RED TERAV - Investigadores

Investigadores

IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Terapia Génica

Inmunoterapia Celular (células CAR)

Terapia de células somáticas y medicina regenerativa

Desarrollos Tecnológicos y Terapéuticos

Investigadores
Publicaciones

Juan Antonio Bueren Roncero

Investigador principal

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Marina I. Garin Ferreira

Investigador principal

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Begoña DÍEZ CABEZAS

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Begoña Sot Sanz

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Guillermo Güenechea Amurrio

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

José Antonio Casado Olea

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

José Carlos Segovia Sanz

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Laura García García

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Laura Ugalde Díaz

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

María Fernández García

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

María García Bravo

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Mariano García Arranz

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Marina Inmaculada Garín Ferreira

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

MERCEDES DESSY RODRIGUEZ

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Mercedes LÓPEZ SANTALLA

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Miriam Hernando Rodríguez

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Nestor Wenceslao Meza

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Óscar Quintana Bustamante

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Paula Río Galdo

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Raúl TORRES RUIZ

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Rebeca Sánchez Domínguez

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Rosa María Yáñez González

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Susana Navarro Ordóñez

Investigador colaborador

Grupo de Investigación: IIS Fundación Jiménez Díaz (CIEMAT) (FJD)

Diez B., Calviño C., Fernández-García M., Rodríguez-Márquez P., Rodríguez-Díaz S., Martínez-Turillas R., Ceballos C., Illarramendi J., Serrano-López J., Miskey C., Navarro-Bailón A., López-Corral L., Llamas P., Redondo M., Sánchez-Guijo F., Rifon J., Alfonso-Piérola A., Ivics Z., Inogés S., López-Díaz de Cerio A., Yanez R., Bueren J.A., Rodríguez-Madoz J.R., Prosper F.. Generation and GMP scale-up of human CAR-T cells using non-viral Sleeping Beauty transposons for B cell malignances. Molecular Therapy Methods and Clinical Development. 13 March 2025. DOI: 10.1016/j.omtm.2025.101425.

Cavazza A., Molina-Estévez F.J., Reyes Á.P., Ronco V., Naseem A., Malenšek Š., Pečan P., Santini A., Heredia P., Aguilar-González A., Boulaiz H., Ni Q., Cortijo-Gutierrez M., Pavlovic K., Herrera I., de la Cerda B., Garcia-Tenorio E.M., Richard E., Granados-Principal S., López-Márquez A., Köber M., Stojanovic M., Vidaković M., Santos-Garcia I., Blázquez L., Haughton E., Yan D., Sánchez-Martín R.M., Mazini L., Aseguinolaza G.G., Miccio A., Rio P., Desviat L.R., Gonçalves M.A.F.V., Peng L., Jiménez-Mallebrera C., Molina F.M., Gupta D., Lainšček D., Luo Y., Benabdellah K.. Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group. Molecular Therapy Nucleic Acids. 11 March 2025. DOI: 10.1016/j.omtn.2025.102457.

Impacto: 6.5. Cuartil: Q1

Peña-Gutiérrez I., Olalla-Sastre B., Río P., Rodríguez-Madoz J.R.. Beyond precision: evaluation of off-target clustered regularly interspaced short palindromic repeats/Cas9–mediated genome editing. Cytotherapy. March 2025. DOI: 10.1016/j.jcyt.2024.10.010.

Impacto: 3.7. Cuartil: Q1

Alarcón-Iniesta H., de Arana G., López-Valls M., Pardo D., Escalona-Noguero C., Rodríguez C., Castellanos M., Cobelo S., Martínez-Ramírez I., Camarero J., Heras S.d.l., de Vicente J., Valera A., Smith W., Bernardo-Gavito R., Cantón R., Galán J.C., Granados D., Miranda R., Guerrero H., Sot B.. CRISPR-associated Plasmonic Colorimeter Method (Ca-PCM): A real-time RGB detection system for gold nanoparticles-based nucleic acid biosensors. Analytica Chimica Acta. 8 February 2025. DOI: 10.1016/j.aca.2024.343601.

