Simonelli, Francesca

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

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

Russell, S., Bennett, J., Wellman, J. A., Chung, D. C., Yu, Z.-F., Tillman, A., Wittes, J., Pappas, J., Elci, O., McCague, S., et al. (2017) · The Lancet

Retinal Development and DisordersVirus-based gene therapy research

Cited by 1,952

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

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

Bennett, J., Wellman, J., Marshall, K. A., McCague, S., Ashtari, M., DiStefano-Pappas, J., Elci, O. U., Chung, D. C., Sun, J., Wright, J. F., et al. (2016) · The Lancet

Retinal Development and DisordersVirus-based gene therapy research

Cited by 461

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy

Maguire, A. M., Russell, S., Wellman, J. A., Chung, D. C., Yu, Z.-F., Tillman, A., Wittes, J., Pappas, J., Elci, O., Marshall, K. A., et al. (2019) · Ophthalmology

Retinal Development and DisordersVirus-based gene therapy research

Cited by 409

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

Bennett, J., Ashtari, M., Wellman, J., Marshall, K. A., Cyckowski, L. L., Chung, D. C., McCague, S., Pierce, E. A., Chen, Y., Bennicelli, J. L., et al. (2012) · Science Translational Medicine

Retinal Development and DisordersVirus-based gene therapy research

Cited by 395

Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease

Maguire, A. M., Russell, S., Chung, D. C., Yu, Z.-F., Tillman, A., Drack, A. V., Simonelli, F., Leroy, B. P., Reape, K. Z., High, K. A., et al. (2021) · Ophthalmology

Retinal Development and DisordersRetinal Diseases and Treatments

Cited by 194

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

Real-world safety and effectiveness of voretigene neparvovec: results up to 2 Years from the prospective, registry-based PERCEIVE study

Fischer, M. D., Simonelli, F., Sahni, J., Holz, F. G., Maier, R., Fasser, C., Suhner, A., Stiehl, D. P., Chen, B., Audo, I., et al. (2024) · Biomolecules

Retinal Development and DisordersVirus-based gene therapy research

Cited by 70

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

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

Cited by 25

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