Genome length determination in adeno-associated virus vectors with mass photometry

Autor: Cornelia Hiemenz, Nadine Baumeister, Constanze Helbig, Andrea Hawe, Sabrina Babutzka, Stylianos Michalakis, Wolfgang Friess, Tim Menzen
Jazyk: angličtina
Rok vydání: 2023
Předmět:
Zdroj: Molecular Therapy: Methods & Clinical Development, Vol 31, Iss , Pp 101162- (2023)
Druh dokumentu: article
ISSN: 2329-0501
DOI: 10.1016/j.omtm.2023.101162
Popis: Recombinant adeno-associated viruses (rAAVs) are attractive therapeutic viral vectors for gene delivery. To ensure the efficacy and safety of rAAV-based therapies, comprehensive characterization of the adeno-associated virus (AAV) capsids is essential. Mass photometry (MP) provides the advantage of short analysis times, low sample consumption, and high accuracy of molecular mass determination. Despite having just recently emerged, MP has already been used to characterize AAV genome content and quantify filled/empty capsid ratios. In this study, we explored three approaches for the application of MP to assess genome length in AAVs. In approach 1, genome length in intact AAVs was approximated with good precision (coefficient of variation [%CV]
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