Impacto: 5.7. Cuartil: Q1

Ramírez M.J., Pujol R., Minguillón J., Bogliolo M., Persico I., Cavero D., de la Cal A., Río P., Navarro S., Casado J.A., Bailador A., de la Fuente A.S., de Heredia M.L., Almazán F., Antelo M.L., Argilés B., Badell I., Baragaño M., Beléndez C., Bermúdez M., Bernués M., Buedo M.I., Carrasco E., Català A., Costa D., Cuesta I., Fernandez-Delgado R., Fernández-Teijeiro A., Figuera Á., García M., Gondra A., González M., Muñiz S.G., Hernández-Rodríguez I., Ibañez F., Kelleher N.J., Lendínez F., López M., López-Almaraz R., Marchante I., Mendoza C., Nieto J., Ojeda E., Payán-Pernía S., Peláez I., de Soto I.P., Portugal R., Ramos-Arroyo M.A., Regueiro A., Rodríguez A., Rosell J., Saez R., Sánchez J., Sánchez M., Senent M., Tapia M., Trujillo-Quintero J.P., Vagace J.M., Verdú-Amorós J., Verdugo V., Vidales I., Villarreal J., Díaz-de-Heredia C., Sevilla J., Bueren J.A., Surrallés J.. Prognostic significance of mutation type and chromosome fragility in Fanconi anemia. American Journal of Hematology. February 2025. DOI: 10.1002/ajh.27520.

Impacto: 10.1. Cuartil: Q1 (1er decil)

Navarro S., Río P.. All-in-one gene therapy alternative for DBAS. Cell Stem Cell. 2 January 2025. DOI: 10.1016/j.stem.2024.12.003.

Impacto: 19.8. Cuartil: Q1 (1er decil)

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 A.L., Schambach A., Wang W., Schmidt M., Larcher L., Segovia J.C., Yáñez R.M., 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., Catalá A., Barquinero J., Rodríguez-Perales S., Rao G., Surrallés J., Soulier J., Díaz-de-Heredia C., Schwartz J.D., Sevilla J., Bueren J.A.. 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. The Lancet. 21 December 2024. DOI: 10.1016/S0140-6736(24)01880-4.

Impacto: 98.4. Cuartil: Q1 (1er decil)

Olalla B., Río P.. A new breakthrough in genome editing: the story of Casgevy. Cytotherapy. November 2024. DOI: 10.1016/j.jcyt.2024.06.003.

Impacto: 3.7. Cuartil: Q1

Guenechea G., Meza N.W.. The gray boundaries of aberrant shortening of the cellular timekeepers’ edges. EMBO Molecular Medicine. 14 October 2024. DOI: 10.1038/s44321-024-00122-1.

Impacto: 9.0. Cuartil: Q1 (1er decil)

Martínez-Beamonte R., Barranquero C., Gascón S., Mariño J., Arnal C., Estopañán G., Rodriguez-Yoldi M.J., Surra J.C., Martín-Belloso O., Odriozola-Serrano I., Orman I., Segovia J.C., Osada J., Navarro M.Á.. Effect of virgin olive oil as spreadable preparation on atherosclerosis compared to dairy butter in Apoe-deficient mice. Journal of Physiology and Biochemistry. August 2024. DOI: 10.1007/s13105-024-01029-8.

Impacto: 3.7. Cuartil: Q1

Giménez Y., Palacios M., Sánchez-Domínguez R., Zorbas C., Peral J., Puzik A., Ugalde L., Alberquilla O., Villanueva M., Río P., Gálvez E., Da Costa L., Strullu M., Catala A., Ruiz-Llobet A., Segovia J.C., Sevilla J., Strahm B., Niemeyer C.M., Beléndez C., Leblanc T., Lafontaine D.L.J., Bueren J., Navarro S.. Lentivirus-mediated gene therapy corrects ribosomal biogenesis and shows promise for Diamond Blackfan anemia. JCI Insight. 22 May 2024. DOI: 10.1172/jci.insight.171650.

Impacto: 6.3. Cuartil: Q1

Rio-Vilariño A., Cenigaonandia-Campillo A., García-Bautista A., Mateos-Gómez P.A., Schlaepfer M.I., del Puerto-Nevado L., Aguilera O., García-García L., Galeano C., de Miguel I., Serrano-López J., Baños N., Fernández-Aceñero M.J., Lacal J.C., Medico E., García-Foncillas J., Cebrián A.. Correction to: Inhibition of the AURKA/YAP1 axis is a promising therapeutic option for overcoming cetuximab resistance in colorectal cancer stem cells (British Journal of Cancer, (2024), 130, 8, (1402-1413), 10.1038/s41416-024-02649-z). British Journal of Cancer. 3 May 2024. DOI: 10.1038/s41416-024-02667-x.

