Helper-dependent adenovirus achieve more efficient and persistent liver transgene expression in non-human primates under immunosuppression von Unzu, C, Melero, I, Hervás-Stubbs, S, Sampedro, A, Mancheño, U, Morales-Kastresana, A, Serrano-Mendioroz, I, de Salamanca, R E, Benito, A, Fontanellas, A

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O138 PHASE 1 CLINICAL TRIAL OF LIVER DIRECTED GENE THERAPY WITH rAAV5-PBGD IN ACUTE INTERMITTENT PORPHYRIA: PRELIMINARY SAFETY DATA von D'Avola, D, López-Franco, E, Harper, P, Fontanellas, A, Grosios, N, Sangro, B, Henrichson, A, Salmon, F, Fuertes, C, Abecia, S, Pañeda, A, Paz, M, Cornet, M.E, Municio, M.D.M, Troconiz, I, Kaeppel, C, Juan, R, von Kalle, C, Schmidt, M, Petry, H, Gonzalez-Aseguinolaza, G, de Salamanca, R. Enriquez, Prieto, J

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Helper-dependent adenovirus achieve more efficient and persistent liver transgene expression in non-human primates under immunosuppression von Unzu, C, Melero, I, Hervás-Stubbs, S, Sampedro, A, Mancheño, U, Morales-Kastresana, A, Serrano-Mendioroz, I, de Salamanca, R E, Benito, A, Fontanellas, A

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Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria von D’Avola, Delia, López-Franco, Esperanza, Sangro, Bruno, Pañeda, Astrid, Grossios, Nadina, Gil-Farina, Irene, Benito, Alberto, Twisk, Jaap, Paz, María, Ruiz, Juan, Schmidt, Manfred, Petry, Harald, Harper, Pauline, de Salamanca, Rafael Enríquez, Fontanellas, Antonio, Prieto, Jesús, González-Aseguinolaza, Gloria

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137 TOXICOLOGY AND LIVER TRANSDUCTION EFFICACY EVALUATION OF A RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR IN NON HUMAN PRIMATES AS A POTENTIAL TREATMENT FOR ACUTE INTERMITTENT PORPH... von Pañeda, A, Lopez-Franco, E, Fontanellas, A, Unzu, C, Olagüe, C, Ferrero, R, Sampedro, A, Mauleon, I, Hermening, S, Beattie, S, Petry, H, Ruiz, J, Benito, A, Davola, D, Prieto, J, Gonzalez-Aseguinolaza, G

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PET imaging of thymidine kinase gene expression in the liver of non-human primates following systemic delivery of an adenoviral vector von Fontanellas, A, Hervas-Stubbs, S, Sampedro, A, Collantes, M, Azpilicueta, A, Mauleón, I, Pañeda, A, Quincoces, G, Prieto, J, Melero, I, Peñuelas, I

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Molecular heterogeneity of familial porphyria cutanea tarda in Spain: characterization of 10 novel mutations in the UROD gene von Méndez, M., Poblete-Gutiérrez, P., García-Bravo, M., Wiederholt, T., Morán-Jiménez, M.J., Merk, H.F., Garrido-Astray, M.C., Frank, J., Fontanellas, A., Enríquez de Salamanca, R.

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A homozygous mutation in the ferrochelatase gene underlies erythropoietic protoporphyria associated with palmar keratoderma von Méndez, M., Poblete-Gutiérrez, P., Morán-Jiménez, M.-J., Rodriguez, M.-E., Garrido-Astray, M.-C., Fontanellas, A., Frank, J., De Salamanca, R.E.

