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...
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 |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2020-06-01
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Series: | Molecular Therapy: Methods & Clinical Development |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050120300371 |
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