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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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Ran, Gai, Chen, Xiao, Xie, Yilin, Zheng, Qingyun, Xie, Jinyan, Yu, Chenghui, Pittman, Nikea, Qi, Sixian, Yu, Fa-Xing, Agbandje-McKenna, Mavis, Srivastava, Arun, Ling, Chen
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7114622/
https://ncbi.nlm.nih.gov/pubmed/32258217
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.03.007
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