Arruda, Valder R.

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 2,163

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

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

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

Manno, C. S., Chew, A. J., Hutchison, S., Larson, P. J., Herzog, R. W., Arruda, V. R., Tai, S. J., Ragni, M. V., Thompson, A., Ozelo, M., et al. (2003) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 749

Induction of Immune Tolerance to Coagulation Factor IX Antigen by In Vivo Hepatic Gene Transfer

Mingozzi, F., Liu, Y.-L., Dobrzynski, E., Kaufhold, A., Liu, J. H., Wang, Y., Arruda, V. R., High, K. A., & Herzog, R. W. (2003) · Journal of Clinical Investigation

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 466

X-Linked Thrombophilia with a Mutant Factor IX (Factor IX Padua)

Simioni, P., Tormene, D., Tognin, G., Gavasso, S., Bulato, C., Iacobelli, N. P., Finn, J. D., Spiezia, L., Radu, C., & Arruda, V. R. (2009) · New England Journal of Medicine

Hemophilia Treatment and ResearchBlood Coagulation and Thrombosis Mechanisms

Cited by 394

Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

Mount, J. D., Herzog, R. W., Tillson, D. M., Goodman, S. A., Robinson, N., McCleland, M. L., Bellinger, D., Nichols, T. C., Arruda, V. R., Lothrop, C. D., et al. (2002) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 339

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

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

Eradication of neutralizing antibodies to factor VIII in canine hemophilia a after liver gene therapy

Finn, J. D., Ozelo, M. C., Sabatino, D. E., Franck, H. W. G., Merricks, E. P., Crudele, J. M., Zhou, S., Kazazian, H. H., Lillicrap, D., Nichols, T. C., et al. (2010) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 157

AAV liver expression of FIX-padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia b dogs and mice

Crudele, J. M., Finn, J. D., Siner, J. I., Martin, N. B., Niemeyer, G. P., Zhou, S., Mingozzi, F., Lothrop, C. D., & Arruda, V. R. (2015) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 153

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

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

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Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy

Samelson-Jones, B. J., & Arruda, V. R. (2019) · Molecular Therapy - Methods & Clinical Development

Hemophilia Treatment and ResearchVirus-based gene therapy research

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Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A

Nguyen, G. N., George, L. A., Siner, J. I., Davidson, R. J., Zander, C. B., Zheng, X. L., Arruda, V. R., Camire, R. M., & Sabatino, D. E. (2017) · Journal of Thrombosis and Haemostasis

Hemophilia Treatment and ResearchVirus-based gene therapy research

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Timing of Intensive Immunosuppression Impacts Risk of Transgene Antibodies after AAV Gene Therapy in Nonhuman Primates

Samelson-Jones, B. J., Finn, J. D., Favaro, P., Wright, J. F., & Arruda, V. R. (2020) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchHemophilia Treatment and Research

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Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity

Samelson-Jones, B. J., Finn, J. D., George, L. A., Camire, R. M., & Arruda, V. R. (2019) · JCI Insight

Hemophilia Treatment and ResearchVirus-based gene therapy research

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Obstacles and future of gene therapy for hemophilia

Arruda, V. R., & Samelson-Jones, B. J. (2015) · Expert Opinion on Orphan Drugs

Hemophilia Treatment and ResearchVirus-based gene therapy research

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Evolutionary Insights Into Coagulation Factor IX Padua and Other High-Specific-Activity Variants

Samelson-Jones, B. J., Finn, J. D., Raffini, L. J., Merricks, E. P., Camire, R. M., Nichols, T. C., & Arruda, V. R. (2021) · Blood Advances

Hemophilia Treatment and ResearchBlood Coagulation and Thrombosis Mechanisms

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Characterization of Naturally-Occurring Humoral Immunity to AAV in Sheep

Tellez, J., Van Vliet, K., Tseng, Y.-S., Finn, J. D., Tschernia, N., Almeida-Porada, G., Arruda, V. R., Agbandje-McKenna, M., & Porada, C. D. (2013) · PLoS ONE

Virus-based gene therapy researchHemophilia Treatment and Research

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