Therapeutic homology-independent targeted integration in retina and liver

Therapeutic homology-independent targeted integration in retina and liver

Limits of AAV-mediated gene therapy include targeting dominant mutations and inducing long-term transgene expression. Here, the authors show that AAV-HITI results in efficient allele-independent integration of a donor DNA in both retina and liver providing therapeutic benefit in mouse models of eith...

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Bibliographic Details
Main Authors: Patrizia Tornabene, Rita Ferla, Manel Llado-Santaeularia, Miriam Centrulo, Margherita Dell’Anno, Federica Esposito, Elena Marrocco, Emanuela Pone, Renato Minopoli, Carolina Iodice, Edoardo Nusco, Settimio Rossi, Hristiana Lyubenova, Anna Manfredi, Lucio Di Filippo, Antonella Iuliano, Annalaura Torella, Giulio Piluso, Francesco Musacchia, Enrico Maria Surace, Davide Cacchiarelli, Vincenzo Nigro, Alberto Auricchio
Format: Article
Language:English
Published: Nature Portfolio 2022-04-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-022-29550-8

Internet

https://doi.org/10.1038/s41467-022-29550-8

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