Evaluating the state of the science for adeno-associated virus integration: An integrated perspective
Sabatino, Denise E., Bushman, Frederic D., Chandler, Randy J., Crystal, Ronald G., Davidson, Beverly L., Dolmetsch, Ricardo, Eggan, Kevin C., Gao, Guangping, Gil-Farina, Irene, Kay, Mark A., McCarty, Douglas M., Montini, Eugenio, Ndu, Adora, Yuan, Jing
Published in Molecular therapy (03.08.2022)
Published in Molecular therapy (03.08.2022)
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Journal Article
Functionalized lipid-like nanoparticles for in vivo mRNA delivery and base editing
Zhang, Xinfu, Zhao, Weiyu, Nguyen, Giang N, Zhang, Chengxiang, Zeng, Chunxi, Yan, Jingyue, Du, Shi, Hou, Xucheng, Li, Wenqing, Jiang, Justin, Deng, Binbin, McComb, David W, Dorkin, Robert, Shah, Aalok, Barrera, Luis, Gregoire, Francine, Singh, Manmohan, Chen, Delai, Sabatino, Denise E, Dong, Yizhou
Published in Science advances (01.08.2020)
Published in Science advances (01.08.2020)
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Journal Article
Promise of gene therapy for congenital neurologic disease due to GPI deficiency
Babushok, Daria V., Sabatino, Denise E.
Published in Molecular therapy. Methods & clinical development (13.06.2024)
Published in Molecular therapy. Methods & clinical development (13.06.2024)
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Journal Article
Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype
Siner, Joshua I., Iacobelli, Nicholas P., Sabatino, Denise E., Ivanciu, Lacramiora, Zhou, Shangzhen, Poncz, Mortimer, Camire, Rodney M., Arruda, Valder R.
Published in Blood (23.05.2013)
Published in Blood (23.05.2013)
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Journal Article
Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy
Finn, Jonathan D., Ozelo, Margareth C., Sabatino, Denise E., Franck, Helen W.G., Merricks, Elizabeth P., Crudele, Julie M., Zhou, Shangzhen, Kazazian, Haig H., Lillicrap, David, Nichols, Timothy C., Arruda, Valder R.
Published in Blood (23.12.2010)
Published in Blood (23.12.2010)
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Journal Article
Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver
Mingozzi, Federico, Hasbrouck, Nicole C., Basner-Tschakarjan, Etiena, Edmonson, Shyrie A., Hui, Daniel J., Sabatino, Denise E., Zhou, Shangzhen, Wright, J. Fraser, Jiang, Haiyan, Pierce, Glenn F., Arruda, Valder R., High, Katherine A.
Published in Blood (01.10.2007)
Published in Blood (01.10.2007)
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Journal Article
Efficacy and Safety of Long-term Prophylaxis in Severe Hemophilia A Dogs Following Liver Gene Therapy Using AAV Vectors
Sabatino, Denise E, Lange, Amy M, Altynova, Ekaterina S, Sarkar, Rita, Zhou, Shangzhen, Merricks, Elizabeth P, Franck, Helen G, Nichols, Timothy C, Arruda, Valder R, Kazazian Jr, Haig H
Published in Molecular therapy (01.03.2011)
Published in Molecular therapy (01.03.2011)
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Journal Article
Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A
Elkouby, Liron, Armour, Sean M., Toso, Raffaella, DiPietro, Marti, Davidson, Robert J., Nguyen, Giang N., Willet, Mallory, Kutza, Stephanie, Silverberg, Joseph, Frick, Jennifer, Crosariol, Marco, Wang, Yuhuan, Wang, Chuansong, High, Katherine A., Sabatino, Denise E., Anguela, Xavier M.
Published in Molecular therapy. Methods & clinical development (10.03.2022)
Published in Molecular therapy. Methods & clinical development (10.03.2022)
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Journal Article
Persistent Expression of hF.IX After Tolerance Induction by In Utero or Neonatal Administration of AAV-1-F.IX in Hemophilia B Mice
Sabatino, Denise E, MacKenzie, Tippi C, Peranteau, William, Edmonson, Shyrie, Campagnoli, Cesare, Liu, Yi-Lin, Flake, Alan W, High, Katherine A
Published in Molecular therapy (01.09.2007)
Published in Molecular therapy (01.09.2007)
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Journal Article
Recombinant canine B-domain–deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model
Sabatino, Denise E., Freguia, Christian Furlan, Toso, Raffaella, Santos, Andrey, Merricks, Elizabeth P., Kazazian, Haig H., Nichols, Timothy C., Camire, Rodney M., Arruda, Valder R.
Published in Blood (12.11.2009)
Published in Blood (12.11.2009)
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Journal Article
A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells
Nguyen, Giang N., Everett, John K., Kafle, Samita, Roche, Aoife M., Raymond, Hayley E., Leiby, Jacob, Wood, Christian, Assenmacher, Charles-Antoine, Merricks, Elizabeth P., Long, C. Tyler, Kazazian, Haig H., Nichols, Timothy C., Bushman, Frederic D., Sabatino, Denise E.
Published in Nature biotechnology (01.01.2021)
Published in Nature biotechnology (01.01.2021)
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Journal Article
Major erythrocyte membrane protein genes in EKLF-deficient mice
Nilson, Douglas G., Sabatino, Denise E., Bodine, David M., Gallagher, Patrick G.
Published in Experimental hematology (01.06.2006)
Published in Experimental hematology (01.06.2006)
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Journal Article
Altered cleavage of human factor VIII at the B-domain and acidic region 3 interface enhances expression after gene therapy in hemophilia A mice
Nguyen, Giang N., Lindgren, Jonathan R., Seleme, Maria C., Kafle, Samita, Zander, Catherine B., Zheng, X. Long, Sabatino, Denise E.
Published in Journal of thrombosis and haemostasis (01.08.2023)
Published in Journal of thrombosis and haemostasis (01.08.2023)
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Journal Article
CD8 + T-cell responses to adeno-associated virus capsid in humans
High, Katherine A, Mingozzi, Federico, Maus, Marcela V, Hui, Daniel J, Sabatino, Denise E, Murphy, Samuel L, Rasko, John E J, Ragni, Margaret V, Manno, Catherine S, Sommer, Jurg, Jiang, Haiyan, Pierce, Glenn F, Ertl, Hildegund C J
Published in Nature medicine (01.04.2007)
Published in Nature medicine (01.04.2007)
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Journal Article
Coagulation factor VIII regulates von Willebrand factor homeostasis invivo
Cao, Wenjing, Trask, Aria R, Bignotti, Antonia I, George, Lindsey A, Doshi, Bhavya S, Sabatino, Denise E, Yada, Noritaka, Zheng, Liang, Camire, Rodney M, Zheng, X Long
Published in Journal of thrombosis and haemostasis (01.12.2023)
Published in Journal of thrombosis and haemostasis (01.12.2023)
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