Intrathymic AAV delivery results in therapeutic site-specific integration at TCR loci in mice

Adeno-associated virus (AAV) vectors have been successfully exploited in gene therapy applications for the treatment of several genetic disorders. AAV is considered an episomal vector, but it has been shown to integrate within the host cell genome after the generation of double-strand DNA breaks or...

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Detalles Bibliográficos
Autores principales: Calabria, Andrea, Cipriani, Carlo, Spinozzi, Giulio, Rudilosso, Laura, Esposito, Simona, Benedicenti, Fabrizio, Albertini, Alessandra, Pouzolles, Marie, Luoni, Mirko, Giannelli, Serena, Broccoli, Vania, Guilbaud, Mickael, Adjali, Oumeya, Taylor, Naomi, Zimmermann, Valérie S., Montini, Eugenio, Cesana, Daniela
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The American Society of Hematology 2023
Materias:
Gene Therapy
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10356579/
https://www.ncbi.nlm.nih.gov/pubmed/36790505
http://dx.doi.org/10.1182/blood.2022017378

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10356579/
https://www.ncbi.nlm.nih.gov/pubmed/36790505
http://dx.doi.org/10.1182/blood.2022017378

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