Adeno-associated virus serotype 2 capsid variants for improved liver-directed gene therapy.

Nadja MeumannMarti Cabanes-CreusMoritz ErteltRenina Gale NavarroJulie LuciforaQinggong YuanKarin Nien-HuberAhmed AbdelrahmanXuan-Khang VuLiang ZhangAnn-Christin FrankeChristian SchmithalsAlbrecht PiiperAnnabelle VogtMaria Gonzalez-CarmonaJochen T FruehEvelyn UllrichPhilip MeulemanSteven R TalbotMargarete OdenthalMichael OttErhard SeifriedClara T SchoederJoachim SchwäbleLeszek LisowskiHildegard Büning

Published in: Hepatology (Baltimore, Md.) (2022)

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.

Keyphrases

gene therapy
copy number
high fat diet induced
dengue virus
escherichia coli
wild type
metabolic syndrome
middle aged
multidrug resistant
liver injury
disease virus
drug induced