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1134 AAV MEDIATED LIVER GENE THERAPY PROVIDES PROLONGED ENZYMATIC CORRECTION AND PROTECTS AGAINST INDUCED MOTOR NEUROPATHY IN ACUTE INTERMITTENT PORPHYRIA MICE von Unzu, C, Sampedro, A, Mauleon, I, Alegre, M, Beattie, S, de Salamanca, R. Enríquez, Twisk, J, Timmermans, E.J, Hernández-Alcoceba, R, Melero, I, Petry, H, González-Aseguinolaza, G, Artieda, J, Rodríguez-Pena, M.-S, Prieto, J, Fontanellas, A

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Association between aminolevulinate dehydrase genotypes and blood lead levels in children from a lead-contaminated area in Antofagasta, Chile von PEREZ-BRAVO, F, RUZ, M, MORAN-JIMENEZ, M. J, OLIVARES, M, REBOLLEDO, A, CODOCEO, J, SEPULVEDA, V, JENKIN, A, SANTOS, J. L, FONTANELLAS, A

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Effect of Adeno-Associated Virus Serotype and Genomic Structure on Liver Transduction and Biodistribution in Mice of Both Genders von PANEDA, Astrid, VANRELL, Lucia, FONTANELLAS, Antonio, SOL RODRIGUEZ-PENA, Maria, GONZALEZ-ASEGUINOLAZA, Gloria, MAULEON, Itsaso, CRETTAZ, Julien S, BERRAONDO, Pedro, TIMMERMANS, Eric J, BEATTIE, Stuart G, TWISK, Jaap, VAN DEVENTER, Sander, PRIETO, Jesus

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Apixaban for the Treatment of Venous Thromboembolism Associated with Cancer von Agnelli, Giancarlo, Becattini, Cecilia, Meyer, Guy, Muñoz, Andres, Huisman, Menno V, Connors, Jean M, Cohen, Alexander, Bauersachs, Rupert, Brenner, Benjamin, Torbicki, Adam, Sueiro, Maria R, Lambert, Catherine, Gussoni, Gualberto, Campanini, Mauro, Fontanella, Andrea, Vescovo, Giorgio, Verso, Melina

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Successful therapeutic effect in a mouse model of erythropoietic protoporphyria by partial genetic correction and fluorescence-based selection of hematopoietic cells von FONTANELLAS, A, MENDEZ, M, ENRIQUEZ DE SALAMANCA, R, DE VERNEUIL, H, MAZURIER, F, CARIO-ANDRE, M, NAVARRO, S, GED, C, TAINE, L, GERONIMI, F, RICHARD, E, MOREAU-GAUDRY, F

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Renal failure affects the enzymatic activities of the three first steps in hepatic heme biosynthesis in the acute intermittent porphyria mouse von Unzu, Carmen, Sampedro, Ana, Sardh, Eliane, Mauleón, Itsaso, Enríquez de Salamanca, Rafael, Prieto, Jesús, Salido, Eduardo, Harper, Pauline, Fontanellas, Antonio

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Nonlinear effects of environment on childhood asthma susceptibility von Kothalawala, Dilini M., Weiss, Veronique B. N., Kadalayil, Latha, Granell, Raquel, Curtin, John A., Murray, Clare S., Simpson, Angela, Custovic, Adnan, Tapper, William J., Rezwan, Faisal I., Arshad, Syed Hasan, Holloway, John W., Roberts, Graham C, Turner, Steve W, Haider, Sadia, Fontanella, Sara, Cullinan, Paul

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Does depression influence the postoperative result of total hip arthroplasties? von Pardo Pol, A, Amestoy Ramos, J, Fontanellas-Fes, A, Lizano-Díez, X, Garcia, A L, Marques López, F

