Chulay, Jeffrey D.

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α 1 -Antitrypsin: Interim Results

Flotte, T. R., Trapnell, B. C., Humphries, M., Carey, B., Calcedo, R., Rouhani, F., Campbell-Thompson, M., Yachnis, A. T., Sandhaus, R. A., McElvaney, N. G., et al. (2011) · Human Gene Therapy

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Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

Brantly, M. L., Chulay, J. D., Wang, L., Mueller, C., Humphries, M., Spencer, L. T., Rouhani, F., Conlon, T. J., Calcedo, R., Betts, M. R., et al. (2009) · Proceedings of the National Academy of Sciences

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Results at 2 Years after gene therapy for RPE65-deficient leber congenital amaurosis and severe early-childhood–onset retinal dystrophy

Weleber, R. G., Pennesi, M. E., Wilson, D. J., Kaushal, S., Erker, L. R., Jensen, L., McBride, M. T., Flotte, T. R., Humphries, M., Calcedo, R., et al. (2016) · Ophthalmology

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Randomized, double-blind, Phase 1 trial of an alphavirus replicon vaccine for cytomegalovirus in CMV seronegative adult volunteers

Bernstein, D. I., Reap, E. A., Katen, K., Watson, A., Smith, K., Norberg, P., Olmsted, R. A., Hoeper, A., Morris, J., Negri, S., et al. (2009) · Vaccine

Cytomegalovirus and herpesvirus researchHerpesvirus Infections and Treatments

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Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression

Mueller, C., Chulay, J. D., Trapnell, B. C., Humphries, M., Carey, B., Sandhaus, R. A., McElvaney, N. G., Messina, L., Tang, Q., Rouhani, F. N., et al. (2013) · Journal of Clinical Investigation

Virus-based gene therapy researchRNA Interference and Gene Delivery

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5 Year expression and neutrophil defect repair after gene therapy in alpha-1 antitrypsin deficiency

Mueller, C., Gernoux, G., Gruntman, A. M., Borel, F., Reeves, E. P., Calcedo, R., Rouhani, F. N., Yachnis, A., Humphries, M., Campbell-Thompson, M., et al. (2017) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

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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

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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

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Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin

Ye, G.-J., Budzynski, E., Sonnentag, P., Miller, P. E., Sharma, A. K., Ver Hoeve, J. N., Howard, K., Knop, D. R., Neuringer, M., McGill, T., et al. (2015) · Human Gene Therapy Clinical Development

Virus-based gene therapy researchRetinal Development and Disorders

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Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa

Dufour, V. L., Cideciyan, A. V., Ye, G.-J., Song, C., Timmers, A., Habecker, P. L., Pan, W., Weinstein, N. M., Swider, M., Durham, A. C., et al. (2020) · Human Gene Therapy

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Safety and Efficacy Evaluation of rAAV2tYF-PR1.7-hCNGA3 Vector Delivered by Subretinal Injection in CNGA3 Mutant Achromatopsia Sheep

Gootwine, E., Ofri, R., Banin, E., Obolensky, A., Averbukh, E., Ezra-Elia, R., Ross, M., Honig, H., Rosov, A., Yamin, E., et al. (2017) · Human Gene Therapy Clinical Development

Retinal Development and DisordersVirus-based gene therapy research

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Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy

Song, C., Dufour, V. L., Cideciyan, A. V., Ye, G.-J., Swider, M., Newmark, J. A., Timmers, A. M., Robinson, P. M., Knop, D. R., Chulay, J. D., et al. (2020) · Human Gene Therapy

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Herpes Simplex Virus Clearance During Purification of a Recombinant Adeno-Associated Virus Serotype 1 Vector

Ye, G.-J., Scotti, M. M., Thomas, D. L., Wang, L., Knop, D. R., & Chulay, J. D. (2014) · Human Gene Therapy Clinical Development

Virus-based gene therapy researchProtein purification and stability

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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

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