Site-Directed Mutagenesis Improves the Transduction Efficiency of Capsid Library-Derived Recombinant AAV Vectors

Site-Directed Mutagenesis Improves the Transduction Efficiency of Capsid Library-Derived Recombinant AAV Vectors

Recombinant adeno-associated virus (rAAV) vectors selected from capsid libraries present enormous advantages in high selectivity of tissue tropism and their potential use in human gene therapy applications. For example, rAAV-LK03, was used in a gene therapy trial for hemophilia A (ClinicalTrials.gov...

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Bibliographic Details
Main Authors: Gai Ran, Xiao Chen, Yilin Xie, Qingyun Zheng, Jinyan Xie, Chenghui Yu, Nikea Pittman, Sixian Qi, Fa-Xing Yu, Mavis Agbandje-McKenna, Arun Srivastava, Chen Ling
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
gene therapy
rAAV vector
library selection
site-directed mutagenesis
transduction efficiency
vector distribution
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120300371

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http://www.sciencedirect.com/science/article/pii/S2329050120300371

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