Atchison RW, Casto BC, Hammon WMD. Adenovirus-associated defective virus particles. Science. 1965;149:754–6.
Article CAS PubMed Google Scholar
Li C, Samulski RJ. Engineering adeno-associated virus vectors for gene therapy. Nat Rev Genet. 2020;21:255–2.
Article CAS PubMed Google Scholar
Pupo A, Fernández A, Low SH, François A, Suárez-Amarán L, Samulski RJ. AAV vectors: The Rubik’s cube of human gene therapy. Mol Ther. 2022;30:3515–41.
Article CAS PubMed PubMed Central Google Scholar
Wang JH, Gessler DJ, Zhan W, Gallagher TL, Gao G. Adeno-associated virus as a delivery vector for gene therapy of human diseases. Signal Transduct Target Ther. 2024;9:1–33.
Darrow JJ. Luxturna: FDA documents reveal the value of a costly gene therapy. Drug Discov Today. 2019;24:949–54.
Ertl HCJ. Immunogenicity and toxicity of AAV gene therapy. Front Immunol. 2022;13:975803.
Article CAS PubMed PubMed Central Google Scholar
Kishimoto TK, Samulski RJ. Addressing high dose AAV toxicity – ‘one and done’ or ‘slower and lower’? Expert Opin Biol Ther. 2022;22:1067–71.
McColl-Carboni A, Dollive S, Laughlin S, Lushi R, MacArthur M, Zhou S, et al. Analytical characterization of full, intermediate, and empty AAV capsids. Gene Ther 2024. 2024;31:285–94.
Hösel M, Broxtermann M, Janicki H, Esser K, Arzberger S, Hartmann P, et al. Toll-like receptor 2-mediated innate immune response in human nonparenchymal liver cells toward adeno-associated viral vectors. Hepatology. 2012;55:287–97.
Jarvi N, Hofman K, Venkatesh A, Gorecki E, Balu-Iyer SV. Immunogenicity risk assessment of empty capsids present in adeno-associated viral vectors using predictive innate immune responses. J Pharm Sci. 2024;113:3457–69.
Article CAS PubMed Google Scholar
Wright JF. Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment. Biomedicines 2014;2:80–97.
Kontogiannis T, Braybrook J, McElroy C, Foy C, Whale AS, Quaglia M, et al. Characterization of AAV vectors: A review of analytical techniques and critical quality attributes. Mol Ther Methods Clin Dev. 2024;32:101309.
Article CAS PubMed PubMed Central Google Scholar
Tai PWL, Xie J, Fong K, Seetin M, Heiner C, Su Q, et al. Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras. Mol Ther Methods Clin Dev. 2018;9:130–41.
Article CAS PubMed PubMed Central Google Scholar
Brimble MA, Winston SM, Davidoff AM. Stowaways in the cargo: Contaminating nucleic acids in rAAV preparations for gene therapy. Mol Ther. 2023;31:2826–38.
Article CAS PubMed PubMed Central Google Scholar
Jiang Z, Dalby PA. Challenges in scaling up AAV-based gene therapy manufacturing. Trends Biotechnol. 2023;41:1268–81.
Article CAS PubMed Google Scholar
Werle AK, Powers TW, Zobel JF, Wappelhorst CN, Jarrold MF, Lyktey NA, et al. Comparison of analytical techniques to quantitate the capsid content of adeno-associated viral vectors. Mol Ther Methods Clin Dev. 2021;23:254–2.
Article CAS PubMed PubMed Central Google Scholar
Wu D, Hwang P, Li T, Piszczek G. Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry. Gene Ther 2021. 2022;29:691–7.
Hiemenz C, Baumeister N, Helbig C, Hawe A, Babutzka S, Michalakis S, et al. Genome length determination in adeno-associated virus vectors with mass photometry. Mol Ther Methods Clin Dev. 2023;31:101162.
