The downregulated membrane expression of CD18 in CD34 + cells defines a primitive population of human hematopoietic stem cells von Mesa-Núñez, Cristina, Leon-Rico, Diego, Aldea, Montserrat, Damián, Carlos, Sanchez-Baltasar, Raquel, Sanchez, Rebeca, Alberquilla, Omaira, Segovia, José Carlos, Bueren, Juan Antonio, Almarza, Elena

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Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I von Mesa-Núñez, Cristina, Damián, Carlos, Fernández-García, María, Díez, Begoña, Rao, Gayatri, Schwartz, Jonathan D., Law, Ken M., Sevilla, Julián, Río, Paula, Yáñez, Rosa, Bueren, Juan A., Almarza, Elena

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T cells and monocytes: A dangerous liaison in adenosine deaminase 2 deficiency von Mesa‐Nuñez, Cristina, Mortellaro, Alessandra

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A novel genetic variant in PTGS1 affects N‐glycosylation of cyclooxygenase‐1 causing a dominant‐negative effect on platelet function and bleeding diathesis von Palma‐Barqueros, Verónica, Crescente, Marilena, Morena, María Eugenia, Chan, Melissa V., Almarza, Elena, Revilla, Nuria, Bohdan, Natalia, Miñano, Antonia, Padilla, José, Allan, Harriet E., Maffucci, Tania, Edin, Matthew L., Zeldin, Darryl C., Mesa‐Nuñez, Cristina, Damian, Carlos, Marín‐Quilez, Ana, Benito, Rocío, Martínez‐Martínez, Irene, Bermejo, Nuria, Casas‐Aviles, Ignacio, Rodríguez‐Alen, Agustín, González‐Porras, José R., Hernández‐Rivas, Jesús María, Vicente, Vicente, Corral, Javier, Lozano, María L., Warner, Timothy D., Bastida, José María, Rivera, José

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Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors von Holstein, Marta, Mesa-Nuñez, Cristina, Miskey, Csaba, Almarza, Elena, Poletti, Valentina, Schmeer, Marco, Grueso, Esther, Ordóñez Flores, Juan Carlos, Kobelt, Dennis, Walther, Wolfgang, Aneja, Manish K., Geiger, Johannes, Bonig, Halvard B., Izsvák, Zsuzsanna, Schleef, Martin, Rudolph, Carsten, Mavilio, Fulvio, Bueren, Juan A., Guenechea, Guillermo, Ivics, Zoltán

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In vivo imaging of lung inflammation with neutrophil-specific 68Ga nano-radiotracer von Pellico, Juan, Lechuga-Vieco, Ana V., Almarza, Elena, Hidalgo, Andrés, Mesa-Nuñez, Cristina, Fernández-Barahona, Irene, Quintana, Juan A., Bueren, Juan, Enríquez, Jose A., Ruiz-Cabello, Jesús, Herranz, Fernando

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Lentiviral Vector-Mediated Correction of a Mouse Model of Leukocyte Adhesion Deficiency Type I von Leon-Rico, Diego, Aldea, Montserrat, Sanchez-Baltasar, Raquel, Mesa-Nuñez, Cristina, Record, Julien, Burns, Siobhan O, Santilli, Giorgia, Thrasher, Adrian J, Bueren, Juan A, Almarza, Elena

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Early bone marrow alterations in patients with adenosine deaminase 2 deficiency across disease phenotypes and severities von Bulté, Dimitri, Barzaghi, Federica, Mesa-Nuñez, Cristina, Rigamonti, Chiara, Basso-Ricci, Luca, Visconti, Camilla, Crippa, Stefania, Pettinato, Emanuela, Gilioli, Diego, Milani, Raffaella, Quaranta, Pamela, Caorsi, Roberta, Cafaro, Alessia, Cangemi, Giuliana, Lupia, Michela, Schena, Francesca, Grossi, Alice, Di Colo, Giulia, Federici, Silvia, Insalaco, Antonella, De Benedetti, Fabrizio, Marktel, Sarah, Di Micco, Raffaella, Bernardo, Maria Ester, Scala, Serena, Cicalese, Maria Pia, Conti, Francesca, Miano, Maurizio, Gattorno, Marco, Dufour, Carlo, Aiuti, Alessandro, Mortellaro, Alessandra

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Preclinical Evaluation for the Gene Therapy of Patients with Leukocyte Adhesion Deficiency Type I von Almarza, Elena, Mesa-Núñez, Cristina, Damián, Carlos, Fernández-García, María, Díez-Cabezas, Begoña, Schwartz, Jonathan D, Bueren, Juan A

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Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I) von Booth, Claire, Sevilla, Julián, Rao, Gayatri R., Chitty Lopez, Maria, Almarza, Elena, Terrazas, Dayna, Zubicaray, Josune, González Vicent, Marta, Chetty, Kritika, O'Toole, Grainne, Xu-Bayford, Jinhua, Nicoletti, Eileen, Fernandes, Augustine, Kuo, Caroline, de Oliveira, Satiro, Moore, Theodore B., Choi, Grace, Zeini, Miriam, Mesa-Núñez, Cristina, Thrasher, Adrian J., Bueren, Juan A., Schwartz, Jonathan D., Kohn, Donald B.

