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AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes

9 Jul 2022

Abstract excerpt

AAV vectors are promising delivery tools for human gene therapy. However, broad tissue tropism and pre-existing immunity against natural serotypes limit their clinical use. We identified two AAV capsid variants, AAV2-THGTPAD and AAV2-NLPGSGD, by in vivo AAV2 peptide display library screening in a murine model of pressure overload-induced cardiac hypertrophy. Both variants showed significantly improved efficacy in...

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PMID: 35810332
DOI: 10.1016/j.ymthe.2022.07.003

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