Integration and the risk of liver cancer-Is there a real risk? von Kasimsetty, Aradhana, Sabatino, Denise E

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Clogging up the pipeline: factor VIII aggregates von Sabatino, Denise E.

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Evaluating the state of the science for adeno-associated virus integration: An integrated perspective von 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

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A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells von 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.

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Topics in AAV integration come front and center at ASGCT AAV Integration Roundtable von Sabatino, Denise E., McCarty, Douglas M.

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Functionalized lipid-like nanoparticles for in vivo mRNA delivery and base editing von 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

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Promise of gene therapy for congenital neurologic disease due to GPI deficiency von Babushok, Daria V., Sabatino, Denise E.

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Altered cleavage of human factor VIII at the B-domain and acidic region 3 interface enhances expression after gene therapy in hemophilia A mice von Nguyen, Giang N., Lindgren, Jonathan R., Seleme, Maria C., Kafle, Samita, Zander, Catherine B., Zheng, X. Long, Sabatino, Denise E.

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Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype von Siner, Joshua I., Iacobelli, Nicholas P., Sabatino, Denise E., Ivanciu, Lacramiora, Zhou, Shangzhen, Poncz, Mortimer, Camire, Rodney M., Arruda, Valder R.

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Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy von 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.

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CD8 + T-cell responses to adeno-associated virus capsid in humans von 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

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Coagulation factor VIII regulates von Willebrand factor homeostasis invivo von 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

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

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Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice von Lange, Amy M, Altynova, Ekaterina S, Nguyen, Giang N, Sabatino, Denise E

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Diverse IgG subclass responses to adeno-associated virus infection and vector administration von Murphy, Samuel L, Li, Hojun, Mingozzi, Federico, Sabatino, Denise E, Hui, Daniel J, Edmonson, Shyrie A, High, Katherine A

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Efficacy and Safety of Long-term Prophylaxis in Severe Hemophilia A Dogs Following Liver Gene Therapy Using AAV Vectors von 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

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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response von High, Katherine A, Manno, Catherine S, Pierce, Glenn F, Arruda, Valder R, Glader, Bertil, Ragni, Margaret, Rasko, John J E, Ozelo, Margareth C, Hoots, Keith, Blatt, Philip, Konkle, Barbara, Dake, Michael, Kaye, Robin, Razavi, Mahmood, Zajko, Albert, Zehnder, James, Rustagi, Pradip, Nakai, Hiroyuki, Chew, Amy, Leonard, Debra, Wright, J Fraser, Lessard, Ruth R, Sommer, Jürg M, Tigges, Michael, Sabatino, Denise, Luk, Alvin, Jiang, Haiyan, Mingozzi, Federico, Couto, Linda, Ertl, Hildegund C, Kay, Mark A

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Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A von 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.

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Persistent Expression of hF.IX After Tolerance Induction by In Utero or Neonatal Administration of AAV-1-F.IX in Hemophilia B Mice von Sabatino, Denise E, MacKenzie, Tippi C, Peranteau, William, Edmonson, Shyrie, Campagnoli, Cesare, Liu, Yi-Lin, Flake, Alan W, High, Katherine A

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Modulation of AAV transduction and integration targeting by topoisomerase poisons von Kasimsetty, Aradhana, Hwang, Young, Everett, John K., McFarland, Alexander G., Zolnoski, Sonja A., Lu, Tianyu, Roche, Aoife M., Martínez-García, Pedro Manuel, Sabatino, Denise E., Bushman, Frederic D.

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