基因疗法中用于工程化的病毒载体。
Viral vectors engineered for gene therapy.
发表日期:2023
作者:
Kenneth Lundstrom
来源:
International Review of Cell and Molecular Biology
摘要:
基因治疗近年来取得了重大进展。通过高效工程改进递送和安全性方面,病毒载体做出了重要贡献。目前已针对多种病症如癌症、心血管病、代谢疾病、血液病、神经病、肌肉病、眼科疾病、传染病和免疫缺陷进行了病毒载体研究。病毒载体方面的研究基于腺病毒、腺相关病毒、单纯疱疹病毒、逆转录病毒(包括慢病毒)、阿尔法病毒、黄病毒、麻疹病毒、恶性贫血病毒、新城疫病毒、痘病毒、小肠病毒、冠状病毒、重组病毒和多模病毒等病毒。通过动物模型已经证明了不同病症的概念验证。在临床试验中也已经取得了治疗疗效。已经批准使用基于病毒载体的治疗药物治疗癌症,以及血液病、代谢病和神经病。此外,已经批准使用基于病毒载体的疫苗用于治疗COVID-19和埃博拉病毒疾病。© 2023版权归Elsevier Inc.所有。
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.Copyright © 2023. Published by Elsevier Inc.