Auricchio, Alberto

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

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

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

Axonal degeneration in paraplegin-deficient mice is associated with abnormal mitochondria and impairment of axonal transport

Ferreirinha, F., Quattrini, A., Pirozzi, M., Valsecchi, V., Dina, G., Broccoli, V., Auricchio, A., Piemonte, F., Tozzi, G., Gaeta, L., et al. (2004) · Journal of Clinical Investigation

Hereditary Neurological DisordersMitochondrial Function and Pathology

Cited by 308

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice

Allocca, M., Doria, M., Petrillo, M., Colella, P., Garcia-Hoyos, M., Gibbs, D., Kim, S. R., Maguire, A., Rex, T. S., Di Vicino, U., et al. (2008) · Journal of Clinical Investigation

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 292

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

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

Cited by 192

Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material

Lock, M., McGorray, S., Auricchio, A., Ayuso, E., Beecham, E. J., Blouin-Tavel, V., Bosch, F., Bose, M., Byrne, B. J., Caton, T., et al. (2010) · Human Gene Therapy

Virus-based gene therapy researchViral Infectious Diseases and Gene Expression in Insects

Cited by 131

Novel adeno-associated viral vectors for retinal gene therapy.

Vandenberghe, L. H., & Auricchio, A. (2012) · Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 131

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

Seeing the light after 25 Years of retinal gene therapy

Trapani, I., & Auricchio, A. (2018) · Trends in Molecular Medicine

Retinal Development and DisordersVirus-based gene therapy research

Cited by 124

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

Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy

Reich, S. J., Auricchio, A., Hildinger, M., Glover, E., Maguire, A. M., Wilson, J. M., & Bennett, J. (2003) · Human Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 96

Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material

Ayuso, E., Blouin, V., Lock, M., McGorray, S., Leon, X., Alvira, M. R., Auricchio, A., Bucher, S., Chtarto, A., Clark, K. R., et al. (2014) · Human Gene Therapy

Virus-based gene therapy researchViral Infectious Diseases and Gene Expression in Insects

Cited by 94

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

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

Cited by 46

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

Myosin7a Deficiency Results in Reduced Retinal Activity Which Is Improved by Gene Therapy

Colella, P., Sommella, A., Marrocco, E., Di Vicino, U., Polishchuk, E., Garrido, M. G., Seeliger, M. W., Polishchuk, R., & Auricchio, A. (2013) · PLoS ONE

Retinal Development and DisordersVirus-based gene therapy research

Cited by 34

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