| Allele-specific editing ameliorates dominant retinitis pigmentosa in a transgenic mouse model C Patrizi, M Llado, D Benati, C Iodice, E Marrocco, R Guarascio, ... The American Journal of Human Genetics 108 (2), 295-308, 2021 | 37 | 2021 |
| Therapeutic homology-independent targeted integration in retina and liver P Tornabene, R Ferla, M Llado-Santaeularia, M Centrulo, M Dell’Anno, ... Nature communications 13 (1), 1963, 2022 | 22 | 2022 |
| Specific knock-down of P347S dominant mutation in rhodopsin gene by CRISPR/Cas9 system C Patrizi, M Llado, D Benati, R Guarascio, M Cheetham, A Auricchio, ... ASGCT, 2020 | 1 | 2020 |
| Homology-independent targeted integration in photoreceptors ML Santaeularia, F Esposito, C Iodice, E Marrocco, A Auricchio Investigative Ophthalmology & Visual Science 60 (9), 4228-4228, 2019 | 1 | 2019 |
| AAV-mediated homology-independent targeted integration leads to sustained secretion of therapeutic proteins from new-born liver F Esposito, F Dell'Aquila, R Ferla, PS Horrach, S Auricchio, M Dell'Anno, ... HUMAN GENE THERAPY 33 (23-24), A11-A11, 2022 | | 2022 |
| Homology Independent Targeted Integration Leads to Highly Efficient Protein Expression and Secretion From Liver F Esposito, FD Aquila, R Ferla, M Llado, M Dell'Anno, PS Horrach, ... MOLECULAR THERAPY 30 (4), 218-218, 2022 | | 2022 |
| Genome editing methods and constructs A Auricchio, ML SANTAEULARIA US Patent App. 17/285,755, 2022 | | 2022 |
| AAV-mediated liver-directed homology-independent targeted integration provides stable therapeutic levels of transgene expression in newborn mice with a lysosomal storage disease. R Ferla, M Llado-Santaeularia, M Dell'Anno, F Esposito, E Pone, ... HUMAN GENE THERAPY 32 (19-20), A64-A65, 2021 | | 2021 |
| Therapeutic AAV-mediated homology-independent targeted integration in the retina P Tornabene, M Llado-Santaeularia, M Centrulo, E Marrocco, C Iodice, ... HUMAN GENE THERAPY 32 (19-20), A24-A24, 2021 | | 2021 |
| Clarissa Patrizi, Manel Llado, 2 Daniela Benati, Carolina Iodice, 2 Elena Marrocco, 2 Rosellina Guarascio, 3 EM Surace, ME Cheetham, A Auricchio, A Recchia | | 2021 |
| AAV-Mediated Homology-Independent Targeted Integration in Newborn Hepatocytes Corrects Mucopolysaccharidosis Type VI ML Santaeularia, F Esposito, M Dell'Anno, R Minopoli, H Lyubenova, ... MOLECULAR THERAPY 28 (4), 418-418, 2020 | | 2020 |
| THERAPEUTIC GENOME EDITING IN RETINA AND LIVER M LLADO SANTAEULARIA Università degli Studi di Milano, 2020 | | 2020 |
| AAV-mediated protein trans-splicing and genome editing for therapy of Haemophilia A H Lyubenova, R Minopoli, M Llado, E Nusco, A Auricchio HUMAN GENE THERAPY 30 (11), A83-A83, 2019 | | 2019 |
| Homology-independent targeted integration to counteract toxic gain-of-functions and loss of transgene expression due to cell proliferation M Llado, F Esposito, C Iodice, R Minopoli, H Lyubenova, E Nusco, ... HUMAN GENE THERAPY 30 (11), A17-A17, 2019 | | 2019 |
| Neuroprotection of photoreceptors bearing the P23H mutation in Rhodopsin V Marigo, A Comitato, C La Marca, D Schiroli, P Subramanian, M Llado, ... Investigative Ophthalmology & Visual Science 59 (9), 2488-2488, 2018 | | 2018 |
| Homology Independent Targeted Integration for Gene Correction in Photoreceptors ML Santaeularia, A Auricchio, C Iodice MOLECULAR THERAPY 26 (5), 261-261, 2018 | | 2018 |
| CRISPR/Cas9-mediated specific knock-down of dominant mutations in Rhodopsin gene D Benati, M Llado, C Patrizi, D Schiroli, V Marigo, A Auricchio, A Recchia -, 2017 | | 2017 |
