Adeno-associated virus serotype 2 capsid variants for improved liver-directed gene therapy.
Hepatology (Baltimore, Md.)（2023）
摘要
In conclusion, MLIV.K and MLIV.A are AAV peptide display variants selected in immunocompetent mice with improved hepatocyte tropism and transduction efficiency. Because these features are maintained across species, MLIV variants provide remarkable potential for translation of therapeutic approaches from mice to men.
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关键词
capsid variants,adeno‐associated,gene,liver‐directed
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