Viral vectors engineered for gene therapy.

3区 生物学 Q1 Biochemistry, Genetics and Molecular Biology
Kenneth Lundstrom
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引用次数: 0

Abstract

Gene therapy has seen major progress in recent years. Viral vectors have made a significant contribution through efficient engineering for improved delivery and safety. A large variety of indications such as cancer, cardiovascular, metabolic, hematological, neurological, muscular, ophthalmological, infectious diseases, and immunodeficiency have been targeted. Viral vectors based on adenoviruses, adeno-associated viruses, herpes simplex viruses, retroviruses including lentiviruses, alphaviruses, flaviviruses, measles viruses, rhabdoviruses, Newcastle disease virus, poxviruses, picornaviruses, reoviruses, and polyomaviruses have been used. Proof-of-concept has been demonstrated for different indications in animal models. Therapeutic efficacy has also been achieved in clinical trials. Several viral vector-based drugs have been approved for the treatment of cancer, and hematological, metabolic, and neurological diseases. Moreover, viral vector-based vaccines have been approved against COVID-19 and Ebola virus disease.

用于基因治疗的病毒载体。
基因治疗近年来取得了重大进展。通过有效的工程设计,病毒载体为改善递送和安全性做出了重大贡献。各种各样的适应症,如癌症、心血管、代谢、血液、神经、肌肉、眼科、传染病和免疫缺陷等已成为目标。基于腺病毒、腺相关病毒、单纯疱疹病毒、逆转录病毒(包括慢病毒、甲病毒、黄病毒、麻疹病毒、横纹肌病毒、新城疫病毒、痘病毒、小核糖核酸病毒、呼肠孤病毒和多瘤病毒)的病毒载体已被使用。不同适应症的概念验证已在动物模型中得到证实。在临床试验中也取得了治疗效果。几种基于病毒载体的药物已被批准用于治疗癌症、血液、代谢和神经系统疾病。此外,针对COVID-19和埃博拉病毒病的病毒载体疫苗已获批准。
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来源期刊
International review of cell and molecular biology
International review of cell and molecular biology BIOCHEMISTRY & MOLECULAR BIOLOGY-CELL BIOLOGY
CiteScore
7.70
自引率
0.00%
发文量
67
审稿时长
>12 weeks
期刊介绍: International Review of Cell and Molecular Biology presents current advances and comprehensive reviews in cell biology-both plant and animal. Articles address structure and control of gene expression, nucleocytoplasmic interactions, control of cell development and differentiation, and cell transformation and growth. Authored by some of the foremost scientists in the field, each volume provides up-to-date information and directions for future research.
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