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A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1 von Kohn, Donald B., Rao, Gayatri R, Almarza, Elena, Terrazas, Dayna, Nicoletti, Eileen, Fernandes, Augustine, Kuo, Caroline, De Oliveira, Satiro N., Moore, Theodore B., Law, Kenneth M, Beard, Brian C, Sevilla, Julian, Mesa-Núñez, Cristina, Bueren, Juan A, Schwartz, Jonathan D

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Lentiviral-Mediated Gene Therapy for the Treatment of Adenosine Deaminase 2 Deficiency von Mortellaro, Alessandra, Zoccolillo, Matteo, Mesa Nuñez, Cristina, Brix, Alessia, Brigida, Immacolata, Barzaghi, Federica, Scala, Serena, Jofra Hernández, Raisa, Basso-Ricci, Luca, Colantuoni, Mariasilvia, Cesaro, Simone, Conti, Francesca, Pession, Andrea, Benedetti, Fabio, Gattorno, Marco, Naldini, Luigi, Cicalese, Maria Pia, Pistocchi, Anna, Aiuti, Alessandro

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A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results von Kohn, Donald B., Booth, Claire, Sevilla, Julián, Rao, Gayatri R, Almarza, Elena, Terrazas, Dayna, Nicoletti, Eileen, Fernandes, Augustine, Kuo, Caroline, de Oliveira, Satiro, Moore, Theodore B., Law, Kenneth M, Beard, Brian C, Choi, Grace, Zeini, Miriam, Mesa-Núñez, Cristina, Thrasher, Adrian J., Bueren, Juan A, Schwartz, Jonathan D

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Gene Therapy for Lad-I Immunodeficiency: Preclinical Evaluation of HSC Transduction Under Optimized GMP-Conditions von Almarza, Elena, Mesa-Núñez, Cristina, Damián, Carlos, Fernández-García, María, Díez-Cabezas, Begoña, Lozano, M Luz, Rio, Paula, Yañez, Rosa M, Law, Ken, Beard, Brian C, Schwartz, Jonathan D, Bueren, Juan A.

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A New Molecular Variant in the PTGS1 Gene That Abrogates Generation of Thromboxane A2 Synthesis and Associates with Platelet Dysfunction and Bleeding von Palma-Barqueros, Veronica, Chan, Melissa, Crescente, Marilena, Bastida, Jose Maria, Almarza, Elena, Suarez Varela, Sara, Mesa-Núñez, Cristina, Casas-Avilés, Ignacio, Bohdan, Natalia, Ruiz-Pividal, Jf, Padilla, Jose, Revilla Calvo, Nuria, Marín-Quilez, A, Martín Izquierdo, Marta, Benito, Rocio, Vicente, Vicente, Bueren, Juan A., Hernández-Rivas, Jesus Maria, González-Porras, José Ramón, Edin, ML, Zelding, DC, Warner, Tim, Rivera, Jose, Lozano, Maria Luisa

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A novel genetic variant in PTGS1 affects N-glycosylation of cyclooxygenase-1 causing a dominant–negative effect von Palma-Barqueros, Verónica, Crescente, Marilena, de la Morena, María Eugenia, Chan, Melissa V, Almarza, Elena, Revilla, Nuria, Bohdan, Natalia, Miñano, Antonia, Padilla, José, Allan, Harriet E, Maffucci, Tania, Edin, Matthew L., Zeldin, Darryl. C., Mesa-Nuñez, Cristina, Damian, Carlos, Marín-Quilez, Ana, Benito, Rocío, Martínez-Martínez, Irene, Bermejo, Nuria, Casas-Aviles, Ignacio, Alen, Agustín Rodríguez, González-Porras, José Ramón, Hernández-Rivas, Jesús María, Vicente, Vicente, Corral, Javier, Lozano, María Luisa, Warner, Timothy D., Bastida, José María, Rivera, José

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3 - Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results from an Ongoing Phase 1/2 Study von Booth, Claire, Sevilla, Julian, Rao, Gayatri, Lopez, Maria Chitty, Almarza, Elena, Terrazas, Dayna, Zubicaray, Josune, Gonzalez-Vicent, Marta, Chetty, Kritika, Xu-Bayford, Jinhua, O’Toole, Grainne, Fernandes, Augustine, Kuo, Caroline Y., De Oliveira, Satiro N., Mesa-Núñez, Cristina, Moore, Theodore B., Nicoletti, Eileen, Choi, Grace, Zeini, Miriam, Thrasher, Adrian J., Bueren, Juan, Schwartz, Jonathan, Kohn, Donald B.

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Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results from an Ongoing Phase 1/2 Study von Booth, Claire, Sevilla, Julian, Rao, Gayatri, Lopez, Maria Chitty, Almarza, Elena, Terrazas, Dayna, Zubicaray, Josune, Gonzalez-Vicent, Marta, Chetty, Kritika, Xu-Bayford, Jinhua, O’Toole, Grainne, Fernandes, Augustine, Kuo, Caroline Y., De Oliveira, Satiro N., Mesa-Núñez, Cristina, Moore, Theodore B., Nicoletti, Eileen, Choi, Grace, Zeini, Miriam, Thrasher, Adrian J., Bueren, Juan, Schwartz, Jonathan, Kohn, Donald B.

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In vivo imaging of lung inflammation with neutrophil-specific 68 Ga nano-radiotracer von Pellico, Juan, Lechuga-Vieco, Ana V, Almarza, Elena, Hidalgo, Andrés, Mesa-Nuñez, Cristina, Fernández-Barahona, Irene, Quintana, Juan A, Bueren, Juan, Enríquez, Jose A, Ruiz-Cabello, Jesús, Herranz, Fernando

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METHODS FOR GENE MODIFICATION OF HEMATOPOIETIC CELLS von SHAH, Gaurav D, RIO GALDO, Paula, SEGOVIA SANZ, Jose Carlos, QUINTANA BUSTAMANTE, Oscar, MESA NUNEZ, Cristina, BUEREN RONCERO, Juan Antonio, BEARD, Brian, ALMARZA NOVOA, Elena, LAW, Kenneth, PATEL, Kinnari, NAVARRO ORDONEZ, Susana

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