Article CAS PubMed PubMed Central Google Scholar
Gimpel AL, Katsikis G, Sha S, Maloney AJ, Hong MS, Nguyen TNT, et al. Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies. Mol Ther Methods Clin Dev. 2021;20:740–54.
Article CAS PubMed PubMed Central Google Scholar
Yarawsky AE, Zai-Rose V, Cunningham HM, Burgner JW, DeLion MT, Paul LN. AAV analysis by sedimentation velocity analytical ultracentrifugation: beyond empty and full capsids. Eur Biophysics J. 2023;52:353–66.
Barnes LF, Draper BE, Chen YT, Powers TW, Jarrold MF. Quantitative analysis of genome packaging in recombinant AAV vectors by charge detection mass spectrometry. Mol Ther Methods Clin Dev. 2021;23:87–97.
Article CAS PubMed PubMed Central Google Scholar
Ebberink EHTM, Ruisinger A, Nuebel M, Thomann M, Heck AJR. Assessing production variability in empty and filled adeno-associated viruses by single molecule mass analyses. Mol Ther Methods Clin Dev. 2022;27:491–501.
Article CAS PubMed PubMed Central Google Scholar
Nakatsuka R, Yamaguchi Y, Hirohata K, Shimojo S, Murakami M, Rocafort MAV, et al. Multimass Analysis of Adeno-Associated Virus Vectors by Orbitrap-Based Charge Detection Mass Spectrometry. Anal Chem. 2024;96:17037–046.
Article CAS PubMed PubMed Central Google Scholar
Hutanu A, Boelsterli D, Schmidli C, Montealegre C, Dang Thai MHN, Bobaly B, et al. Stronger together: Analytical techniques for recombinant adeno associated virus. Electrophoresis. 2021;43:1107.
Article PubMed PubMed Central Google Scholar
Kaddis CS, Lomeli SH, Yin S, Berhane B, Apostol BT, Loo JA. Sizing Large Proteins and Protein Complexes by Electrospray Ionization Mass Spectrometry and Ion Mobility. J Am Soc Mass Spectrom. 2007;18:1206–16.
Article CAS PubMed PubMed Central Google Scholar
Martin-González N, Guérin SM, Durana A, López-Marijuan I, de Pablo PJ, Guérin DMA, et al. Exploring the role of genome and structural ions in preventing viral capsid collapse during dehydration. J Phys: Condens Matter. 2018;30:104001.
PubMed PubMed Central Google Scholar
Weiss VU, Bereszcazk JZ, Havlik M, Kallinger P, Gösler I, Kumar M, et al. Analysis of a Common Cold Virus and Its Subviral Particles by Gas-Phase Electrophoretic Mobility Molecular Analysis and Native Mass Spectrometry. Anal Chem. 2015;87:8709–17.
Article CAS PubMed PubMed Central Google Scholar
Kaufman SL, Skogen JW, Dorman FD, Zarrin F, Lewis KC. Macromolecule analysis based on electrophoretic mobility in air: globular proteins. Anal Chem. 1996;68:1895–904.
Article CAS PubMed Google Scholar
Guha S, Li M, Tarlov MJ, Zachariah MR. Electrospray-differential mobility analysis of bionanoparticles. Trends Biotechnol. 2012;30:291–300.
Article CAS PubMed Google Scholar
Kallinger P, Szymanski WW. Experimental determination of the steady-state charging probabilities and particle size conservation in non-radioactive and radioactive bipolar aerosol chargers in the size range of 5–40 nm. J Nanopart Res 2015;17:1–12.
Fernandez De La Mora J. High-Resolution Mobility Analysis of Charge-Reduced Electrosprayed Protein Ions. Anal Chem. 2015;87:3729–35.
Article CAS PubMed Google Scholar
Knutson EO, Whitby KT. Aerosol classification by electric mobility: apparatus, theory, and applications. J Aerosol Sci. 1975;6:443–51.
Comments (0)