Impacto: 6.4. Cuartil: Q1

Rio-Vilariño A., Cenigaonandia-Campillo A., García-Bautista A., Mateos-Gómez P.A., Schlaepfer M.I., del Puerto-Nevado L., Aguilera O., García-García L., Galeano C., de Miguel I., Serrano-López J., Baños N., Fernández-Aceñero M.J., Lacal J.C., Medico E., García-Foncillas J., Cebrián A.. Inhibition of the AURKA/YAP1 axis is a promising therapeutic option for overcoming cetuximab resistance in colorectal cancer stem cells. British Journal of Cancer. 3 May 2024. DOI: 10.1038/s41416-024-02649-z.

Impacto: 6.4. Cuartil: Q1

Nieto-Romero V., García-Torralba A., Molinos-Vicente A., Moya F.J., Rodríguez-Perales S., García-Escudero R., Salido E., Segovia J.C., García-Bravo M.. Restored glyoxylate metabolism after AGXT gene correction and direct reprogramming of primary hyperoxaluria type 1 fibroblasts. iScience. 19 April 2024. DOI: 10.1016/j.isci.2024.109530.

Impacto: 4.6. Cuartil: Q1

Garcia Gómez-Heras S., Garcia-Arranz M., Vega-Clemente L., Olivera-Salazar R., Vélez Pinto J.F., Fernández-García M., Guadalajara H., Yáñez R., Garcia-Olmo D.. Study of the Effect of Wild-Type and Transiently Expressing CXCR4 and IL-10 Mesenchymal Stromal Cells in a Mouse Model of Peritonitis. International Journal of Molecular Sciences. January 2024. DOI: 10.3390/ijms25010520.

Impacto: 4.9. Cuartil: Q1

Díez-Alonso L., Falgas A., Arroyo-Ródenas J., Romencín P.A., Martínez A., Gómez-Rosel M., Blanco B., Jiménez-Reinoso A., Mayado A., Pérez-Pons A., Aguilar-Sopeña Ó., Ramírez-Fernández Á., Segura-Tudela A., Perez-Amill L., Tapia-Galisteo A., Domínguez-Alonso C., Rubio-Pérez L., Jara M., Solé F., Hangiu O., Almagro L., Albitre Á., Penela P., Sanz L., Anguita E., Valeri A., García-Ortiz A., Río P., Juan M., Martínez-López J., Roda-Navarro P., Martín-Antonio B., Orfao A., Menéndez P., Bueno C., Álvarez-Vallina L.. Engineered T cells secreting anti-BCMA T cell engagers control multiple myeloma and promote immune memory in vivo. Science Translational Medicine. 2024. DOI: 10.1126/scitranslmed.adg7962.

Impacto: 15.8. Cuartil: Q1 (1er decil)

Lopez-Santalla M., Conde C., Rodriguez-Trillo A., Garin M.I.. Corrigendum: Assessment of mesenchymal stem/stromal cell-based therapy in K/BxN serum transfer-induced arthritis (Frontiers in Immunology, (2022), 13, (943293), 10.3389/fimmu.2022.943293). Frontiers in Immunology. 2024. DOI: 10.3389/fimmu.2024.1384171.

Impacto: 5.7. Cuartil: Q1

Cavazza A., Hendel A., Bak R.O., Rio P., Güell M., Lainšček D., Arechavala-Gomeza V., Peng L., Hapil F.Z., Harvey J., Ortega F.G., Gonzalez-Martinez C., Lederer C.W., Mikkelsen K., Gasiunas G., Kalter N., Gonçalves M.A.F.V., Petersen J., Garanto A., Montoliu L., Maresca M., Seemann S.E., Gorodkin J., Mazini L., Sanchez R., Rodriguez-Madoz J.R., Maldonado-Pérez N., Laura T., Schmueck-Henneresse M., Maccalli C., Grünewald J., Carmona G., Kachamakova-Trojanowska N., Miccio A., Martin F., Turchiano G., Cathomen T., Luo Y., Tsai S.Q., Benabdellah K.. Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi. Molecular Therapy Nucleic Acids. 12 December 2023. DOI: 10.1016/j.omtn.2023.102066.

