High, Katherine A.

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

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

McIntosh, J., Lenting, P. J., Rosales, C., Lee, D., Rabbanian, S., Raj, D., Patel, N., Tuddenham, E. G. D., Christophe, O. D., McVey, J. H., et al. (2013) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 278

Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy

Niemeyer, G. P., Herzog, R. W., Mount, J., Arruda, V. R., Tillson, D. M., Hathcock, J., van Ginkel, F. W., High, K. A., & Lothrop, C. D. (2009) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 276

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

Long-term follow-up of the first in human intravascular delivery of AAV for gene transfer: AAV2-hFIX16 for severe hemophilia b

George, L. A., Ragni, M. V., Rasko, J. E. J., Raffini, L. J., Samelson-Jones, B. J., Ozelo, M., Hazbon, M., Runowski, A. R., Wellman, J. A., Wachtel, K., et al. (2020) · Molecular Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 213

Intramuscular Administration of AAV1-Lipoprotein Lipase S447X Lowers Triglycerides in Lipoprotein Lipase–Deficient Patients

Stroes, E. S., Nierman, M. C., Meulenberg, J. J., Franssen, R., Twisk, J., Henny, C. P., Maas, M. M., Zwinderman, A. H., Ross, C., Aronica, E., et al. (2008) · Arteriosclerosis, Thrombosis, and Vascular Biology

Lipid metabolism and disordersVirus-based gene therapy research

Cited by 209

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape

Mingozzi, F., & High, K. A. (2017) · Annual Review of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 206

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

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

Muscle-Directed Gene Transfer and Transient Immune Suppression Result in Sustained Partial Correction of Canine Hemophilia B Caused by a Null Mutation

Herzog, R. W., Mount, J. D., Arruda, V. R., High, K. A., & Lothrop, C. D. (2001) · Molecular Therapy

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 176

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells

Martino, A. T., Basner-Tschakarjan, E., Markusic, D. M., Finn, J. D., Hinderer, C., Zhou, S., Ostrov, D. A., Srivastava, A., Ertl, H. C. J., Terhorst, C., et al. (2013) · Blood

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 162

Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy

Herzog, R. W., Fields, P. A., Arruda, V. R., Brubaker, J. O., Armstrong, E., McClintock, D., Bellinger, D. A., Couto, L. B., Nichols, T. C., & High, K. A. (2002) · Human Gene Therapy

Hemophilia Treatment and ResearchVirus-based gene therapy research

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

Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue

Mingozzi, F., Chen, Y., Edmonson, S. C., Zhou, S., Thurlings, R. M., Tak, P. P., High, K. A., & Vervoordeldonk, M. J. (2013) · Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 138

Risk and Prevention of Anti-factor IX Formation in AAV-Mediated Gene Transfer in the Context of a Large Deletion of F9

Fields, P. A., Arruda, V. R., Armstrong, E., Chu, K., Mingozzi, F., Hagstrom, J. N., Herzog, R. W., & High, K. A. (2001) · Molecular Therapy

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 127

Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

Markusic, D. M., Hoffman, B. E., Perrin, G. Q., Nayak, S., Wang, X., LoDuca, P. A., High, K. A., & Herzog, R. W. (2013) · EMBO Molecular Medicine

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 116

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

Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid

Vercauteren, K., Hoffman, B. E., Zolotukhin, I., Keeler, G. D., Xiao, J. W., Basner-Tschakarjan, E., High, K. A., Ertl, H. C., Rice, C. M., Srivastava, A., et al. (2016) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 114

Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia b

Mingozzi, F., Chen, Y., Murphy, S. L., Edmonson, S. C., Tai, A., Price, S. D., Metzger, M. E., Zhou, S., Wright, J. F., Donahue, R. E., et al. (2012) · Molecular Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 112

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

Gene therapy for the hemophilias

Kay, M. A., & High, K. (1999) · Proceedings of the National Academy of Sciences

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 96

Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials With Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy

Ertl, H. C. J., & High, K. A. (2017) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 94

Impact of the Underlying Mutation and the Route of Vector Administration on Immune Responses to Factor IX in Gene Therapy for Hemophilia B

Cao, O., Hoffman, B. E., Moghimi, B., Nayak, S., Cooper, M., Zhou, S., Ertl, H. C., High, K. A., & Herzog, R. W. (2009) · Molecular Therapy

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 92