Rossi, Settimio

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

Maguire, A. M., Simonelli, F., Pierce, E. A., Pugh, E. N., Mingozzi, F., Bennicelli, J., Banfi, S., Marshall, K. A., Testa, F., Surace, E. M., et al. (2008) · New England Journal of Medicine

Retinal Development and DisordersVirus-based gene therapy research

Cited by 2,145

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

Maguire, A. M., High, K. A., Auricchio, A., Wright, J. F., Pierce, E. A., Testa, F., Mingozzi, F., Bennicelli, J. L., Ying, G.-S., Rossi, S., et al. (2009) · The Lancet

Retinal Development and DisordersVirus-based gene therapy research

Cited by 865

Effective delivery of large genes to the retina by dual AAV vectors

Trapani, I., Colella, P., Sommella, A., Iodice, C., Cesi, G., de Simone, S., Marrocco, E., Rossi, S., Giunti, M., Palfi, A., et al. (2014) · EMBO Molecular Medicine

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 269

Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina

Tornabene, P., Trapani, I., Minopoli, R., Centrulo, M., Lupo, M., de Simone, S., Tiberi, P., Dell’Aquila, F., Marrocco, E., Iodice, C., et al. (2019) · Science Translational Medicine

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 192

Triple Vectors Expand AAV Transfer Capacity in the Retina

Maddalena, A., Tornabene, P., Tiberi, P., Minopoli, R., Manfredi, A., Mutarelli, M., Rossi, S., Simonelli, F., Naggert, J. K., Cacchiarelli, D., et al. (2018) · Molecular Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 128

Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors

Colella, P., Trapani, I., Cesi, G., Sommella, A., Manfredi, A., Puppo, A., Iodice, C., Rossi, S., Simonelli, F., Giunti, M., et al. (2014) · Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 103

AAV-mediated photoreceptor transduction of the pig cone-enriched retina

Mussolino, C., della Corte, M., Rossi, S., Viola, F., Di Vicino, U., Marrocco, E., Neglia, S., Doria, M., Testa, F., Giovannoni, R., et al. (2011) · Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 102

Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease

Trapani, I., Toriello, E., de Simone, S., Colella, P., Iodice, C., Polishchuk, E. V., Sommella, A., Colecchi, L., Rossi, S., Simonelli, F., et al. (2015) · Human Molecular Genetics

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 94

MicroRNA-Restricted Transgene Expression in the Retina

Karali, M., Manfredi, A., Puppo, A., Marrocco, E., Gargiulo, A., Allocca, M., Corte, M. D., Rossi, S., Giunti, M., Bacci, M. L., et al. (2011) · PLoS ONE

Retinal Development and DisordersVirus-based gene therapy research

Cited by 60

Attenuation of High Glucose-Induced Damage in RPE Cells through p38 MAPK Signaling Pathway Inhibition

Maugeri, G., Bucolo, C., Drago, F., Rossi, S., Di Rosa, M., Imbesi, R., D’Agata, V., & Giunta, S. (2021) · Frontiers in Pharmacology

Retinal Diseases and TreatmentsAdvanced Glycation End Products research

Cited by 47

Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models

Ferla, R., Pugni, E., Lupo, M., Tiberi, P., Fioretto, F., Perota, A., Duchi, R., Lagutina, I., Gesualdo, C., Rossi, S., et al. (2025) · Science Advances

Retinal Development and DisordersVirus-based gene therapy research

Cited by 19