Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells

Long-range gene editing by homology-directed repair (HDR) in hematopoietic stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno-associated viral vector (AAV) for template delivery. Here, we uncover unexpected load and prolonged persistence of AAV genomes and their...

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Detalles Bibliográficos
Autores principales: Ferrari, Samuele, Jacob, Aurelien, Cesana, Daniela, Laugel, Marianne, Beretta, Stefano, Varesi, Angelica, Unali, Giulia, Conti, Anastasia, Canarutto, Daniele, Albano, Luisa, Calabria, Andrea, Vavassori, Valentina, Cipriani, Carlo, Castiello, Maria Carmina, Esposito, Simona, Brombin, Chiara, Cugnata, Federica, Adjali, Oumeya, Ayuso, Eduard, Merelli, Ivan, Villa, Anna, Di Micco, Raffaella, Kajaste-Rudnitski, Anna, Montini, Eugenio, Penaud-Budloo, Magalie, Naldini, Luigi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cell Press 2022
Materias:
Clinical and Translational Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9550218/
https://www.ncbi.nlm.nih.gov/pubmed/36206730
http://dx.doi.org/10.1016/j.stem.2022.09.001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9550218/
https://www.ncbi.nlm.nih.gov/pubmed/36206730
http://dx.doi.org/10.1016/j.stem.2022.09.001

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