Hui, Daniel J.

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

George, L. A., Sullivan, S. K., Giermasz, A., Rasko, J. E. J., Samelson-Jones, B. J., Ducore, J., Cuker, A., Sullivan, L. M., Majumdar, S., Teitel, J., et al. (2017) · New England Journal of Medicine

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 762

Cd8(+) T-Cell Responses to Adeno-Associated Virus Capsid in Humans

Mingozzi, F., Maus, M. V., Hui, D. J., Sabatino, D. E., Murphy, S. L., Rasko, J. E. J., Ragni, M. V., Manno, C. S., Sommer, J., Jiang, H., et al. (2007) · Nature Medicine

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 748

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

Mingozzi, F., Anguela, X. M., Pavani, G., Chen, Y., Davidson, R. J., Hui, D. J., Yazicioglu, M., Elkouby, L., Hinderer, C. J., Faella, A., et al. (2013) · Science Translational Medicine

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 355

AAV-1–mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

Mingozzi, F., Meulenberg, J. J., Hui, D. J., Basner-Tschakarjan, E., Hasbrouck, N. C., Edmonson, S. A., Hutnick, N. A., Betts, M. R., Kastelein, J. J., Stroes, E. S., et al. (2009) · Blood

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 295

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

Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver

Mingozzi, F., Hasbrouck, N. C., Basner-Tschakarjan, E., Edmonson, S. A., Hui, D. J., Sabatino, D. E., Zhou, S., Wright, J. F., Jiang, H., Pierce, G. F., et al. (2007) · Blood

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 254

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

Pien, G. C., Basner-Tschakarjan, E., Hui, D. J., Mentlik, A. N., Finn, J. D., Hasbrouck, N. C., Zhou, S., Murphy, S. L., Maus, M. V., Mingozzi, F., et al. (2009) · Journal of Clinical Investigation

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 192

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

Proteasome Inhibitors Decrease AAV2 Capsid derived Peptide Epitope Presentation on MHC Class I Following Transduction

Finn, J. D., Hui, D., Downey, H. D., Dunn, D., Pien, G. C., Mingozzi, F., Zhou, S., & High, K. A. (2010) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 115

Diverse IgG subclass responses to adeno‐associated virus infection and vector administration

Murphy, S. L., Li, H., Mingozzi, F., Sabatino, D. E., Hui, D. J., Edmonson, S. A., & High, K. A. (2009) · Journal of Medical Virology

Virus-based gene therapy researchMonoclonal and Polyclonal Antibodies Research

Cited by 101

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes

Hui, D. J., Edmonson, S. C., Podsakoff, G. M., Pien, G. C., Ivanciu, L., Camire, R. M., Ertl, H., Mingozzi, F., High, K. A., & Basner-Tschakarjan, E. (2015) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 84

A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds

High, K. A., George, L. A., Eyster, M. E., Sullivan, S. K., Ragni, M. V., Croteau, S. E., Samelson-Jones, B. J., Evans, M., Joseney-Antoine, M., Macdougall, A., et al. (2018) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 57

Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes

Hui, D. J., Basner-Tschakarjan, E., Chen, Y., Davidson, R. J., Buchlis, G., Yazicioglu, M., Pien, G. C., Finn, J. D., Haurigot, V., Tai, A., et al. (2013) · Molecular Therapy

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 47