Stone, Edwin 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

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

Mutation of a nuclear receptor gene, NR2E3, causes enhanced S cone syndrome, a disorder of retinal cell fate

Haider, N. B., Jacobson, S. G., Cideciyan, A. V., Swiderski, R., Streb, L. M., Searby, C., Beck, G., Hockey, R., Hanna, D. B., Gorman, S., et al. (2000) · Nature Genetics

Retinal Development and DisordersRetinal Diseases and Treatments

Cited by 507

Missense Variations in the Fibulin 5 Gene and Age-Related Macular Degeneration

Stone, E. M., Braun, T. A., Russell, S. R., Kuehn, M. H., Lotery, A. J., Moore, P. A., Eastman, C. G., Casavant, T. L., & Sheffield, V. C. (2004) · New England Journal of Medicine

Retinal Diseases and TreatmentsRetinal Development and Disorders

Cited by 324

Peroxisome biogenesis disorders in the Zellweger spectrum: An overview of current diagnosis, clinical manifestations, and treatment guidelines

Braverman, N. E., Raymond, G. V., Rizzo, W. B., Moser, A. B., Wilkinson, M. E., Stone, E. M., Steinberg, S. J., Wangler, M. F., Rush, E. T., Hacia, J. G., et al. (2016) · Molecular Genetics and Metabolism

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 281

Rapid restoration of visual pigment and function with oral retinoid in a mouse model of childhood blindness

Van Hooser, J. P., Aleman, T. S., He, Y.-G., Cideciyan, A. V., Kuksa, V., Pittler, S. J., Stone, E. M., Jacobson, S. G., & Palczewski, K. (2000) · Proceedings of the National Academy of Sciences

Retinal Development and DisordersRetinoids in leukemia and cellular processes

Cited by 252

Aflibercept Therapy for Exudative Age-related Macular Degeneration Resistant to Bevacizumab and Ranibizumab

Bakall, B., Folk, J. C., Boldt, H. C., Sohn, E. H., Stone, E. M., Russell, S. R., & Mahajan, V. B. (2013) · American Journal of Ophthalmology

Retinal Diseases and TreatmentsRetinal and Macular Surgery

Cited by 229

ABCA4 disease progression and a proposed strategy for gene therapy

Cideciyan, A. V., Swider, M., Aleman, T. S., Tsybovsky, Y., Schwartz, S. B., Windsor, E. A. M., Roman, A. J., Sumaroka, A., Steinberg, J. D., Jacobson, S. G., et al. (2009) · Human Molecular Genetics

Retinal Development and DisordersRetinal Diseases and Treatments

Cited by 186

Association between the SERPING1 gene and age-related macular degeneration: a two-stage case–control study

Ennis, S., Jomary, C., Mullins, R., Cree, A., Chen, X., MacLeod, A., Jones, S., Collins, A., Stone, E., & Lotery, A. (2008) · The Lancet

Retinal Diseases and TreatmentsGenetic Associations and Epidemiology

Cited by 171

Retinal Laminar Architecture in Human Retinitis Pigmentosa Caused by Rhodopsin Gene Mutations

Aleman, T. S., Cideciyan, A. V., Sumaroka, A., Windsor, E. A. M., Herrera, W., White, D. A., Kaushal, S., Naidu, A., Roman, A. J., Schwartz, S. B., et al. (2008) · Investigative Opthalmology & Visual Science

Retinal Development and DisordersRetinal Diseases and Treatments

Cited by 133

Defining the Residual Vision in Leber Congenital Amaurosis Caused by RPE65 Mutations

Jacobson, S. G., Aleman, T. S., Cideciyan, A. V., Roman, A. J., Sumaroka, A., Windsor, E. A. M., Schwartz, S. B., Heon, E., & Stone, E. M. (2009) · Investigative Opthalmology & Visual Science

Retinal Development and DisordersRetinal Diseases and Treatments

Cited by 121

Complement component C5a activates ICAM-1 expression on human choroidal endothelial cells

Skeie, J. M., Fingert, J. H., Russell, S. R., Stone, E. M., & Mullins, R. F. (2010) · Investigative Opthalmology & Visual Science

Retinal Diseases and TreatmentsComplement system in diseases

Cited by 114

Adeno-Associated Virus Type 5: Transduction Efficiency and Cell-Type Specificity in the Primate Retina

Lotery, A. J., Yang, G. S., Mullins, R. F., Russell, S. R., Schmidt, M., Stone, E. M., Lindbloom, J. D., Chiorini, J. A., Kotin, R. M., & Davidson, B. L. (2003) · Human Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 107

Molecular genetics of age-related macular degeneration

Stone, E. M. (2001) · Human Molecular Genetics

Retinal Diseases and TreatmentsRetinal Development and Disorders

Cited by 79

Retinal tropism and transduction of adeno-associated virus varies by serotype and route of delivery (intravitreal, subretinal, or suprachoroidal) in rats

Han, I. C., Cheng, J. L., Burnight, E. R., Ralston, C. L., Fick, J. L., Thomsen, G. J., Tovar, E. F., Russell, S. R., Sohn, E. H., Mullins, R. F., et al. (2020) · Human Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 60

Molecular response of chorioretinal endothelial cells to complement injury: Implications for macular degeneration

Zeng, S., Whitmore, S. S., Sohn, E. H., Riker, M. J., Wiley, L. A., Scheetz, T. E., Stone, E. M., Tucker, B. A., & Mullins, R. F. (2016) · The Journal of Pathology

Retinal Diseases and TreatmentsComplement system in diseases

Cited by 56

Light aversion in mice depends on nonimage-forming irradiance detection

Thompson, S., Recober, A., Vogel, T. W., Kuburas, A., Owens, J. A., Sheffield, V. C., Russo, A. F., & Stone, E. M. (2010) · Behavioral Neuroscience

Circadian rhythm and melatoninRetinal Development and Disorders

Cited by 37

The degree of adeno-associated virus-induced retinal inflammation varies based on serotype and route of delivery: intravitreal, subretinal, or suprachoroidal

Wiley, L. A., Boyce, T. M., Meyering, E. E., Ochoa, D., Sheehan, K. M., Stone, E. M., Mullins, R. F., Tucker, B. A., & Han, I. C. (2023) · Human Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 30

Helper-dependent adenovirus transduces the human and rat retina but elicits an inflammatory reaction when delivered subretinally in rats

Han, I. C., Burnight, E. R., Ulferts, M. J., Worthington, K. S., Russell, S. R., Sohn, E. H., Mullins, R. F., Stone, E. M., Tucker, B. A., & Wiley, L. A. (2019) · Human Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 30