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The Current Status of Gene Therapy for the Treatment of Cancer

机译:基因疗法治疗癌症的当前状态

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Gene therapy is the administration of foreign genomic material into the host tissue to modify the expression of a gene product or to change the biological properties of cells for therapeutic use. Initially, the major objective of gene therapy was to manage genetic diseases, but now different disorders with several patterns of acquired and inherited disorders are targets of gene therapy. Over three decades, the advancement of Genome engineering technologies facilitated gene therapy for the prevention and management of intractable diseases. Researchers are advancing with cautious optimism that safe and effective treatment will give to patients with single-gene disorders and complex acquired disorders. To date, over 3000 genes associates with disease-causing mutations, and about 2600 gene therapy trials are undergoing for the management of various disorders. This review summarizes the principles of genome-editing approaches, such as zinc finger nucleases, transcription activator-like effector nucleases, meganucleases, and the CRISPR/Cas9 system with the underlying mechanisms. This review also explains the types of gene delivery systems as viral [adenoviral, adeno association, herpes simplex virus] and nonviral delivery systems (physical: DNA bombardment, electroporation) and (chemical: Cationic lipids, cationic polymers). Finally, this review summarizes gene therapy medicines approved to treat cancer in detail, including names, indications, vectors, and mode of gene therapy. Gene therapy becomes an alternative to an existing management for different diseases. Therefore, gene products with safe vectors and better biotechnologies play a significant role in the prophylaxis and management of various disorders in the future.
机译:基因治疗是将外来基因组材料的施用到宿主组织中以改变基因产物的表达或改变细胞的生物学性能以进行治疗用途。最初,基因治疗的主要目标是管理遗传疾病,但现在具有多种获得的和遗传性疾病模式的不同障碍是基因治疗的目标。三十年来,基因组工程技术的进步促进了基因治疗,用于预防和管理难治性疾病。研究人员正在谨慎态度,即安全有效的治疗将向患有单基因障碍和复杂的患者的患者提供。迄今为止,超过3000个基因缔结疾病突变,并对各种疾病的管理进行了大约2600个基因治疗试验。本综述总结了基因组编辑方法的原理,例如锌指核酸酶,转录活化剂样效应核酸酶,Meganucl酶和CRISPR / CAS9系统,具有下面的机制。该审查还解释了基因递送系统的类型作为病毒[腺病毒,腺嘌呤关联,单纯疱疹病毒]和非血管递送系统(物理:DNA轰击,电穿孔)和(化学品:阳离子脂质,阳离子聚合物)。最后,本综述总结了批准的基因治疗药物详细治疗癌症,包括基因治疗的名称,适应症,载体和模式。基因治疗成为对不同疾病的现有管理的替代方案。因此,具有安全载体和更好的生物技术的基因产品在未来的预防和管理中发挥着重要作用。

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