Ligand-modified rAAV6 vectors with nanoblades allow high-level gene knockin in HSPCs without compromising cell survival

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Authors: Alejandra Gutierrez-Guerrero, Séverine Périan, Aurélien Leray, Chiara Martinello, Maria Jimena Abrey Recalde, Caroline Costa, Cecilia Iglesias Herrero, Mohammed Bouzelha, Dimitri Alvarez-Dorta, Sébastien G. Gouin, Eduard Ayuso, Oumeya Adjali, Hildegard Büning, David Deniaud, Mathieu Mével, Els Verhoeyen

Journal title: Molecular Therapy Nucleic Acids

Journal number: 36

Journal publisher: Elsevier BV

Published year: 2025

DOI identifier: 10.1016/J.OMTN.2025.102495

ISSN: 2162-2531