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Viral Vector-Based Gene Therapy.

Authors :
Li, Xuedan
Le, Yang
Zhang, Zhegang
Nian, Xuanxuan
Liu, Bo
Yang, Xiaoming
Source :
International Journal of Molecular Sciences. May2023, Vol. 24 Issue 9, p7736. 21p.
Publication Year :
2023

Abstract

Gene therapy is a technique involving the modification of an individual's genes for treating a particular disease. The key to effective gene therapy is an efficient carrier delivery system. Viral vectors that have been artificially modified to lose their pathogenicity are used widely as a delivery system, with the key advantages of their natural high transduction efficiency and stable expression. With decades of development, viral vector-based gene therapies have achieved promising clinical outcomes. Currently, the three key vector strategies are based on adeno-associated viruses, adenoviruses, and lentiviruses. However, certain challenges, such as immunotoxicity and "off-target", continue to exist. In the present review, the above three viral vectors are discussed along with their respective therapeutic applications. In addition, the major translational challenges encountered in viral vector-based gene therapies are summarized, and the possible strategies to address these challenges are also discussed. [ABSTRACT FROM AUTHOR]

Details

Language :
English
ISSN :
16616596
Volume :
24
Issue :
9
Database :
Academic Search Index
Journal :
International Journal of Molecular Sciences
Publication Type :
Academic Journal
Accession number :
163689872
Full Text :
https://doi.org/10.3390/ijms24097736