Therapeutic homology-independent targeted integration in retina and liver

Challenges to the widespread application of gene therapy with adeno-associated viral (AAV) vectors include dominant conditions due to gain-of-function mutations which require allele-specific knockout, as well as long-term transgene expression from proliferating tissues, which is hampered by AAV DNA...

Descripción completa

Detalles Bibliográficos
Autores principales: Tornabene, Patrizia, Ferla, Rita, Llado-Santaeularia, Manel, Centrulo, Miriam, Dell’Anno, Margherita, Esposito, Federica, Marrocco, Elena, Pone, Emanuela, Minopoli, Renato, Iodice, Carolina, Nusco, Edoardo, Rossi, Settimio, Lyubenova, Hristiana, Manfredi, Anna, Di Filippo, Lucio, Iuliano, Antonella, Torella, Annalaura, Piluso, Giulio, Musacchia, Francesco, Surace, Enrico Maria, Cacchiarelli, Davide, Nigro, Vincenzo, Auricchio, Alberto
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9005519/
https://www.ncbi.nlm.nih.gov/pubmed/35414130
http://dx.doi.org/10.1038/s41467-022-29550-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9005519/
https://www.ncbi.nlm.nih.gov/pubmed/35414130
http://dx.doi.org/10.1038/s41467-022-29550-8

Ejemplares similares