Maguire, Albert 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

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

Gene therapy restores vision in a canine model of childhood blindness

Acland, G. M., Aguirre, G. D., Ray, J., Zhang, Q., Aleman, T. S., Cideciyan, A. V., Pearce-Kelling, S. E., Anand, V., Zeng, Y., Maguire, A. M., et al. (2001) · Nature Genetics

Retinal Development and DisordersVirus-based gene therapy research

Cited by 1,202

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

Genetic variants near TIMP3 and high-density lipoprotein–associated loci influence susceptibility to age-related macular degeneration

Chen, W., Stambolian, D., Edwards, A. O., Branham, K. E., Othman, M., Jakobsdottir, J., Tosakulwong, N., Pericak-Vance, M. A., Campochiaro, P. A., Klein, M. L., et al. (2010) · Proceedings of the National Academy of Sciences

Retinal Diseases and TreatmentsGenetic Associations and Epidemiology

Cited by 506

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

Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy

Bennett, J., Tanabe, T., Sun, D., Zeng, Y., Kjeldbye, H., Gouras, P., & Maguire, A. M. (1996) · Nature Medicine

Retinal Development and DisordersVirus-based gene therapy research

Cited by 316

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

Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer

Bennicelli, J., Wright, J. F., Komaromy, A., Jacobs, J. B., Hauck, B., Zelenaia, O., Mingozzi, F., Hui, D., Chung, D., Rex, T. S., et al. (2008) · Molecular Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 267

Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey

Vandenberghe, L. H., Bell, P., Maguire, A. M., Cearley, C. N., Xiao, R., Calcedo, R., Wang, L., Castle, M. J., Maguire, A. C., Grant, R., et al. (2011) · Science Translational Medicine

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 242

Gene Editing for CEP290 -Associated Retinal Degeneration

Pierce, E. A., Aleman, T. S., Jayasundera, K. T., Ashimatey, B. S., Kim, K., Rashid, A., Jaskolka, M. C., Myers, R. L., Lam, B. L., Bailey, S. T., et al. (2024) · New England Journal of Medicine

Retinal Development and DisordersRetinal Diseases and Treatments

Cited by 237

Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection

Jacobson, S. G., Acland, G. M., Aguirre, G. D., Aleman, T. S., Schwartz, S. B., Cideciyan, A. V., Zeiss, C. J., Komaromy, A. M., Kaushal, S., Roman, A. J., et al. (2006) · Molecular Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 224

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

Persistent transgene product in retina, optic nerve and brain after intraocular injection of rAAV

Dudus, L., Anand, V., Acland, G. M., Chen, S.-J., Wilson, J. M., Fisher, K. J., Maguire, A. M., & Bennett, J. (1999) · Vision Research

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 160

Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-Human Primate Retina

Ramachandran, P. S., Lee, V., Wei, Z., Song, J. Y., Casal, G., Cronin, T., Willett, K., Huckfeldt, R., Morgan, J. I. W., Aleman, T. S., et al. (2017) · Human Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 147

Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness

Amado, D., Mingozzi, F., Hui, D., Bennicelli, J. L., Wei, Z., Chen, Y., Bote, E., Grant, R. L., Golden, J. A., Narfstrom, K., et al. (2010) · Science Translational Medicine

Retinal Development and DisordersVirus-based gene therapy research

Cited by 140

AAV9 targets cone photoreceptors in the nonhuman primate retina

Vandenberghe, L. H., Bell, P., Maguire, A. M., Xiao, R., Hopkins, T. B., Grant, R., Bennett, J., & Wilson, J. M. (2013) · PLoS ONE

Retinal Development and DisordersVirus-based gene therapy research

Cited by 97

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

A Deviant Immune Response to Viral Proteins and Transgene Product Is Generated on Subretinal Administration of Adenovirus and Adeno-associated Virus

Anand, V., Duffy, B., Yang, Z., Dejneka, N. S., Maguire, A. M., & Bennett, J. (2002) · Molecular Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 69

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

Safety of same-eye subretinal sequential readministration of AAV2-hRPE65v2 in non-human primates

Weed, L., Ammar, M. J., Zhou, S., Wei, Z., Serrano, L. W., Sun, J., Lee, V., Maguire, A. M., Bennett, J., & Aleman, T. S. (2019) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 27

Correction: AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina

Vandenberghe, L. H., Bell, P., Maguire, A. M., Xiao, R., Hopkins, T. B., Grant, R., Bennett, J., & Wilson, J. M. (2013) · PLoS ONE

Retinal Development and DisordersVirus-based gene therapy research

Cited by 16