Alberto Auricchio, MD PhD
Affiliations: | Telethon Institute of Genetics and Medicine, Italy |
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"Alberto Auricchio"
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Publications
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| Dell'Aquila F, Di Cunto R, Marrocco E, et al. (2024) Combined intraocular and intravenous gene delivery for therapy of gyrate atrophy of the choroid and retina. Molecular Therapy : the Journal of the American Society of Gene Therapy |
| Testa F, Carreño E, van den Born LI, et al. (2024) Multicentric Longitudinal Prospective Study in a European Cohort of MYO7A Patients: Disease Course and Implications for Gene Therapy. Investigative Ophthalmology & Visual Science. 65: 25 |
| Bueren JA, Auricchio A. (2023) Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases. Human Gene Therapy. 34: 763-775 |
| Ferla R, Dell'Aquila F, Doria M, et al. (2023) Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B. Molecular Therapy. Methods & Clinical Development. 28: 396-411 |
| Boffa I, Polishchuk E, De Stefano L, et al. (2023) Liver-directed gene therapy for ornithine aminotransferase deficiency. Embo Molecular Medicine. e17033 |
| Brunetti-Pierri N, Ferla R, Ginocchio VM, et al. (2022) Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI. Nejm Evidence. 1: EVIDoa2200052 |
| Padula A, Petruzzelli R, Philbert SA, et al. (2022) Full-length ATP7B reconstituted through protein -splicing corrects Wilson disease in mice. Molecular Therapy. Methods & Clinical Development. 26: 495-504 |
| Tornabene P, Ferla R, Llado-Santaeularia M, et al. (2022) Therapeutic homology-independent targeted integration in retina and liver. Nature Communications. 13: 1963 |
| Tornabene P, Trapani I, Centrulo M, et al. (2021) Inclusion of a degron reduces levelsof undesired inteins after AAV-mediated proteinsplicing in the retina. Molecular Therapy. Methods & Clinical Development. 23: 448-459 |
| Patrizi C, Llado M, Benati D, et al. (2021) Allele-specific editing ameliorates dominant retinitis pigmentosa in a transgenic mouse model. American Journal of Human Genetics. 108: 295-308 |