20.500.12556/DiRROS-30382 It’s what’s on the inside that counts: capsid content as a critical quality attribute for AAV characterization Recombinant adeno-associated virus (rAAV) vectors exhibit complex heterogeneity in capsid content. Although a growing array of analytical platforms can interrogate different capsid attributes, no single method provides a comprehensive or unequivocal assessment. Critically, current approaches often fail to evaluate genome integrity, an emerging important quality attribute. Incomplete genomes can contribute to viral genome titers without conferring therapeutic benefit, while simultaneously increasing immunogenic burden and risk of adverse events. Discordant results from orthogonal assays further complicate interpretation and cross-laboratory comparisons. Here, we highlight the limitations of existing characterization strategies and advocate for robust analytical frameworks that directly assess genome completeness. We propose best-practice guidelines, including improved genome integrity assays and harmonized reporting, to better link capsid content analytics with potency and patient safety in gene therapy development. gene therapy AAV genomic integrity capsid content  genska terapija AAV integriteta genoma vsebnost kapsid true false false Angleški jezik Slovenski jezik Neznano 2026-06-23 15:53:58 2026-06-23 15:53:58 2026-06-24 03:46:10 0000-00-00 00:00:00 2026 0 0 Nasl. z nasl. zaslona; Opis vira z dne 23. 6. 2026; Soavtorji: Stuart Nelson, Ana Dolinar Češarek, David Dobnik; str. 1-5 issue 3, [article no.] 201765 Vol. 34 2026 0000-00-00 Zaloznikova NiDoloceno NiDoloceno 0000-00-00 0000-00-00 2026-01-01 577.2 3117-387X 10.1016/j.omta.2026.201765 282615555 https://www.cell.com/molecular-therapy-family/advances/fulltext/S3117-387X(26)00100-X 1 fc5e69b3-6f0a-11f1-98c5-001a4af901a5 https://dirros.openscience.si/Dokument.php?lang=slv&id=46108 RAZ_Earley_Lauriel_F._2026.pdf RAZ_Earley_Lauriel_F._2026.pdf 1 F9DAF9DDE3C6C7BC41B4CEB8279311E0 7220737a920ed894d15bf6b3370bc69807cd8ebba6423456943e7b247d32adc0 061322a4-6f0b-11f1-98c5-001a4af901a5 https://dirros.openscience.si/Dokument.php?lang=slv&id=46110 Nacionalni inštitut za biologijo 0 0 0