Surace, Enrico M.

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

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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

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors

Allocca, M., Mussolino, C., Garcia-Hoyos, M., Sanges, D., Iodice, C., Petrillo, M., Vandenberghe, L. H., Wilson, J. M., Marigo, V., Surace, E. M., et al. (2007) · Journal of Virology

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 246

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

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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

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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

Delivery of Adeno-Associated Virus Vectors to the Fetal Retina: Impact of Viral Capsid Proteins on Retinal Neuronal Progenitor Transduction

Surace, E. M., Auricchio, A., Reich, S. J., Rex, T., Glover, E., Pineles, S., Tang, W., O'Connor, E., Lyubarsky, A., Savchenko, A., et al. (2003) · Journal of Virology

Virus-based gene therapy researchRetinal Development and Disorders

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Light‐responsive microRNA miR‐211 targets Ezrin to modulate lysosomal biogenesis and retinal cell clearance

Naso, F., Intartaglia, D., Falanga, D., Soldati, C., Polishchuk, E., Giamundo, G., Tiberi, P., Marrocco, E., Scudieri, P., Di Malta, C., et al. (2020) · The EMBO Journal

Retinal Development and DisordersAutophagy in Disease and Therapy

Cited by 46

Inclusion of a degron reduces levels of undesired inteins after AAV-mediated protein trans-splicing in the retina

Tornabene, P., Trapani, I., Centrulo, M., Marrocco, E., Minopoli, R., Lupo, M., Iodice, C., Gesualdo, C., Simonelli, F., Surace, E. M., et al. (2021) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRetinal Development and Disorders

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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

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