Characterization and biodistribution of under-employed gene therapy vector AAV7.
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ABSTRACT: Importance
The use of adeno-associated viruses (AAVs) as gene delivery vectors has vast potential for the treatment of many severe human diseases. Over one hundred naturally existing AAV capsid variants have been described and classified into phylogenetic clades based on their sequences. AAV8, AAV9, AAVrh.10, and other intensively studied capsids have been propelled into pre-clinical and clinical use, and more recently, marketed products; however, less-studied capsids may also have desirable properties (e.g., potency differences, tissue tropism, reduced immunogenicity, etc.) that have yet to be thoroughly described. These data will help build a broader structure-function knowledge base in the field, present capsid engineering opportunities, and enable the use of novel capsids with unique properties.
SUBMITTER: Yost SA
PROVIDER: S-EPMC10688378 | biostudies-literature | 2023 Nov
REPOSITORIES: biostudies-literature
Publications
Characterization and biodistribution of under-employed gene therapy vector AAV7.
Yost Samantha A SA Firlar Emre E Glenn Justin D JD Carroll Hayley B HB Foltz Steven S Giles April R AR Egley Jenny M JM Firnberg Elad E Cho Sungyeon S Nguyen Trang T Henry William M WM Janczura Karolina J KJ Bruder Joseph J Liu Ye Y Danos Olivier O Karumuthil-Melethil Subha S Pannem Sanjana S Yost Valerie V Engelson Yelena Y Kaelber Jason T JT Dimant Hemi H Smith Jared B JB Mercer Andrew C AC
Journal of virology 20231016 11
<h4>Importance</h4>The use of adeno-associated viruses (AAVs) as gene delivery vectors has vast potential for the treatment of many severe human diseases. Over one hundred naturally existing AAV capsid variants have been described and classified into phylogenetic clades based on their sequences. AAV8, AAV9, AAVrh.10, and other intensively studied capsids have been propelled into pre-clinical and clinical use, and more recently, marketed products; however, less-studied capsids may also have desir ...[more]
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