Publications
Rita Ferla, Fabio Dell’Aquila et al., Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B.
Molecular Therapy: Methods & Clinical Development, March 2023
https://doi.org/10.1016/j.omtm.2023.02.002
Patrizia Tornabene et al., Inclusion of a degron reduces levels of undesired inteins after AAV-mediated protein trans-splicing in the retina.
Molecular Therapy: Methods & Clinical Development, December 2021
https://doi.org/10.1016/j.omtm.2021.10.004
Patrizia Tornabene, Ivana Trapani et al., Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina.
Science Translational Medicine, May 2019.
https://doi.org/10.1126/scitranslmed.aav4523
Pasqualina Colella et al., Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors.
Gene Therapy, April 2014.
https://doi.org/10.1038/gt.2014.8
Ivana Trapani, Pasqualina Colella et al., Effective delivery of large genes to the retina by dual AAV vectors.
Embo Molecular Medicine, February 2014
https://doi.org/10.1002/emmm.201302948