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1.

Integration and the risk of liver cancer-Is there a real risk?

Kasimsetty, Aradhana; Sabatino, Denise E.

J Viral Hepat ; 31 Suppl 1: 26-34, 2024 04.

Artículo en Inglés | MEDLINE | ID: mdl-38606944

2.

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.

Mol Ther ; 30(8): 2646-2663, 2022 08 03.

Artículo en Inglés | MEDLINE | ID: mdl-35690906

3.

Origins and organization of the NHLBI State of the Science Workshop: Generating a national blueprint for future research on factor VIII inhibitors.

Sabatino, Denise E; Pipe, Steven W; Nugent, Diane J; Soucie, J Michael; Hooper, W Craig; Hoots, W Keith; DiMichele, Donna M.

Haemophilia ; 25(4): 575-580, 2019 Jul.

Artículo en Inglés | MEDLINE | ID: mdl-31329369

4.

Clogging up the pipeline: factor VIII aggregates.

Sabatino, Denise E.

Blood ; 135(21): 1825-1827, 2020 05 21.

Artículo en Inglés | MEDLINE | ID: mdl-32437559

5.

Topics in AAV integration come front and center at ASGCT AAV Integration Roundtable.

Sabatino, Denise E; McCarty, Douglas M.

Mol Ther ; 29(12): 3319-3320, 2021 12 01.

Artículo en Inglés | MEDLINE | ID: mdl-34758291

6.

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.

Blood ; 121(21): 4396-403, 2013 May 23.

Artículo en Inglés | MEDLINE | ID: mdl-23372167

7.

Executive summary of the NHLBI State of the Science (SOS) Workshop: Overview and next steps in generating a national blueprint for future research on factor VIII inhibitors.

Pipe, Steven W; Sabatino, Denise E; Nugent, Diane J; Hooper, W Craig; Soucie, J Michael; Keith Hoots, W; DiMichele, Donna M.

Haemophilia ; 25(4): 610-615, 2019 Jul.

Artículo en Inglés | MEDLINE | ID: mdl-31329363

8.

CD8(+) T-cell responses to adeno-associated virus capsid in humans.

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; High, Katherine A.

Nat Med ; 13(4): 419-22, 2007 Apr.

Artículo en Inglés | MEDLINE | ID: mdl-17369837

9.

Optimizing Liver Health Before and After Gene Therapy for Hemophilia A.

Ragni, Margaret V; Mead, Henry; de Jong, Ype P; Kaczmarek, Radoslaw; Leavitt, Andrew D; Long, Brian; Nugent, Diane; Sabatino, Denise E; Fong, Sylvia; von Drygalski, Annette; Walsh, Christopher E; Luxon, Bruce.

Blood Adv ; 2024 Jun 06.

Artículo en Inglés | MEDLINE | ID: mdl-38843379

10.

Light chain of factor VIII is sufficient for accelerating cleavage of von Willebrand factor by ADAMTS13 metalloprotease.

Cao, Wenjing; Sabatino, Denise E; Altynova, Ekaterina; Lange, Amy M; Casina, Veronica C; Camire, Rodney M; Zheng, X Long.

J Biol Chem ; 287(39): 32459-66, 2012 Sep 21.

Artículo en Inglés | MEDLINE | ID: mdl-22854959

11.

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.

J Thromb Haemost ; 21(8): 2101-2113, 2023 08.

Artículo en Inglés | MEDLINE | ID: mdl-37080538

12.

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.

J Thromb Haemost ; 21(12): 3477-3489, 2023 12.

Artículo en Inglés | MEDLINE | ID: mdl-37726033

13.

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.

Blood ; 116(26): 5842-8, 2010 Dec 23.

Artículo en Inglés | MEDLINE | ID: mdl-20876851

14.

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, Haig H.

Mol Ther ; 19(3): 442-9, 2011 Mar.

Artículo en Inglés | MEDLINE | ID: mdl-21081906

15.

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.

Mol Ther Methods Clin Dev ; 24: 20-29, 2022 Mar 10.

Artículo en Inglés | MEDLINE | ID: mdl-34977269

16.

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.

Blood ; 114(20): 4562-5, 2009 Nov 12.

Artículo en Inglés | MEDLINE | ID: mdl-19770361

17.

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.

Nat Biotechnol ; 39(1): 47-55, 2021 01.

Artículo en Inglés | MEDLINE | ID: mdl-33199875

18.

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.

Sci Adv ; 6(34)2020 08.

Artículo en Inglés | MEDLINE | ID: mdl-32937374

19.

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

Murphy, Samuel L; Li, Hojun; Mingozzi, Federico; Sabatino, Denise E; Hui, Daniel J; Edmonson, Shyrie A; High, Katherine A.

J Med Virol ; 81(1): 65-74, 2009 Jan.

Artículo en Inglés | MEDLINE | ID: mdl-19031458

20.

Promise of gene therapy for congenital neurologic disease due to GPI deficiency.

Babushok, Daria V; Sabatino, Denise E.

Mol Ther Methods Clin Dev ; 32(2): 101240, 2024 Jun 13.

Artículo en Inglés | MEDLINE | ID: mdl-38572066

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