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  • Río P, Zubicaray J, Navarro S, Gálvez E, Sánchez-Domínguez R, Nicoletti E, Sebastián E, Rothe M, Pujol R, Bogliolo M, John-Neek P, Bastone AL, Schambach A, Wang W, Schmidt M, Larcher L, Segovia JC, Yáñez RM, Alberquilla O, Díez B, Fernández-García M, García-García L, Ramírez M, Galy A, Lefrere F, Cavazzana M, Leblanc T, García de Andoin N, López-Almaraz R, Català-Temprano A, Barquinero J, Rodríguez-Perales S, Rao G, Surrallés J, Soulier J, Díaz-de-Heredia C, Schwartz JD, Sevilla J and Bueren JA.

    Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials.

    Lancet . 404(10471): 2584-2592. Nº de citas: 15

    [doi:10.1016/S0140-6736(24)01880-4]

  • Pera J, Romero-Moya D, Torralba-Sales E, Andersson R, García-Hernández V, Magallon-Mosella M, Distefano M, Berenguer Balaguer C, Juan Castaños Asins, De Giorgio F, Qiu Z, Iglesias A, Spurk P, Montserrat-Vazquez S, Pasquali L, Liang Z, Català-Temprano A, Florian MC, Wlodarski MW, Bigas A, Marin-Bejar O and Giorgetti A.

    Human iPSCs-based modeling unveils SETBP1 as a driver of chromatin rewiring in GATA2 deficiency.

    NATURE COMMUNICATIONS . 16(1): 10035-10035.

    [doi:10.1038/s41467-025-65806-9]

  • Ghanjati, F, Heck, A, Lebrecht, D, Nöllke, P, Andresen, F, Rotari, N, Schoof, M, Schönung, M, Lipka, DB, Dworzak, M, De Moerloose, B, Sukova, M, Hasle, H, Jahnukainen, K, Malone, A, Masetti, R, Buechner, J, Ussowicz, M, Català-Temprano A, Turkiewicz, D, de Haas, V, Schmugge, M, Erlacher, M, Niemeyer, CM and Flotho, C.

    Epigenetic risk stratification in juvenile myelomonocytic leukemia by targeted methylation analysis of the BMP4 locus

    CLINICAL EPIGENETICS . 17(1): .

    [doi:10.1186/s13148-025-01983-0]

  • Iriondo J, Zubicaray J, Río P, Català-Temprano A, Solsona M, Sanz A, Gómez A, Sebastián E, de la Cruz A, Galán A, Navarro S, Larcher L, de Andoin NG, Uriz JJ, Vagace JM, Gonzalez de Pablo J, Pujol MR, Nicoletti E, Surrallés J, Martín-Prado S, Schwartz JD, Soulier J, Bueren JA and Sevilla J.

    Eltrombopag for Bone Marrow Failure in Fanconi Anemia: Results From the Phase II Clinical Trial FANCREV

    EUROPEAN JOURNAL OF HAEMATOLOGY . 115(4): 403-412.

    [doi:10.1111/ejh.70007]

  • Romero-Moya D, Torralba-Sales E, Calvo C, Marin-Bejar O, Magallon-Mosella M, Distefano-Muñoz ME, Pera J, Juan Castaños Asins, De Giorgio F, Gonzalez J, Iglesias A, Berenguer-Balaguer C, Schilling M, Plass M, Pasquali L, Català-Temprano A, Molina O, Wlodarski MW, Bigas A and Giorgetti A.

    CRISPR-engineered human GATA2 deficiency model uncovers mitotic dysfunction and premature aging in HSPCs, impairing hematopoietic fitness

    Leukemia . : .

