Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome by Rai, Rajeev, Romito, Marianna, Rivers, Elizabeth, Turchiano, Giandomenico, Blattner, Georges, Vetharoy, Winston, Ladon, Dariusz, Andrieux, Geoffroy, Zhang, Fang, Zinicola, Marta, Leon-Rico, Diego, Santilli, Giorgia, Thrasher, Adrian J., Cavazza, Alessia

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Brief Report: Reduced Expression of CD18 Leads to the In Vivo Expansion of Hematopoietic Stem Cells in Mouse Bone Marrow by Leon‐Rico, Diego, Aldea, Montserrat, Sanchez, Rebeca, Segovia, José C., Weiss, Linnea A., Hidalgo, Andrés, Bueren, Juan A., Almarza, Elena

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The downregulated membrane expression of CD18 in CD34 + cells defines a primitive population of human hematopoietic stem cells by 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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Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease by Piras, Giuseppa, Montiel-Equihua, Claudia, Chan, Yee-Ka Agnes, Wantuch, Slawomir, Stuckey, Daniel, Burke, Derek, Prunty, Helen, Phadke, Rahul, Chambers, Darren, Partida-Gaytan, Armando, Leon-Rico, Diego, Panchal, Neelam, Whitmore, Kathryn, Calero, Miguel, Benedetti, Sara, Santilli, Giorgia, Thrasher, Adrian J., Gaspar, H. Bobby

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BCR-JAK2 drives a myeloproliferative neoplasm in transplanted mice by Cuesta-Domínguez, Álvaro, León-Rico, Diego, Álvarez, Lara, Díez, Begoña, Bodega-Mayor, Irene, Baños, Rocío, Martín-Rey, Miguel Ángel, Santos-Roncero, Matilde, Gaspar, María Luisa, Martín-Acosta, Paloma, Almarza, Elena, Bueren, Juan A., Río, Paula, Fernández-Ruiz, Elena

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Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application by Schott, Juliane W., León-Rico, Diego, Ferreira, Carolina B., Buckland, Karen F., Santilli, Giorgia, Armant, Myriam A., Schambach, Axel, Cavazza, Alessia, Thrasher, Adrian J.

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Case report of the first patient treated with ex-vivo autologous haematopoietic stem cell gene therapy transplant in mucopolysaccharidosis type IIIA by Kinsella, Jane Louise, Church, Heather, Ogden, Wendy, Jones, Ceri, Buckland, Karen, Rivat, Christine, Thrasher, Adrian, Snell, Katie, Leon-Rico, Diego, Vaz, Fred, Wijburg, Frits, Jones, Simon, Bigger, Brian, Wynn, Robert

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Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion by Booth, Claire, Kohn, Donald B., Armant, Myriam, Prockop, Susan, Buckland, Karen, Chandra, Sharat, Chandrakasan, Shanmuganathan, Chetty, Kritika, Dansereau, Colleen, de Oliveira, Satiro, Everett, John, León Rico, Diego, London, Wendy B., Xu-Bayford, Jin Hua, Moore, Theodore B., Nagarsheth, Nisha, Parikh, Suhag, Terrazas, Dayna, White, Shanna L, Schambach, Axel, Bushman, Frederic D., Thrasher, Adrian J., Williams, David A., Pai, Sung-Yun

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