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George, Lindsey A., Ragni, Margaret V., Rasko, John E.J., Raffini, Leslie J., Samelson-Jones, Benjamin J., Ozelo, Margareth, Hazbon, Maria, Runowski, Alexa R., Wellman, Jennifer A., Wachtel, Katie, Chen, Yifeng, Anguela, Xavier M., Kuranda, Klaudia, Mingozzi, Federico, High, Katherine A.
Published in Molecular therapy (02.09.2020)
Published in Molecular therapy (02.09.2020)
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In vivo genome editing of the albumin locus as a platform for protein replacement therapy
Sharma, Rajiv, Anguela, Xavier M., Doyon, Yannick, Wechsler, Thomas, DeKelver, Russell C., Sproul, Scott, Paschon, David E., Miller, Jeffrey C., Davidson, Robert J., Shivak, David, Zhou, Shangzhen, Rieders, Julianne, Gregory, Philip D., Holmes, Michael C., Rebar, Edward J., High, Katherine A.
Published in Blood (08.10.2015)
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Overcoming preexisting humoral immunity to AAV using capsid decoys
Mingozzi, Federico, Anguela, Xavier M, Pavani, Giulia, Chen, Yifeng, Davidson, Robert J, Hui, Daniel J, Yazicioglu, Mustafa, Elkouby, Liron, Hinderer, Christian J, Faella, Armida, Howard, Carolann, Tai, Alex, Podsakoff, Gregory M, Zhou, Shangzhen, Basner-Tschakarjan, Etiena, Wright, John Fraser, High, Katherine A
Published in Science translational medicine (17.07.2013)
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Robust ZFN-mediated genome editing in adult hemophilic mice
Anguela, Xavier M., Sharma, Rajiv, Doyon, Yannick, Miller, Jeffrey C., Li, Hojun, Haurigot, Virginia, Rohde, Michelle E., Wong, Sunnie Y., Davidson, Robert J., Zhou, Shangzhen, Gregory, Philip D., Holmes, Michael C., High, Katherine A.
Published in Blood (07.11.2013)
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Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice
Zou, Chenhui, Vercauteren, Koen O.A., Michailidis, Eleftherios, Kabbani, Mohammad, Zoluthkin, Irene, Quirk, Corrine, Chiriboga, Luis, Yazicioglu, Mustafa, Anguela, Xavier M., Meuleman, Philip, High, Katherine A., Herzog, Roland W., de Jong, Ype P.
Published in Molecular therapy. Methods & clinical development (11.09.2020)
Published in Molecular therapy. Methods & clinical development (11.09.2020)
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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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Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
George, Lindsey A, Monahan, Paul E, Eyster, M. Elaine, Sullivan, Spencer K, Ragni, Margaret V, Croteau, Stacy E, Rasko, John E.J, Recht, Michael, Samelson-Jones, Benjamin J, MacDougall, Amy, Jaworski, Kristen, Noble, Robert, Curran, Marla, Kuranda, Klaudia, Mingozzi, Federico, Chang, Tiffany, Reape, Kathleen Z, Anguela, Xavier M, High, Katherine A
Published in The New England journal of medicine (18.11.2021)
Published in The New England journal of medicine (18.11.2021)
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Reversal of Type 1 Diabetes by Engineering a Glucose Sensor in Skeletal Muscle
MAS, Alex, MONTANE, Joel, ANGUELA, Xavier M, MUNOZ, Sergio, DOUAR, Anne M, RIU, Efren, OTAEGUI, Pedro, BOSCH, Fatima
Published in Diabetes (New York, N.Y.) (01.06.2006)
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In vivo genome editing restores haemostasis in a mouse model of haemophilia
Li, Hojun, Haurigot, Virginia, Doyon, Yannick, Li, Tianjian, Wong, Sunnie Y., Bhagwat, Anand S., Malani, Nirav, Anguela, Xavier M., Sharma, Rajiv, Ivanciu, Lacramiora, Murphy, Samuel L., Finn, Jonathan D., Khazi, Fayaz R., Zhou, Shangzhen, Paschon, David E., Rebar, Edward J., Bushman, Frederic D., Gregory, Philip D., Holmes, Michael C., High, Katherine A.
