Progress and pitfalls of gene editing technology in CAR-T cell therapy: a state-of-the-art review

Frontiers in Oncology, Год журнала: 2024, Номер 14

Опубликована: Июнь 7, 2024

CAR-T cell therapy has shown remarkable promise in treating B-cell malignancies, which sparked optimism about its potential to treat other types of cancer as well. Nevertheless, the Expectations solid tumors and non-B hematologic malignancies have not been met. Furthermore, safety concerns regarding use viral vectors current personalized production process are bottlenecks that limit widespread use. In recent years gene editing technology opened a new way unleash latent potentials lessen associated challenges. Moreover, tools paved manufacturing cells fully non-viral approach well providing universal, off-the-shelf product. Despite all advantages strategies, off-target activity classical (ZFNs, TALENs, CRISPR/Cas9) remains major concern. Accordingly, several efforts made reduce their genotoxicity, leading introduction advanced with an improved profile. this review, we begin by examining tools, overview how these technologies currently being applied clinical trials therapies. Following this, explore various strategies aimed at enhancing efficacy therapy.

Язык: Английский

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Novel epigenetic therapeutic strategies and targets in cancer

Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease, Год журнала: 2022, Номер 1868(12), С. 166552 - 166552

Опубликована: Сен. 17, 2022

The critical role of dysregulated epigenetic pathways in cancer genesis, development, and therapy has typically been established as a result scientific technical innovations next generation sequencing. RNA interference, histone modification, DNA methylation chromatin remodelling are processes that control gene expression without causing mutations the DNA. Although abnormalities thought to be symptom cell tumorigenesis malignant events impact tumor growth drug resistance, physicians believe related might key therapeutic target for treatment prevention due reversible nature these processes. A plethora novel strategies addressing epigenetics immuno-oncological complications currently available - ranging from basic editing. – they will subject this comprehensive review. In review, we cover most advancements made field targeting with special emphasis on microbiology, plasma science, biophysics, pharmacology, molecular biology, phytochemistry, nanoscience.

Язык: Английский

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CRISPR-Cas engineering in food science and sustainable agriculture: recent advancements and applications

Bioprocess and Biosystems Engineering, Год журнала: 2023, Номер 46(4), С. 483 - 497

Опубликована: Янв. 28, 2023

Язык: Английский

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A synthetic biology approach for the treatment of pollutants with microalgae

Frontiers in Bioengineering and Biotechnology, Год журнала: 2024, Номер 12

Опубликована: Апрель 5, 2024

The increase in global population and industrial development has led to a significant release of organic inorganic pollutants into water streams, threatening human health ecosystems. Microalgae, encompassing eukaryotic protists prokaryotic cyanobacteria, have emerged as sustainable cost-effective solution for removing these mitigating carbon emissions. Various microalgae species, such C. vulgaris, P. tricornutum, N. oceanica, A. platensis, reinhardtii, demonstrated their ability eliminate heavy metals, salinity, plastics, pesticides. Synthetic biology holds the potential enhance microalgae-based technologies by broadening scope treatment targets improving pollutant removal rates. This review provides an overview recent advances synthetic microalgae, focusing on genetic engineering tools facilitate (heavy metals salinity) (pesticides plastics) compounds. is crucial enhancing mechanisms through gene expression manipulation, DNA introduction cells, generation mutants with altered phenotypes. Additionally, discusses principles tools, emphasizing significance targeting specific metabolic pathways creating phenotypic changes. It also explores use precise CRISPR/Cas9 TALENs, adapt various species. concludes that there much based approaches using but need expansion involved, including universal cloning toolkits efficient rapid assembly transgenic strains, adaptation wider range

Язык: Английский

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Accelerating Diverse Cell-Based Therapies Through Scalable Design

Annual Review of Chemical and Biomolecular Engineering, Год журнала: 2024, Номер 15(1), С. 267 - 292

Опубликована: Апрель 10, 2024

Augmenting cells with novel, genetically encoded functions will support therapies that expand beyond natural capacity for immune surveillance and tissue regeneration. However, engineering at scale transgenic cargoes remains a challenge in realizing the potential of cell-based therapies. In this review, we introduce range applications primary stem We highlight tools advances have launched mammalian cell from bioproduction to precision editing therapeutically relevant cells. Additionally, examine how transgenesis methods genetic cargo designs can be tailored performance. Altogether, offer vision accelerating translation innovative by harnessing diverse types, integrating expanding array synthetic biology tools, building cellular through advanced genome writing techniques.

