Characterization and biodistribution of under-employed gene therapy vector AAV7.

Autor:	Yost SA; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Firlar E; Institute of Quantitative Biomedicine and Rutgers CryoEM & Nanoimaging Facility, Rutgers, The State University of New Jersey , Piscataway, New Jersey, USA., Glenn JD; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Carroll HB; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Foltz S; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Giles AR; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Egley JM; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Firnberg E; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Cho S; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Nguyen T; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Henry WM; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Janczura KJ; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Bruder J; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Liu Y; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Danos O; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Karumuthil-Melethil S; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Pannem S; Invicro LLC , Needham, Massachusetts, USA., Yost V; Invicro LLC , Needham, Massachusetts, USA., Engelson Y; Invicro LLC , Needham, Massachusetts, USA., Kaelber JT; Institute of Quantitative Biomedicine and Rutgers CryoEM & Nanoimaging Facility, Rutgers, The State University of New Jersey , Piscataway, New Jersey, USA., Dimant H; Invicro LLC , Needham, Massachusetts, USA.; Emit Imaging , Baltimore, Maryland, USA., Smith JB; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA., Mercer AC; Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA.
Jazyk:	angličtina
Zdroj:	Journal of virology [J Virol] 2023 Nov 30; Vol. 97 (11), pp. e0116323. Date of Electronic Publication: 2023 Oct 16.
DOI:	10.1128/jvi.01163-23
Abstrakt:	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. Competing Interests: All REGENXBIO employees earn salary and own stock in REGENXBIO, Inc. All Invicro employees earn salary from Invicro.
Databáze:	MEDLINE
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