Genome editing of PD-L1 mediated by nucleobase-modified polyamidoamine for cancer immunotherapy

Wei, Simeng; Shao, Xinxin; Liu, Yong; Xiong, Boyu; Cui, Pengfei; Liu, Ziling; Li, Quanshun

doi:10.1039/d1tb02688g

Cited by 10 publications

(9 citation statements)

References 46 publications

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“…One of the most commonly used methods to deliver CRISPR to the tumor site is through the use of viral vectors [ 247 ]. Viral vectors are modified versions of viruses that can be used to introduce genes or other genetic material into cells [ 248 ]. The most commonly used viral vectors for CRISPR delivery are adeno-associated viruses (AAVs) and lentiviruses.…”

Section: Safety and Delivery Challengesmentioning

confidence: 99%

Comprehensive review of CRISPR-based gene editing: mechanisms, challenges, and applications in cancer therapy

Chehelgerdi,

Khorramian-Ghahfarokhi

et al. 2024

Mol Cancer

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The CRISPR system is a revolutionary genome editing tool that has the potential to revolutionize the field of cancer research and therapy. The ability to precisely target and edit specific genetic mutations that drive the growth and spread of tumors has opened up new possibilities for the development of more effective and personalized cancer treatments. In this review, we will discuss the different CRISPR-based strategies that have been proposed for cancer therapy, including inactivating genes that drive tumor growth, enhancing the immune response to cancer cells, repairing genetic mutations that cause cancer, and delivering cancer-killing molecules directly to tumor cells. We will also summarize the current state of preclinical studies and clinical trials of CRISPR-based cancer therapy, highlighting the most promising results and the challenges that still need to be overcome. Safety and delivery are also important challenges for CRISPR-based cancer therapy to become a viable clinical option. We will discuss the challenges and limitations that need to be overcome, such as off-target effects, safety, and delivery to the tumor site. Finally, we will provide an overview of the current challenges and opportunities in the field of CRISPR-based cancer therapy and discuss future directions for research and development. The CRISPR system has the potential to change the landscape of cancer research, and this review aims to provide an overview of the current state of the field and the challenges that need to be overcome to realize this potential.

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Section: Safety and Delivery Challengesmentioning

confidence: 99%

Comprehensive review of CRISPR-based gene editing: mechanisms, challenges, and applications in cancer therapy

Chehelgerdi,

Khorramian-Ghahfarokhi

et al. 2024

Mol Cancer

View full text Add to dashboard Cite

show abstract

“…Then immunogenic cell death (ICD) and the specific expression of Cas9 protein to achieve the disruption of PD‐L1 were induced under 660 nm laser irradiation, which reprogrammed the immunosuppressive microenvironment and stimulated robust cancer immunotherapy to inhibit primary, distant, and lung metastatic tumor growth. Moreover, Li and co‐workers developed a nano‐delivery system composed of 2‐amino‐6‐chloropurine‐modified polyamidoamine (PAMAM) dendrimer and CRISPR/Cas9‐based PD‐L1 knockout plasmid (Figure 6f; Wei, Shao, et al, 2022). The nanoplatform kept its stability during the circulation and facilitated endosome escape of plasmid to disrupt the expression of PD‐L1 leading to the activation of anti‐tumor immunity which inhibited the melanoma pulmonary metastases.…”

Section: Delivery Strategies Of Crispr/cas Systems For Cancer Therapymentioning

confidence: 99%

“…(f) Design and action process of modified PAMAM loading CRISPR/Cas9 plasmid targeting PD‐L1, which exhibited efficient gene editing. Reproduced with permission from Wei, Shao, et al (2022). Copyright 2022.…”

Section: Delivery Strategies Of Crispr/cas Systems For Cancer Therapymentioning

confidence: 99%

CRISPR/Cas gene editing and delivery systems for cancer therapy

Li,

Zhou,

et al. 2024

WIREs Nanomed Nanobiotechnol

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CRISPR/Cas systems stand out because of simplicity, efficiency, and other superiorities, thus becoming attractive and brilliant gene‐editing tools in biomedical field including cancer therapy. CRISPR/Cas systems bring promises for cancer therapy through manipulating and engineering on tumor cells or immune cells. However, there have been concerns about how to overcome the numerous physiological barriers and deliver CRISPR components to target cells efficiently and accurately. In this review, we introduced the mechanisms of CRISPR/Cas systems, summarized the current delivery strategies of CRISPR/Cas systems by physical methods, viral vectors, and nonviral vectors, and presented the current application of CRISPR/Cas systems in cancer clinical treatment. Furthermore, we discussed prospects related to delivery approaches of CRISPR/Cas systems.This article is categorized under: Therapeutic Approaches and Drug Discovery > Emerging Technologies Therapeutic Approaches and Drug Discovery > Nanomedicine for Oncologic Disease

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“…However, further in vivo studies are required to confirm its application. PAMAM dendrimers have also been used in CRISPR-Cas9 gene editing in HCT-116 and HT-29 cells [ 192 ] and to promote a CRISPR-Cas9-mediated gene editing of programmed death protein-1 (PD-L1) to obtain tumor immunotherapy in melanoma B16F10 cells [ 193 ]. So far there was no published studies using PAMAM dendrimers to deliver CRISPR-Cas9 machinery into the retina.…”

Section: Nanoparticles (Nps)mentioning

confidence: 99%

Nanoparticles-mediated CRISPR-Cas9 gene therapy in inherited retinal diseases: applications, challenges, and emerging opportunities

et al. 2022

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Inherited Retinal Diseases (IRDs) are considered one of the leading causes of blindness worldwide. However, the majority of them still lack a safe and effective treatment due to their complexity and genetic heterogeneity. Recently, gene therapy is gaining importance as an efficient strategy to address IRDs which were previously considered incurable. The development of the clustered regularly-interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9) system has strongly empowered the field of gene therapy. However, successful gene modifications rely on the efficient delivery of CRISPR-Cas9 components into the complex three-dimensional (3D) architecture of the human retinal tissue. Intriguing findings in the field of nanoparticles (NPs) meet all the criteria required for CRISPR-Cas9 delivery and have made a great contribution toward its therapeutic applications. In addition, exploiting induced pluripotent stem cell (iPSC) technology and in vitro 3D retinal organoids paved the way for prospective clinical trials of the CRISPR-Cas9 system in treating IRDs. This review highlights important advances in NP-based gene therapy, the CRISPR-Cas9 system, and iPSC-derived retinal organoids with a focus on IRDs. Collectively, these studies establish a multidisciplinary approach by integrating nanomedicine and stem cell technologies and demonstrate the utility of retina organoids in developing effective therapies for IRDs.

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Genome editing of PD-L1 mediated by nucleobase-modified polyamidoamine for cancer immunotherapy

Abstract: Immune checkpoint blockade therapy against programmed death protein-1 and its ligand (PD-1/PD-L1) has been accepted as a promising approach to activate the immune system's anti-tumor response. Although small interfering RNA...

Cited by 10 publications

References 46 publications

Comprehensive review of CRISPR-based gene editing: mechanisms, challenges, and applications in cancer therapy

Comprehensive review of CRISPR-based gene editing: mechanisms, challenges, and applications in cancer therapy

CRISPR/Cas gene editing and delivery systems for cancer therapy

Nanoparticles-mediated CRISPR-Cas9 gene therapy in inherited retinal diseases: applications, challenges, and emerging opportunities

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