腺相关病毒载体受体AAVR和共受体及其突变对基因治疗过程中腺相关病毒回归和转导的影响——Tirthal Rai Usha Adiga Deepika Kamath M综述

Q3 Pharmacology, Toxicology and Pharmaceutics
Tirthal Rai, U. Adiga, Deepika Kamath
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引用次数: 0

摘要

基因治疗是一种正在研究的实验性治疗方法,旨在纠正导致疾病发展的缺陷基因。为了治疗癌症和遗传疾病,研究人员正在研究各种基因治疗技术。寻找合适的载体将DNA转移到组织中是基因治疗最困难的方面之一。一些基因治疗载体存在感染静止细胞和分裂细胞、引发免疫反应、缺乏不确定表达和可重复的高滴度的问题。腺病毒、逆转录病毒和重组修饰腺相关病毒是基因治疗载体(rAAV)的主要竞争者。基因治疗的问题可以通过腺相关病毒(AAV)来解决。血友病是一种可以通过使用AAV作为载体的基因治疗获益的疾病。本综述的目的是强调腺相关病毒受体(AAVR)和共受体在腺相关病毒(AAV)向性和转导中的作用,在体外和体内血友病的基因治疗中。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The Influence Of Adeno Associated Virus Vector Receptor AAVR And Co-Receptors And Their Mutations On Tropism And Transduction Of Adeno Associated Virus During Gene Therapy A Review Tirthal Rai Usha Adiga Deepika Kamath M
Gene therapy is an experimental treatment being investigated to correct defective genes that are responsible for disease development. To treat cancer and genetic illnesses, researchers are looking into a variety of gene therapy techniques. Finding a proper vector to transfer DNA into tissues is one of the most difficult aspects of gene therapy. Some gene therapy vectors have issues with infecting both quiescent and dividing cells, provoking an immunological response, lack of indefinite expression, and reproducible high titre. The adenovirus, retrovirus, and recombinant modified adeno-associated virus are top contenders for gene therapy vectors (rAAV). The issues with gene therapy may be resolved by the adeno associated virus (AAV). Hemophilia is a condition which may be benefited by the gene therapy using AAV as vector. Aim of this review is to emphasize on the role of adeno associated virus receptors(AAVR)  and co-receptors in tropism and transduction of adeno associated virus(AAV) in the gene therapy of hemophilia In Vitro as well as In Vivo.  
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