Published in Nature (London) (14.07.2011)
Published in Nature (London) (14.07.2011)
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Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates
Costa-Verdera, Helena, Collaud, Fanny, Riling, Christopher R., Sellier, Pauline, Nordin, Jayme M. L., Preston, G. Michael, Cagin, Umut, Fabregue, Julien, Barral, Simon, Moya-Nilges, Maryse, Krijnse-Locker, Jacomina, van Wittenberghe, Laetitia, Daniele, Natalie, Gjata, Bernard, Cosette, Jeremie, Abad, Catalina, Simon-Sola, Marcelo, Charles, Severine, Li, Mathew, Crosariol, Marco, Antrilli, Tom, Quinn, William J., Gross, David A., Boyer, Olivier, Anguela, Xavier M., Armour, Sean M., Colella, Pasqualina, Ronzitti, Giuseppe, Mingozzi, Federico
Published in Nature communications (04.11.2021)
Published in Nature communications (04.11.2021)
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IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies
Leborgne, Christian, Barbon, Elena, Alexander, Jeffrey M., Hanby, Hayley, Delignat, Sandrine, Cohen, Daniel M., Collaud, Fanny, Muraleetharan, Saghana, Lupo, Dan, Silverberg, Joseph, Huang, Karen, van Wittengerghe, Laetitia, Marolleau, Béatrice, Miranda, Adeline, Fabiano, Anna, Daventure, Victoria, Beck, Heena, Anguela, Xavier M., Ronzitti, Giuseppe, Armour, Sean M., Lacroix-Desmazes, Sebastien, Mingozzi, Federico
Published in Nature Medicine (01.07.2020)
Published in Nature Medicine (01.07.2020)
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Liver Production of Sulfamidase Reverses Peripheral and Ameliorates CNS Pathology in Mucopolysaccharidosis IIIA Mice
Ruzo, Albert, Garcia, Miquel, Ribera, Albert, Villacampa, Pilar, Haurigot, Virginia, Marcó, Sara, Ayuso, Eduard, Anguela, Xavier M, Roca, Carles, Agudo, Judith, Ramos, David, Ruberte, Jesús, Bosch, Fatima
Published in Molecular therapy (01.02.2012)
Published in Molecular therapy (01.02.2012)
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Nonviral-Mediated Hepatic Expression of IGF-I Increases Treg Levels and Suppresses Autoimmune Diabetes in Mice
ANGUELA, Xavier M, TAFURO, Sabrina, BOSCH, Fatima, ROCA, Carles, CALLEJAS, David, AGUDO, Judith, OBACH, Merce, RIBERA, Albert, RUZO, Albert, MANN, Christopher J, CASELLAS, Alba
Published in Diabetes (New York, N.Y.) (01.02.2013)
Published in Diabetes (New York, N.Y.) (01.02.2013)
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Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
George, Lindsey A, Sullivan, Spencer K, Giermasz, Adam, Rasko, John E.J, Samelson-Jones, Benjamin J, Ducore, Jonathan, Cuker, Adam, Sullivan, Lisa M, Majumdar, Suvankar, Teitel, Jerome, McGuinn, Catherine E, Ragni, Margaret V, Luk, Alvin Y, Hui, Daniel, Wright, J. Fraser, Chen, Yifeng, Liu, Yun, Wachtel, Katie, Winters, Angela, Tiefenbacher, Stefan, Arruda, Valder R, van der Loo, Johannes C.M, Zelenaia, Olga, Takefman, Daniel, Carr, Marcus E, Couto, Linda B, Anguela, Xavier M, High, Katherine A
Published in The New England journal of medicine (07.12.2017)
Published in The New England journal of medicine (07.12.2017)
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