Dendritic cells in inborn errors of immunity

Gupta, Sudhir; Agrawal, Anshu

doi:10.3389/fimmu.2023.1080129

Cited by 6 publications

(3 citation statements)

References 182 publications

(233 reference statements)

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“…DCs are critical in antigen presentation to reduce adaptive anti‐tumor immunity. [ 26 ] The proportion of mature DCs was further quantified (Figure 6H,J ). The percentage of mature DCs (CD11c + CD80 + CD86 + ) in KET/P780 NPs and CS@KET/P780 NPs groups were 9.25% and 11.4%, respectively, both of which are higher than the control group (2.72%).…”

Section: Resultsmentioning

confidence: 99%

Mitochondrial‐Targeted CS@KET/P780 Nanoplatform for Site‐Specific Delivery and High‐Efficiency Cancer Immunotherapy in Hepatocellular Carcinoma

Liu,

Tian,

Ming

et al. 2024

Advanced Science

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Hepatocellular carcinoma (HCC) is a form of malignancy with limited curative options available. To improve therapeutic outcomes, it is imperative to develop novel, potent therapeutic modalities. Ketoconazole (KET) has shown excellent therapeutic efficacy against HCC by eliciting apoptosis. However, its limited water solubility hampers its application in clinical treatment. Herein, a mitochondria‐targeted chemo‐photodynamic nanoplatform, CS@KET/P780 NPs, is designed using a nanoprecipitation strategy by integrating a newly synthesized mitochondria‐targeted photosensitizer (P780) and chemotherapeutic agent KET coated with chondroitin sulfate (CS) to amplify HCC therapy. In this nanoplatform, CS confers tumor‐targeted and subsequently pH‐responsive drug delivery behavior by binding to glycoprotein CD44, leading to the release of P780 and KET. Mechanistically, following laser irradiation, P780 targets and destroys mitochondrial integrity, thus inducing apoptosis through the enhancement of reactive oxygen species (ROS) buildup. Meanwhile, KET‐induced apoptosis synergistically enhances the anticancer effect of P780. In addition, tumor cells undergoing apoptosis can trigger immunogenic cell death (ICD) and a longer‐term antitumor response by releasing tumor‐associated antigens (TAAs) and damage‐associated molecular patterns (DAMPs), which together contribute to improved therapeutic outcomes in HCC. Taken together, CS@KET/P780 NPs improve the bioavailability of KET and exhibit excellent therapeutic efficacy against HCC by exerting chemophototherapy and antitumor immunity.

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Section: Resultsmentioning

confidence: 99%

Mitochondrial‐Targeted CS@KET/P780 Nanoplatform for Site‐Specific Delivery and High‐Efficiency Cancer Immunotherapy in Hepatocellular Carcinoma

Liu,

Tian,

Ming

et al. 2024

Advanced Science

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“…The CRISPR-Cas9 system demonstrates superior specificity and efficacy, making it a promising tool for treating various genetic and non-genetic diseases. Its extensive utilization ranges from addressing challenging conditions such as inborn errors of immunity and Huntington's disease [137,138] to overcoming drug resistance in cancer cells. [139,140] Clinical trials are underway to evaluate the safety and effectiveness of therapeutic approaches using CRISPR-Cas (Table S1, started after June 9th, 2021.…”

Section: Potential Applicationsmentioning

confidence: 99%

The immune system of prokaryotes: potential applications and implications for gene editing

Liu,

Wang

et al. 2024

Biotechnology Journal

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Gene therapy has revolutionized the treatment of genetic diseases. Spearheading this revolution are sophisticated genome editing methods such as TALENs, ZFNs, and CRISPR‐Cas, which trace their origins back to prokaryotic immune systems. Prokaryotes have developed various antiviral defense systems to combat viral attacks and the invasion of genetic elements. The comprehension of these defense mechanisms has paved the way for the development of indispensable tools in molecular biology. Among them, restriction endonuclease originates from the innate immune system of bacteria. The CRISPR‐Cas system, a widely applied genome editing technology, is derived from the prokaryotic adaptive immune system. Single‐base editing is a precise editing tool based on CRISPR‐Cas system that involves deamination of target base. It is worth noting that prokaryotes possess deamination enzymes as part of their defense arsenal over foreign genetic material. Furthermore, prokaryotic Argonauts (pAgo) proteins, also function in anti‐phage defense, play an important role in complementing the CRISPR‐Cas system by addressing certain limitations it may have. Recent studies have also shed light on the significance of Retron, a reverse transcription transposon previously showed potential in genome editing, has also come to light in the realm of prokaryotic immunity. These noteworthy findings highlight the importance of studying prokaryotic immune system for advancing genome editing techniques. Here, both the origin of prokaryotic immunity underlying aforementioned genome editing tools, and potential applications of deaminase, pAgo protein and reverse transcriptase in genome editing among prokaryotes were introduced, thus emphasizing the fundamental mechanism and significance of prokaryotic immunity.

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“…Zinc finger nucleases (ZFN), transcription activator-like effector nucleases (TALEN), and clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein (Cas) systems, particularly CRISPR/Cas9, have significantly advanced the field of gene therapy. All of these methods achieve site-specific gene editing, and several related products using these technologies are currently in clinical trialss [7,8]. Gene-editing technologies are used not only to correct pathogenic mutations for clinical applications but also to modify immune cells, such as chimeric antigen receptor T (CAR-T) cells [9].…”

Section: Introductionmentioning

confidence: 99%

The research progress of correcting pathogenic mutations by base editing

Li,

Zhang,

Huang

2024

Obstetrics and Gynecology

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Over 6500 Mendelian disorders have been documented, with approximately 4500 genes linked to these conditions. The majority of inherited diseases present in childhood and, currently, lack effective treatments, which imposes significant economic and psychological burdens on families and society. Gene editing, particularly base editing, offers an effective and safe strategy for repairing pathogenic point mutations. It has the potential to become a treatment, even a cure, for rare diseases. Currently, multiple gene editing-related drugs have entered clinical trials. In this chapter, we summarize the various gene editing systems, including CRISPR/Cas, base editing, and prime editing. We then focus on the current research progress of base editing in correcting pathogenic mutations. This includes applications such as building animal models, correcting mutations in various diseases, germline cell editing, delivery methods, and approved clinical trials. Finally, we discuss current challenges related to delivery methods, efficiency, precision, and cost.

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Dendritic cells in inborn errors of immunity

Cited by 6 publications

References 182 publications

Mitochondrial‐Targeted CS@KET/P780 Nanoplatform for Site‐Specific Delivery and High‐Efficiency Cancer Immunotherapy in Hepatocellular Carcinoma

Mitochondrial‐Targeted CS@KET/P780 Nanoplatform for Site‐Specific Delivery and High‐Efficiency Cancer Immunotherapy in Hepatocellular Carcinoma

The immune system of prokaryotes: potential applications and implications for gene editing

The research progress of correcting pathogenic mutations by base editing

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