Biomaterials as vectors for the delivery of CRISPR–Cas9

Eoh, Joon; Gu, Luo

doi:10.1039/c8bm01310a

Cited by 81 publications

(63 citation statements)

References 135 publications

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“…Gene therapy is one of the most promising approaches for the treatment of serious human diseases, the choice of gene vectors, and therapeutic strategies are of primary concern. Supramolecular gene vectors are widely studied in the realm of gene therapy because of their special responsiveness . In this work, the key issue of our research is to artificially control the redox‐response process of supramolecular assemblies to achieve efficient gene delivery.…”

Section: Introductionmentioning

confidence: 99%

“…Supramolecular gene vectors are widely studied in the realm of gene therapy because of their special responsiveness. [16,[25][26][27] In this work, the key issue of our research is to artificially control the redox-response process of supramolecular assemblies to achieve efficient gene delivery. We hypothesized that the production of singlet oxygen ( 1 O 2 ) from a photosensitizer upon light illumination could accelerate the disassembly of the supramolecular-based delivery system and facilitate the release of pDNA.…”

Section: Introductionmentioning

confidence: 99%

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Oxidation‐Responsive Nanoassemblies for Light‐Enhanced Gene Therapy

Zhang

Wang

et al. 2019

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Microenvironment‐responsive supramolecular assemblies have attracted great interest in the biomedical field due to their potential applications in controlled drug release. In this study, oxidation‐responsive supramolecular polycationic assemblies named CPAs are prepared for nucleic acid delivery via the host–guest interaction of β‐cyclodextrin based polycations and a ferrocene‐functionalized zinc tetraaminophthalocyanine core. The reactive oxygen species (ROS) can accelerate the disassembly of CPA/pDNA complexes, which would facilitate the release of pDNA in the complexes and further benefit the subsequent transfection. Such improvement in transfection efficiency is proved in A549 cells with high H2O2 concentration. Interestingly, the transfection efficiencies mediated by CPAs are also different in the presence or absence of light in various cell lines such as HEK 293 and 4T1. The single oxygen (1O2), produced by photosensitizers in the core of CPAs under light, increases the ROS amount and accelerates the disassembly of CPAs/pDNA complexes. In vitro and in vivo studies further illustrate that suppressor tumor gene p53 delivered by CPAs exhibits great antitumor effects under illumination. This work provides a promising strategy for the design and fabrication of oxidation‐responsive nanoassemblies with light‐enhanced gene transfection performance.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Oxidation‐Responsive Nanoassemblies for Light‐Enhanced Gene Therapy

Zhang

Wang

et al. 2019

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“…Non‐viral vectors with a better safety profile usually do not possess sufficient transfection efficiency. [ 7–12 ] The reported non‐viral vectors cover diverse materials such as lipid‐like materials, [ 13,14 ] cell exosomes, [ 15 ] polymers and their hybrids, [ 16–22 ] nucleic acid based materials, [ 23 ] and metal containing materials. [ 24–26 ] Nevertheless, in spite of extensive studies on functionalization of non‐viral vectors, the studies on the vectors for delivery of genome editing plasmids are relatively rare.…”

Section: Methodsmentioning

confidence: 99%

Aptamer/Peptide‐Functionalized Genome‐Editing System for Effective Immune Restoration through Reversal of PD‐L1‐Mediated Cancer Immunosuppression

et al. 2020

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Effective reversal of tumor immunosuppression is of critical importance in cancer therapy. A multifunctional delivery vector that can effectively deliver CRISPR‐Cas9 plasmid for β‐catenin knockout to reverse tumor immunosuppression is constructed. The multi‐functionalized delivery vector is decorated with aptamer‐conjugated hyaluronic acid and peptide‐conjugated hyaluronic acid to combine the tumor cell/nuclear targeting function of AS1411 with the cell penetrating/nuclear translocation function of TAT‐NLS. Due to the significantly enhanced plasmid enrichment in malignant cell nuclei, the genome editing system can induce effective β‐catenin knockout and suppress Wnt/β‐catenin pathway, resulting in notably downregulated proteins involved in tumor progression and immunosuppression. Programmed death‐ligand 1 (PD‐L1) downregulation in edited tumor cells not only releases the PD‐1/PD‐L1 brake to improve the cancer killing capability of CD8+ T cells, but also enhances antitumor immune responses of immune cells. This provides a facile strategy to reverse tumor immunosuppression and to restore immunosurveillance and activate anti‐tumor immunity.

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“…In circulation, innate immune responses can occur through the activation of different kind of toll-like receptors (TLRs) by impurities such as endotoxins or by bacterial components such as CpG motifs present on the genetic material. Furthermore, an adaptive immune response can also be activated in the case of pre-existing immunity [28]. In the case of genetic material delivered by non-viral vectors, some physicochemical parameters such as zeta potential, particle size destruction, polydispersity index, or hydrophobicity/hydrophilicity balance can also contribute to the compatibility with the immune system [29].…”

Section: Extracellular Barriersmentioning

confidence: 99%

Niosome-Based Approach for In Situ Gene Delivery to Retina and Brain Cortex as Immune-Privileged Tissues

et al. 2020

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Non-viral vectors have emerged as a promising alternative to viral gene delivery systems due to their safer profile. Among non-viral vectors, recently, niosomes have shown favorable properties for gene delivery, including low toxicity, high stability, and easy production. The three main components of niosome formulations include a cationic lipid that is responsible for the electrostatic interactions with the negatively charged genetic material, a non-ionic surfactant that enhances the long-term stability of the niosome, and a helper component that can be added to improve its physicochemical properties and biological performance. This review is aimed at providing recent information about niosome-based non-viral vectors for gene delivery purposes. Specially, we will discuss the composition, preparation methods, physicochemical properties, and biological evaluation of niosomes and corresponding nioplexes that result from the addition of the genetic material onto their cationic surface. Next, we will focus on the in situ application of such niosomes to deliver the genetic material into immune-privileged tissues such as the brain cortex and the retina. Finally, as future perspectives, non-invasive administration routes and different targeting strategies will be discussed.

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Biomaterials as vectors for the delivery of CRISPR–Cas9

Abstract: The emergence of the CRISPR–Cas9 gene editing system has generated considerable hope and excitement in the field of gene therapy and the larger scientific community. Recently, biomaterials have become an attractive option for the delivery of Cas9 due to their remarkable versatility.

Cited by 81 publications

References 135 publications

Oxidation‐Responsive Nanoassemblies for Light‐Enhanced Gene Therapy

Oxidation‐Responsive Nanoassemblies for Light‐Enhanced Gene Therapy

Aptamer/Peptide‐Functionalized Genome‐Editing System for Effective Immune Restoration through Reversal of PD‐L1‐Mediated Cancer Immunosuppression

Niosome-Based Approach for In Situ Gene Delivery to Retina and Brain Cortex as Immune-Privileged Tissues

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