Mosaicism in Fanconi anemia: concise review and evaluation of published cases with focus on clinical course of blood count normalization

por Nicoletti, Eileen, Rao, Gayatri, Bueren, Juan A., Río, Paula, Navarro, Susana, Surrallés, Jordi, Choi, Grace, Schwartz, Jonathan D.
Publicado 2020

Advances in NK cell therapy for brain tumors

por Fares, Jawad, Davis, Zachary B., Rechberger, Julian S., Toll, Stephanie A., Schwartz, Jonathan D., Daniels, David J., Miller, Jeffrey S., Khatua, Soumen
Publicado 2023

Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal

por Moscatelli, Ilana, Almarza, Elena, Schambach, Axel, Ricks, David, Schulz, Ansgar, Herzog, Christopher D., Henriksen, Kim, Askmyr, Maria, Schwartz, Jonathan D., Richter, Johan
Publicado 2020

Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I

por 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
Publicado 2022

Who should be eligible for gene therapy clinical trials in red blood cell pyruvate kinase deficiency (PKD)?: Toward an expanded definition of severe PKD

por Schwartz, Jonathan D., Barcellini, Wilma, Grace, Rachel F., Bianchi, Paola, Zanella, Alberto, López Lorenzo, José Luis, Sevilla, Julián, Shah, Ami J., Glader, Bertil, Nicoletti, Eileen, Navarro Ordoñez, Susana, Segovia, José Carlos
Publicado 2022

Gene therapy restores the transcriptional program of hematopoietic stem cells in Fanconi anemia

por Lasaga, Miren, Río, Paula, Vilas-Zornoza, Amaia, Planell, Nuria, Navarro, Susana, Alignani, Diego, Fernández-Varas, Beatriz, Mouzo, Daniel, Zubicaray, Josune, Pujol, Roser M., Nicoletti, Eileen, Schwartz, Jonathan D., Sevilla, Julián, Ainciburi, Marina, Ullate-Agote, Asier, Surrallés, Jordi, Perona, Rosario, Sastre, Leandro, Prosper, Felipe, Gomez-Cabrero, David, Bueren, Juan A.
Publicado 2023

Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment

por Navarro, Susana, Quintana-Bustamante, Oscar, Sanchez-Dominguez, Rebeca, Lopez-Manzaneda, Sergio, Ojeda-Perez, Isabel, Garcia-Torralba, Aida, Alberquilla, Omaira, Law, Kenneth, Beard, Brian C., Bastone, Antonella, Rothe, Michael, Villanueva, Mariela, Ramirez, Juan C., Fañanas-Baquero, Sara, Nieto-Romero, Virginia, Molinos-Vicente, Andrea, Gutierrez, Sonia, Nicoletti, Eileen, García-Bravo, María, Bueren, Juan A., Schwartz, Jonathan D., Segovia, Jose-Carlos
Publicado 2021