Nature Communications (Apr 2022)
Therapeutic homology-independent targeted integration in retina and liver
- 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
Affiliations
- Patrizia Tornabene
- Telethon Institute of Genetics and Medicine (TIGEM)
- Rita Ferla
- Telethon Institute of Genetics and Medicine (TIGEM)
- Manel Llado-Santaeularia
- Telethon Institute of Genetics and Medicine (TIGEM)
- Miriam Centrulo
- Telethon Institute of Genetics and Medicine (TIGEM)
- Margherita Dell’Anno
- Telethon Institute of Genetics and Medicine (TIGEM)
- Federica Esposito
- Telethon Institute of Genetics and Medicine (TIGEM)
- Elena Marrocco
- Telethon Institute of Genetics and Medicine (TIGEM)
- Emanuela Pone
- Telethon Institute of Genetics and Medicine (TIGEM)
- Renato Minopoli
- Telethon Institute of Genetics and Medicine (TIGEM)
- Carolina Iodice
- Telethon Institute of Genetics and Medicine (TIGEM)
- Edoardo Nusco
- Telethon Institute of Genetics and Medicine (TIGEM)
- Settimio Rossi
- Eye Clinic, Multidisciplinary Department of Medical, Surgical and Dental Sciences, University of Campania L. Vanvitelli
- Hristiana Lyubenova
- Telethon Institute of Genetics and Medicine (TIGEM)
- Anna Manfredi
- Telethon Institute of Genetics and Medicine (TIGEM), Armenise/Harvard Laboratory of Integrative Genomics
- Lucio Di Filippo
- Next Generation Diagnostic Srl
- Antonella Iuliano
- Telethon Institute of Genetics and Medicine (TIGEM)
- Annalaura Torella
- Telethon Institute of Genetics and Medicine (TIGEM)
- Giulio Piluso
- Department of Precision Medicine, University of Campania L. Vanvitelli
- Francesco Musacchia
- Telethon Institute of Genetics and Medicine (TIGEM)
- Enrico Maria Surace
- Medical Genetics, Department of Translational Medicine, Federico II University
- Davide Cacchiarelli
- Telethon Institute of Genetics and Medicine (TIGEM), Armenise/Harvard Laboratory of Integrative Genomics
- Vincenzo Nigro
- Telethon Institute of Genetics and Medicine (TIGEM)
- Alberto Auricchio
- Telethon Institute of Genetics and Medicine (TIGEM)
- DOI
- https://doi.org/10.1038/s41467-022-29550-8
- Journal volume & issue
-
Vol. 13,
no. 1
pp. 1 – 14
Abstract
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 either a genetic form of blindness or a lysosomal storage disease, respectively.