    [doi:10.1038/s41375-025-02771-8]

  • Kotmayer L, Kozyra EJ, Kang G, Strahm B, Yoshimi A, Sahoo SS, Pastor VB, Attardi E, Voss R, Vinci L, Kaiser M, Dworzak MN, De Moerloose B, Sukova M, Starý J, Hasle H, Jahnukainen K, Polychronopoulou S, Kállay K, Smith OP, Malone A, Barzilai Birenboim S, Masetti R, Buechner J, Ussowicz M, Kjöllerström P, Bodova I, Kavcic M, Català-Temprano A, Turkiewicz D, Schmugge M, de Haas V, Okhomina VI, Sotomayor C, Catalán P, Wehr C, Salzer U, Germing U, Gattermann N, Bödör C, Gray N, Lewis S, Shimamura A, Giorgetti A, Erlacher M, Niemeyer CM and Wlodarski MW.

    Age-dependent phenotypic and molecular evolution of pediatric MDS arising from GATA2 deficiency

    BLOOD CANCER JOURNAL . 15(1): 121-121. Nº de citas: 3

    [doi:10.1038/s41408-025-01309-6]

  • Romero-Moya D, Pera J, Marin-Bejar O, Torralba-Sales E, Murillo-Sanjuán L, Diaz-de-Heredia C, Montoro J, Rodríguez-Ubreva J, Liquori A, Cervera J, Wlodarski MW, Català-Temprano A and Giorgetti A.

    Multiple phenotypes and epigenetic profiles in a three-generation family history with GATA2 deficiency

    Leukemia . 39(4): 962-966. Nº de citas: 1

    [doi:10.1038/s41375-025-02519-4]

  • Marina Caballero Bellón, Bobillo-Perez S, Català-Temprano A, Alonso-Saladrigues A, Valls-Lafon A, Rives-Solà S and Jordán-García I.

    Role of procalcitonin, C-reactive protein and ferritin in cytokine release syndrome after CAR T-cell therapy in children and young adults

    Biomarkers . 30(2): 115-122. Nº de citas: 2

    [doi:10.1080/1354750X.2025.2454471]

  • Ramírez MJ, Pujol R, Minguillón J, Bogliolo M, Persico I, Cavero D, de la Cal A, Río P, Navarro S, Casado JA, Bailador A, de la Fuente AS, de Heredia ML, Almazán F, Antelo ML, Argilés B, Badell I, Baragaño M, Beléndez C, Bermúdez M, Bernués M, Buedo MI, Carrasco E, Català-Temprano A, Costa D, Cuesta I, Fernandez-Delgado R, Fernández-Teijeiro A, Figuera Á, García M, Gondra A, González M, Muñiz SG, Hernández-Rodríguez I, Ibañez F, Kelleher NJ, Lendínez F, López M, López-Almaraz R, Marchante I, Mendoza C, Nieto J, Ojeda E, Payán-Pernía S, Peláez I, de Soto IP, Portugal R, Ramos-Arroyo MA, Regueiro A, Rodríguez A, Rosell J, Saez R, Sánchez J, Sánchez M, Senent M, Tapia M, Trujillo-Quintero JP, Vagace JM, Verdú-Amorós J, Verdugo V, Vidales I, Villarreal J, Díaz-de-Heredia C, Sevilla J, Bueren JA and Surrallés J.

    Prognostic significance of mutation type and chromosome fragility in Fanconi anemia

    AMERICAN JOURNAL OF HEMATOLOGY . 100(2): 272-284. Nº de citas: 9

    [doi:10.1002/ajh.27520]

  • Theron A, Alonso-Saladrigues A, Dapena JL, López-Duarte M, Diaz de Heredia C, Verdú-Amorós J, Sarrate E, Esperanza-Cebollada E, Cuatrecasas E, Andreu S, Conde N, Sanchez-Sierra N, Isola I, Camós-Guijosa M, Torrebadell-Burriel M, Rives-Solà S and Català-Temprano A.

    Secondary haematological dysplasia after CAR-T-cell therapy for acute lymphoblastic leukaemia in children

    BRITISH JOURNAL OF HAEMATOLOGY . 206(1): 186-194.

    [doi:10.1111/bjh.19862]