Virus-like nanoparticle as a co-delivery system to enhance efficacy of CRISPR/Cas9-based cancer immunotherapy

Liu, Qi; Chün, Wang; Zheng, Yadan; Zhao, Yu; Wang, Ying; Hao, Jialei; Zhao, Xinzhi; Yi, Kaikai; Shi, Linqi; Kang, Chunsheng; Yang, Liu

doi:10.1016/j.biomaterials.2020.120275

Cited by 102 publications

(88 citation statements)

References 51 publications

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“…[ 16 ] PD‐L1 is identified as a prominent negative immunoregulatory molecule that can protect tumor cells from T‐cell‐mediated immune surveillance by inhibiting the proliferation and effector functions of T lymphocytes after specific binding to its receptor, PD‐1 on T cells. [ 17 ] As a powerful genome‐editing tool, CRISPR/Cas9 has shown its great potential to precisely and effectively disrupt PD‐L1 pathway in recent studies, [ 13,18 ] which could potentially bring a new modality for anti‐PD‐L1 therapy in cancer treatment. To this end, the specific sgRNA against PD‐L1 was designed and further optimized to achieve efficient genome editing of PD‐L1 genomic locus (Figure S13, Supporting Information).…”

Section: Methodsmentioning

confidence: 99%

Reprogramming the Tumor Microenvironment through Second‐Near‐Infrared‐Window Photothermal Genome Editing of PD‐L1 Mediated by Supramolecular Gold Nanorods for Enhanced Cancer Immunotherapy

et al. 2021

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Section: Methodsmentioning

confidence: 99%

Reprogramming the Tumor Microenvironment through Second‐Near‐Infrared‐Window Photothermal Genome Editing of PD‐L1 Mediated by Supramolecular Gold Nanorods for Enhanced Cancer Immunotherapy

et al. 2021

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“…In vivo studies demonstrated the high efficiency of NC-mediated gene editing upon local injection in the retina or muscles (Chen et al, 2019). Liu et al (2020) used bioreducible lipid NPs (LNPs) to deliver Cas9 mRNA and sgRNAs. This represents one of the most efficacious tools for non-viral CRISPR/Cas9 gene editing with an in vitro target efficiency of up to ∼90% in green fluorescent protein (GFP)-expressing cells, and an in vivo editing efficiency of ∼80% for the Pck1 gene, a therapeutic target for cardiovascular disease.…”

Section: Non-viral Methods For Gene Editing Applicationsmentioning

confidence: 99%

Delivery Platforms for CRISPR/Cas9 Genome Editing of Glial Cells in the Central Nervous System

et al. 2021

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Glial cells (astrocytes, oligodendrocytes, and microglia) are emerging as key players in several physiological and pathological processes of the central nervous system (CNS). Astrocytes and oligodendrocytes are not only supportive cells that release trophic factors or regulate energy metabolism, but they also actively modulate critical neuronal processes and functions in the tripartite synapse. Microglia are defined as CNS-resident cells that provide immune surveillance; however, they also actively contribute to shaping the neuronal microenvironment by scavenging cell debris or regulating synaptogenesis and pruning. Given the many interconnected processes coordinated by glial cells, it is not surprising that both acute and chronic CNS insults not only cause neuronal damage but also trigger complex multifaceted responses, including neuroinflammation, which can critically contribute to the disease progression and worsening of symptoms in several neurodegenerative diseases. Overall, this makes glial cells excellent candidates for targeted therapies to treat CNS disorders. In recent years, the application of gene editing technologies has redefined therapeutic strategies to treat genetic and age-related neurological diseases. In this review, we discuss the advantages and limitations of clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9-based gene editing in the treatment of neurodegenerative disorders, focusing on the development of viral- and nanoparticle-based delivery methods for in vivo glial cell targeting.

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“…When utilized for CRISPR delivery in preclinical studies, these nanoparticles are commonly mesoporous and formulated with tetraethyl orthosilicate (TEOS) as well as conjugated with some form of lipid or organic compound. Multiple preclinical applications demonstrated the potential of silica nanoparticles for in vivo delivery of CRISPR-Cas9 to treat murine models of hypercholesterolemia 120 , hepatocellular carcinoma 121 , pulmonary adenocarcinoma 122 , and melanoma 123 as well as proof-of-concept editing in retinal pigment epithelium 124 and brain neurons 125 .…”

Section: Current Vectors For In Vivo Delivery Of Crispr-cas9 Therapeuticsmentioning

confidence: 99%

In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges

Behr

Zhou²,

et al. 2021

Acta Pharmaceutica Sinica B

124

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Within less than a decade since its inception, CRISPR-Cas9-based genome editing has been rapidly advanced to human clinical trials in multiple disease areas. Although it is highly anticipated that this revolutionary technology will bring novel therapeutic modalities to many diseases by precisely manipulating cellular DNA sequences, the low efficiency of in vivo delivery must be enhanced before its therapeutic potential can be fully realized. Here we discuss the most recent progress of in vivo delivery of CRISPR-Cas9 systems, highlight innovative viral and non-viral delivery technologies, emphasize outstanding delivery challenges, and provide the most updated perspectives.

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Virus-like nanoparticle as a co-delivery system to enhance efficacy of CRISPR/Cas9-based cancer immunotherapy

Cited by 102 publications

References 51 publications

Reprogramming the Tumor Microenvironment through Second‐Near‐Infrared‐Window Photothermal Genome Editing of PD‐L1 Mediated by Supramolecular Gold Nanorods for Enhanced Cancer Immunotherapy

Reprogramming the Tumor Microenvironment through Second‐Near‐Infrared‐Window Photothermal Genome Editing of PD‐L1 Mediated by Supramolecular Gold Nanorods for Enhanced Cancer Immunotherapy

Delivery Platforms for CRISPR/Cas9 Genome Editing of Glial Cells in the Central Nervous System

In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges

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