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Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges

Liyuan Zhao, Zixuan Yang, Minhui Zheng, Lei Shi, Mengyun Gu, Gang Liu, Feng Miao, Yan Chang, Huang Fanghua, Naping Tang

2023Genes & Diseases32 citationsDOIOpen Access PDF

Abstract

In recent years, significant breakthroughs have been made in the field of gene therapy. Adeno-associated virus (AAV) is one of the most promising gene therapy vectors and a powerful tool for delivering the gene of interest. Among the AAV vectors, AAV serotype 8 (AAV8) has attracted much attention for its efficient and stable gene transfection into specific tissues. Currently, recombinant AAV8 has been widely used in gene therapy research on a variety of diseases, including genetic diseases, cancers, autoimmune diseases, and viral diseases. This paper reviewed the applications and challenges of using AAV8 as a vector for gene therapy, with the aim of providing a valuable resource for those pursuing the application of viral vectors in gene therapy.

Topics & Concepts

Adeno-associated virusGenetic enhancementVector (molecular biology)Viral vectorGeneVirologyVectors in gene therapyVirusRecombinant DNAGene deliveryComputational biologyBiologyTransfectionMedicineBioinformaticsGeneticsVirus-based gene therapy researchViral Infectious Diseases and Gene Expression in InsectsRNA Interference and Gene Delivery
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