Hauswirth, William W.

Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia

Banin, E., Gootwine, E., Obolensky, A., Ezra-Elia, R., Ejzenberg, A., Zelinger, L., Honig, H., Rosov, A., Yamin, E., Sharon, D., et al. (2015) · Molecular Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 113

Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus

Lopes, V. S., Boye, S. E., Louie, C. M., Boye, S., Dyka, F., Chiodo, V, Fofo, H., Hauswirth, W. W., & Williams, D. S. (2013) · Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 112

Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach

Mowat, F. M., Gornik, K. R., Dinculescu, A., Boye, S. L., Hauswirth, W. W., Petersen-Jones, S. M., & Bartoe, J. T. (2014) · Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 108

ERK1/2 activation is a therapeutic target in age-related macular degeneration

Dridi, S., Hirano, Y., Tarallo, V., Kim, Y., Fowler, B. J., Ambati, B. K., Bogdanovich, S., Chiodo, V. A., Hauswirth, W. W., Kugel, J. F., et al. (2012) · Proceedings of the National Academy of Sciences

Retinal Diseases and TreatmentsRetinal Development and Disorders

Cited by 105

Novel anti-VEGF chimeric molecules delivered by AAV vectors for inhibition of retinal neovascularization

Pechan, P., Rubin, H., Lukason, M., Ardinger, J., DuFresne, E., Hauswirth, W. W., Wadsworth, S. C., & Scaria, A. (2009) · Gene Therapy

Angiogenesis and VEGF in CancerRetinal Development and Disorders

Cited by 104

Comparative analysis of in vivo and in vitro AAV vector transduction in the neonatal mouse retina: Effects of serotype and site of administration

Pang, J.-J., Lauramore, A., Deng, W.-T., Li, Q., Doyle, T. J., Chiodo, V., Li, J., & Hauswirth, W. W. (2008) · Vision Research

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 97

rAAV2/5 gene-targeting to rods:dose-dependent efficiency and complications associated with different promoters

Beltran, W. A., Boye, S. L., Boye, S. E., Chiodo, V. A., Lewin, A. S., Hauswirth, W. W., & Aguirre, G. D. (2010) · Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 96

Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations

Beltran, W. A., Cideciyan, A. V., Boye, S. E., Ye, G.-J., Iwabe, S., Dufour, V. L., Marinho, L. F., Swider, M., Kosyk, M. S., Sha, J., et al. (2017) · Molecular Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 82

Long-term Structural Outcomes of Late-stage RPE65 Gene Therapy

Gardiner, K. L., Cideciyan, A. V., Swider, M., Dufour, V. L., Sumaroka, A., Komáromy, A. M., Hauswirth, W. W., Iwabe, S., Jacobson, S. G., Beltran, W. A., et al. (2020) · Molecular Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 67

LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile

Koilkonda, R., Yu, H., Talla, V., Porciatti, V., Feuer, W. J., Hauswirth, W. W., Chiodo, V., Erger, K. E., Boye, S. L., Lewin, A. S., et al. (2014) · Investigative Ophthalmology & Visual Science

Retinal Development and DisordersVirus-based gene therapy research

Cited by 65

Safety and biodistribution evaluation in cynomolgus macaques of rAAV2tYF-PR1. 7-hCNGB3, a recombinant AAV vector for treatment of achromatopsia

Ye, G.-J., Budzynski, E., Sonnentag, P., Nork, T. M., Miller, P. E., Sharma, A. K., Ver Hoeve, J. N., Smith, L. M., Arndt, T., Calcedo, R., et al. (2016) · Human Gene Therapy Clinical Development

Retinal Development and DisordersVirus-based gene therapy research

Cited by 64

Ab-Externo AAV-Mediated Gene Delivery to the Suprachoroidal Space Using a 250 Micron Flexible Microcatheter

Peden, M. C., Min, J., Meyers, C., Lukowski, Z., Li, Q., Boye, S. L., Levine, M., Hauswirth, W. W., Ratnakaram, R., Dawson, W., et al. (2011) · PLoS ONE

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 63

Dual ABCA4 -AAV Vector Treatment Reduces Pathogenic Retinal A2E Accumulation in a Mouse Model of Autosomal Recessive Stargardt Disease

Dyka, F. M., Molday, L. L., Chiodo, V. A., Molday, R. S., & Hauswirth, W. W. (2019) · Human Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 56

Functional interchangeability of rod and cone transducin α-subunits

Deng, W.-T., Sakurai, K., Liu, J., Dinculescu, A., Li, J., Pang, J., Min, S.-H., Chiodo, V. A., Boye, S. L., Chang, B., et al. (2009) · Proceedings of the National Academy of Sciences

Retinal Development and DisordersReceptor Mechanisms and Signaling

Cited by 46

Reduced retinal transduction and enhanced transgene-directed immunogenicity with intravitreal delivery of rAAV following posterior vitrectomy in dogs

Boyd, R. F., Boye, S. L., Conlon, T. J., Erger, K. E., Sledge, D. G., Langohr, I. M., Hauswirth, W. W., Komáromy, A. M., Boye, S. E., Petersen-Jones, S. M., et al. (2016) · Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 37

Six Years and Counting: Restoration of Photopic Retinal Function and Visual Behavior Following Gene Augmentation Therapy in a Sheep Model of CNGA3 Achromatopsia

Ofri, R., Averbukh, E., Ezra-Elia, R., Ross, M., Honig, H., Obolensky, A., Rosov, A., Hauswirth, W. W., Gootwine, E., & Banin, E. (2018) · Human Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 31

Retinal Disease in Rpe65-Deficient Mice: Comparison to Human Leber Congenital Amaurosis Due to RPE65 Mutations

Caruso, R. C., Aleman, T. S., Cideciyan, A. V., Roman, A. J., Sumaroka, A., Mullins, C. L., Boye, S. L., Hauswirth, W. W., & Jacobson, S. G. (2010) · Investigative Opthalmology & Visual Science

Retinal Development and DisordersRetinal Diseases and Treatments

Cited by 26

Toxicology and pharmacology of an AAV vector expressing codon-optimized RPGR in RPGR-deficient Rd9 mice

Song, C., Conlon, T. J., Deng, W.-T., Coleman, K. E., Zhu, P., Plummer, C., Mandapati, S., Van Hoosear, M., Green, K. B., Sonnentag, P., et al. (2018) · Human Gene Therapy Clinical Development

Retinal Development and DisordersVirus-based gene therapy research

Cited by 20

Retina transduction by rAAV2 after intravitreal injection: Comparison between mouse and rat

Dias, M. S., Araujo, V. G., Vasconcelos, T., Li, Q., Hauswirth, W. W., Linden, R., & Petrs-Silva, H. (2019) · Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 17

Evaluation of Photoreceptor Transduction Efficacy of Capsid-Modified Adeno-Associated Viral Vectors Following Intravitreal and Subretinal Delivery in Sheep

Ross, M., Obolensky, A., Averbukh, E., Ezra-Elia, R., Yamin, E., Honig, H., Dvir, H., Rosov, A., Hauswirth, W. W., Gootwine, E., et al. (2020) · Human Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 16