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Development of AAV Variants with Human Hepatocyte Tropism and Neutralizing Antibody Escape Capacity

Adeno-associated virus (AAV) vectors have been successfully used in patients with bleeding disorders and blindness. For human liver targeting, two major factors restrict effective AAV transduction after systemic administration of AAV vectors: human hepatocyte tropism and neutralizing antibodies (Nab...

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發表在:Mol Ther Methods Clin Dev
Main Authors: Pei, Xiaolei, Shao, Wenwei, Xing, Allene, Askew, Charles, Chen, Xiaojing, Cui, Caibin, Abajas, Yasmina L., Gerber, David A., Merricks, Elizabeth P., Nichols, Timothy C., Li, Wuping, Samulski, R. Jude, Li, Chengwen
格式: Artigo
語言:Inglês
出版: American Society of Gene & Cell Therapy 2020
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC7329936/
https://ncbi.nlm.nih.gov/pubmed/32637455
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.06.003
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