Enhancement of recombinant adeno-associated virus activity by improved stoichiometry and homogeneity of capsid protein assembly.
adeno-associated virus
analytical ultracentrifugation
charge detection-mass spectrometry
transduction efficiency
viral protein stoichiometry
Journal
Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857
Informations de publication
Date de publication:
14 Dec 2023
14 Dec 2023
Historique:
received:
17
07
2023
accepted:
21
10
2023
medline:
29
11
2023
pubmed:
29
11
2023
entrez:
29
11
2023
Statut:
epublish
Résumé
Studies of recombinant adeno-associated virus (rAAV) revealed the mixture of full particles with different densities in rAAV. There are no conclusive results because of the lack of quantitative stoichiometric viral proteins, encapsidated DNA, and particle level analyses. We report the first comprehensive characterization of low- and high-density rAAV serotype 2 particles. Capillary gel electrophoresis showed high-density particles possessing a designed DNA encapsidated in the capsid composed of (VP1 + VP2)/VP3 = 0.27, whereas low-density particles have the same DNA but with a different capsid composition of (VP1 + VP2)/VP3 = 0.31, supported by sedimentation velocity-analytical ultracentrifugation and charge detection-mass spectrometry.
Identifiants
pubmed: 38027055
doi: 10.1016/j.omtm.2023.101142
pii: S2329-0501(23)00181-X
pmc: PMC10663676
doi:
Types de publication
Journal Article
Langues
eng
Pagination
101142Informations de copyright
© 2023 The Author(s).
Déclaration de conflit d'intérêts
The authors declare the following financial interests/personal relationships that may be considered as potential competing interests: T.M. and M.F. report a relationship with U-Medico Inc. that includes employment. M.M. reports a relationship with Osaka Consolidated Laboratory that includes employment. S.A., A.H., K.R., K.G., and S.P. report a relationship with Waters corporation that includes employment.
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