Язык: Английский

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Gene editing in small and large animals for translational medicine: a review

Animal Reproduction, Год журнала: 2024, Номер 21(1)

Опубликована: Янв. 1, 2024

The CRISPR/Cas9 system is a simpler and more versatile method compared to other engineered nucleases such as Zinc Finger Nucleases (ZFNs) Transcription Activator-Like Effector (TALENs), since its discovery, the efficiency of CRISPR-based genome editing has increased point that multiple different types edits can be made simultaneously. These advances in gene have revolutionized biotechnology by enabling precise with greater simplicity efficacy than ever before. This tool been successfully applied wide range animal species, including cattle, pigs, dogs, small animals. Engineered cut at specific target positions, triggering cell's mechanisms repair damage introduce mutation genomic site. review discusses novel genome-based tools, methods developed improve specificity, use gene-editing on models translational medicine, main challenges limitations approaches.

Язык: Английский

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Biotechnological techniques for sugarcane crop improvement, applications and challenges

Journal of Crop Science and Biotechnology, Год журнала: 2025, Номер unknown

Опубликована: Янв. 27, 2025

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Bacterial DNA methylases as novel molecular and synthetic biology tools: recent developments

Applied Microbiology and Biotechnology, Год журнала: 2025, Номер 109(1)

Опубликована: Март 6, 2025

Bacterial DNA methylases are a diverse group of enzymes which have been pivotal in the development technologies with applications including genetic engineering, bacteriology, biotechnology and agriculture. This review describes bacterial methylase types, main for targeted methylation or demethylation recent roles these molecular synthetic biology. can be exocyclic endocyclic exist as orphan part restriction-modifications (R-M) systems. As group, they display rich diversity sequence-specificity. Additional targeting involve using fusion proteins combining DNA-binding protein (DNBP) such zinc-finger (ZF), transcription activator-like effector (TALE) CRISPR/dCas9. contributed significantly to creation novel assembly techniques, improvement transformation crop plant engineering. Future studies define characteristics more potential identify new tools value biology widespread applications. KEY POINTS: • used direct specific sequences target has applied improve increase efficiency Site-selective alter gene expression phenotype.

Язык: Английский

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Molecular tools for genome editing in plants: a synthetic overview

Vegetable Research, Год журнала: 2025, Номер 5(1), С. 0 - 0

Опубликована: Янв. 1, 2025

Язык: Английский

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Precision Editing as a Therapeutic Approach for β-Hemoglobinopathies

International Journal of Molecular Sciences, Год журнала: 2023, Номер 24(11), С. 9527 - 9527

Опубликована: Май 31, 2023

Beta-hemoglobinopathies are the most common genetic disorders worldwide, caused by a wide spectrum of mutations in β-globin locus, and associated with morbidity early mortality case patient non-adherence to supportive treatment. Allogeneic transplantation hematopoietic stem cells (allo-HSCT) used be only curative option, although indispensable need for an HLA-matched donor markedly restricted its universal application. The evolution gene therapy approaches made possible ex vivo delivery therapeutic β- or γ- globin into patient-derived followed corrected myeloablated patients, having led high rates transfusion independence (thalassemia) complete resolution painful crises (sickle cell disease-SCD). Hereditary persistence fetal hemoglobin (HPFH), syndrome characterized increased γ-globin levels, when co-inherited β-thalassemia SCD, converts hemoglobinopathies benign condition mild clinical phenotype. rapid development precise genome editing tools (ZFN, TALENs, CRISPR/Cas9) over last decade has allowed targeted introduction mutations, resulting disease-modifying outcomes. In this context, have successfully been HPFH-like both

Язык: Английский

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