in CASGEVY ™ approved for sickle cell anemia. Derived from a microbial immune defense mechanism, CRISPR/Cas9, characterized as precision, maneuverability and universality in gene editing, has been harnessed as a versatile tool for precisely manipulating DNA in mammals. In the process of applying it to practice, the consecutive exploitation of novel orthologs and variants never ceases. It's conducive to understanding the essentialities of diseases, particularly cancer, which is crucial for diagnosis, prevention, and treatment. CRISPR/Cas9 is used not only to investigate tumorous genes functioning but also to model disparate cancers, providing valuable insights into tumor biology, resistance, and immune evasion. Upon cancer therapy, CRISPR/Cas9 is instrumental in developing individual and precise cancer therapies that can selectively activate or deactivate genes within tumor cells, aiming to cripple tumor growth and invasion and sensitize cancer cells to treatments. Furthermore, it facilitates the development of innovative treatments, enhancing the targeting efficiency of reprogrammed immune cells, exemplified by advancements in CAR-T regimen. Beyond therapy, it is a potent tool for screening susceptible genes, offering the possibility of intervening before the tumor initiative or progresses. However, despite its vast potential, the application of CRISPR/Cas9 in cancer research and therapy is accompanied by significant efficacy, efficiency, technical, and safety considerations. Escalating technology innovations are warranted to address these issues. The CRISPR/Cas9 system is revolutionizing cancer research and treatment, opening up new avenues for advancements in our understanding and management of cancers. The integration of this evolving technology into clinical practice promises a new era of precision oncology, with targeted, personalized, and potentially curative therapies for cancer patients.

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Synthetic nanoparticles for the delivery of CRISPR/Cas9 gene editing system: classification and biomedical applications

Abstract: Gene editing has great potential in biomedical research including disease diagnosis and treatment. Clustered regularly interspaced short palindromic repeats (CRISPR) is the most straightforward and cost-effective method. The efficient and...

Cited by 3 publications

References 167 publications

Non-viral vector-based genome editing for cancer immunotherapy

Non-viral vector-based genome editing for cancer immunotherapy

A Nanozyme-Boosted MOF-CRISPR Platform for Treatment of Alzheimer’s Disease

Harnessing the evolving CRISPR/Cas9 for precision oncology

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