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Lung-function trajectories: relevance and implementation in clinical practice von Melén, Erik, Faner, Rosa, Allinson, James P, Bui, Dinh, Bush, Andrew, Custovic, Adnan, Garcia-Aymerich, Judith, Guerra, Stefano, Breyer-Kohansal, Robab, Hallberg, Jenny, Lahousse, Lies, Martinez, Fernando D, Merid, Simon Kebede, Powell, Pippa, Pinnock, Hilary, Stanojevic, Sanja, Vanfleteren, Lowie E G W, Wang, Gang, Dharmage, Shyamali C, Wedzicha, Jadwiga, Agusti, Alvar, Abellan, Alicia, Adcock, Ian, Afzal, Shoaib, Alter, Peter, Backman, Helena, Bertels, Xander, Bloom, Chloe, Bønnelykke, Klaus, Breyer, Marie-Kathrin, Casas, Sandra, Chung, Fan (Kian), Colak, Yunus, Cosio, Borja G., Duijts, Liesbeth, Fabbri, Leonardo, Fontanella, Sara, Fuertes, Elaine, Gonzalez, Juan Ramón, Granell, Raquel, Hartl, Sylvia, Hernandez-Pacheco, Natalia, Holloway, John, Jarvis, Deborah, Koefoed, Hans Jacob, Kole, Tessa, Kumar, Ashish, Langhammer, Arnulf, Lindberg, Anne, Llopis, Maria, Maitland van der Zee, Anke-Hilse, Meteran, Howraman, Minelli, Cosetta, Nwaru, Bright, Olvera, Nuria, Peralta, Gabriela, Ritchie, Andrew, Rönmark, Eva, Ross Chapman, James, Sangüesa Boix, Júlia, Schikowski, Tamara, Schlünssen, Vivi, Shaheen, Seif, Sigsgaard, Torben, Standl, Marie, Talaei, Mohammad, Ullah, Anhar, Ullman, Anders, Valencia-Hernandez, Carlos, van den Berge, Maarten, van Dijk, Yoni, Vestbo, Jørgen, Vijverberg, Susanne, Vikjord, Sigrid Anna, Volgelmeier, Claus, Vonk, Judith, Zounemat Kermani, Nazanin

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Eltrombopag before Procedures in Patients with Cirrhosis and Thrombocytopenia von Afdhal, Nezam H, Giannini, Edoardo G, Tayyab, Ghias, Mohsin, Aftab, Lee, Jin-Woo, Andriulli, Angelo, Jeffers, Lennox, McHutchison, John, Chen, Pei-Jer, Han, Kwang-Hyub, Campbell, Fiona, Hyde, Denise, Brainsky, Andres, Theodore, Dickens

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Vitamin D-binding protein as a biomarker of active disease in acute intermittent porphyria von Serrano-Mendioroz, Irantzu, Sampedro, Ana, Mora, María Isabel, Mauleón, Itsaso, Segura, Victor, Enríquez de Salamanca, Rafael, Harper, Pauline, Sardh, Eliane, Corrales, Fernando José, Fontanellas, Antonio

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International Society of Nephrology first consensus guidance for preclinical animal studies in translational nephrology von Nangaku, Masaomi, Kitching, A. Richard, Boor, Peter, Fornoni, Alessia, Floege, Jürgen, Coates, P. Toby, Himmelfarb, Jonathan, Lennon, Rachel, Anders, Hans-Joachim, Humphreys, Benjamin D., Caskey, Fergus J., Fogo, Agnes B., Angeletti, Andrea, Bedard, Patricia W., Benigni, Ariela, Granqvist, Anna Björnson, Chabova, Vera Certikova, Chatziantoniou, Christos, Cross, Jennifer, Damster, Sandrine, Donner, Jo-Ann, Eitner, Frank, Faguer, Stanislas, Fontanella, Antonio, Fujimoto, Yuri, Gaut, Joseph, Gewin, Leslie, Hansen, Pernille B.L., He, John Cijiang, Hughes, Jeremy, Inagi, Reiko, Jenkinson, Celia, Jha, Vivekanand, Kato, Mikio, Kelly, Darren, Kopp, Jeffrey, Korstanje, Ron, Mačiulaitis, Romaldas, Mark, Patrick B., Marti, Hans-Peter, McAdoo, Stephen P., Miner, Jeffrey H., Ortiz, Alberto, Parikh, Samir M., Pozzi, Ambra, Romagnani, Paola, Ronco, Pierre, Rovin, Brad H., Saez-Rodriguez, Julio, Saleem, Moin A., Sayer, John A., Shankland, Stuart, Shaw, Andrey S., Soler Romeo, Maria José, Suzuki, Yusuke, Takano, Tomoko, Tang, Sydney, Tolba, Rene, Viel, John, Yamada, Yoshihisa, Yanagita, Motoko, Yokoo, Takashi, Yoshida, Nobuya, Yuen, Darren, Zent, Roy, Zhang, Aihua

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