microRNA-based Genetic Therapy in Leukemia: Properties, Delivery, and Experimental Models.

IF 3.8 4区医学 Q2 GENETICS & HEREDITY

Current gene therapy Pub Date : 2023-01-01 DOI:10.2174/1566523223666230426153622

Nayra Oliveira Prado, Denise Kusma Wosniaki, Anelis Maria Marin, Carolina Mathias, Heloisa Bruna Soligo Sanchuki, Dalila Luciola Zanette, Mateus Nóbrega Aoki

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Abstract

Leukemia is a type of cancer that affects white blood cells. In this disease, immature blood cells undergo genetic mutations, leading to excessive replication and reduced cell death compared to healthy cells. In cancer, there may be the activation of oncogenes and the deactivation of tumor suppressor genes that control certain cellular functions. Despite the undeniable contribution to the patient's recovery, conventional cancer treatments may have some not-so-beneficial effects. In this case, gene therapy appears as an alternative to classical treatments. Gene therapy delivers genetic material to cells to replace or modify dysfunctional genes, a safe method for neoplasms. One of the types of nucleic acids explored in gene therapy is microRNA (miRNA), a group of endogenous, non-proteincoding, small single-stranded RNA molecules involved in the regulation of gene expression, cell division, differentiation, angiogenesis, migration, apoptosis, and carcinogenesis. This review aims to bring together the most recent advances found in the literature on cancer gene therapy based on microRNAs in the oncological context, focusing on leukemia.

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基于微rna的白血病基因治疗:特性、传递和实验模型。

白血病是一种影响白细胞的癌症。在这种疾病中，未成熟的血细胞发生基因突变，导致与健康细胞相比过度复制和减少细胞死亡。在癌症中，可能存在致癌基因的激活和控制某些细胞功能的肿瘤抑制基因的失活。尽管对病人的康复有不可否认的贡献，但传统的癌症治疗可能有一些不那么有益的影响。在这种情况下，基因治疗似乎是传统治疗的替代方案。基因治疗将遗传物质传递到细胞中，以取代或修饰功能失调的基因，这是治疗肿瘤的一种安全方法。在基因治疗中探索的核酸类型之一是microRNA (miRNA)，这是一组内源性、非蛋白质编码的小单链RNA分子，参与基因表达、细胞分裂、分化、血管生成、迁移、凋亡和癌变的调控。本综述旨在汇集基于microRNAs的肿瘤基因治疗的最新进展，重点是白血病。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Current gene therapy 医学-遗传学

CiteScore

6.70

自引率

2.80%

发文量

期刊介绍： Current Gene Therapy is a bi-monthly peer-reviewed journal aimed at academic and industrial scientists with an interest in major topics concerning basic research and clinical applications of gene and cell therapy of diseases. Cell therapy manuscripts can also include application in diseases when cells have been genetically modified. Current Gene Therapy publishes full-length/mini reviews and original research on the latest developments in gene transfer and gene expression analysis, vector development, cellular genetic engineering, animal models and human clinical applications of gene and cell therapy for the treatment of diseases. Current Gene Therapy publishes reviews and original research containing experimental data on gene and cell therapy. The journal also includes manuscripts on technological advances, ethical and regulatory considerations of gene and cell therapy. Reviews should provide the reader with a comprehensive assessment of any area of experimental biology applied to molecular medicine that is not only of significance within a particular field of gene therapy and cell therapy but also of interest to investigators in other fields. Authors are encouraged to provide their own assessment and vision for future advances. Reviews are also welcome on late breaking discoveries on which substantial literature has not yet been amassed. Such reviews provide a forum for sharply focused topics of recent experimental investigations in gene therapy primarily to make these results accessible to both clinical and basic researchers. Manuscripts containing experimental data should be original data, not previously published.