Impacto: 6.5. Cuartil: Q1

Bueno C., Torres-Ruiz R., Velasco-Hernandez T., Molina O., Petazzi P., Martinez A., Rodriguez V., Vinyoles M., Cantilena S., Williams O., Vega-Garcia N., Rodriguez-Perales S., Segovia J.C., Quintana-Bustamante O., Roy A., Meyer C., Marschalek R., Smith A.L., Milne T.A., Fraga M.F., Tejedor J.R., Menéndez P.. A human genome editing–based MLL::AF4 ALL model recapitulates key cellular and molecular leukemogenic features. Blood. 16 November 2023. DOI: 10.1182/blood.2023020858.

Impacto: 21.0. Cuartil: Q1 (1er decil)

Escalona-Noguero C., Alarcón-Iniesta H., López-Valls M., del Carpio L.P., Piulats J.M., Somoza A., Sot B.. Detection of the Uveal Melanoma-Associated Mutation GNAQ Q209P from Liquid Biopsy Using CRISPR/Cas12a Technology. Analytical Chemistry. 14 November 2023. DOI: 10.1021/acs.analchem.3c03460.

Impacto: 6.7. Cuartil: Q1 (1er decil)

Lasaga M., Río P., Vilas-Zornoza A., Planell N., Navarro S., Alignani D., Fernández-Varas B., Mouzo D., Zubicaray J., Pujol R.M., Nicoletti E., Schwartz J.D., Sevilla J., Ainciburi M., Ullate- Agote A., Surrallés J., Perona R., Sastre L., Prosper F., Gomez-Cabrero D., Bueren J.A.. Gene therapy restores the transcriptional program of hematopoietic stem cells in Fanconi anemia. Haematologica. October 2023. DOI: 10.3324/haematol.2022.282418.

Impacto: 8.2. Cuartil: Q1

Bueren J.A., Auricchio A.. Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases. Human Gene Therapy. 1 September 2023. DOI: 10.1089/hum.2023.152.

Impacto: 3.9. Cuartil: Q1

Miguez A., Gomis C., Vila C., Monguió-Tortajada M., Fernández-García S., Bombau G., Galofré M., García-Bravo M., Sanders P., Fernández-Medina H., Poquet B., Salado-Manzano C., Roura S., Alberch J., Segovia J.C., Allen N.D., Borràs F.E., Canals J.M.. Soluble mutant huntingtin drives early human pathogenesis in Huntington’s disease. Cellular and Molecular Life Sciences. August 2023. DOI: 10.1007/s00018-023-04882-w.

Impacto: 6.2. Cuartil: Q1

Lahera A., Vela-Martín L., López-Nieva P., Salgado R.N., Rodríguez-Perales S., Torres-Ruiz R., López-Lorenzo J.L., Cornago J., Llamas P., Fernández-Navarro P., Sánchez-Domínguez R., Segovia J.C., Sastre I., Cobos-Fernández M., Menéndez P., Santos J., Fernández-Piqueras J., Villa-Morales M.. Comprehensive characterization of a novel, oncogenic and targetable SEPTIN6::ABL2 fusion in T-ALL. British Journal of Haematology. August 2023. DOI: 10.1111/bjh.18901.

Impacto: 5.1. Cuartil: Q1

Fernández-Tabanera E., García-García L., Rodríguez-Martín C., Cervera S.T., González-González L., Robledo C., Josa S., Martínez S., Chapado L., Monzón S., Melero-Fernández de Mera R.M., Alonso J.. CD44 Modulates Cell Migration and Invasion in Ewing Sarcoma Cells. International Journal of Molecular Sciences. July 2023. DOI: 10.3390/ijms241411774.

Impacto: 4.9. Cuartil: Q1

Sánchez-Guijo F., Avendaño-Solá C., Badimón L., Bueren J.A., Canals J.M., Delgadillo J., Delgado J., Eguizábal C., Fernández-Santos M.E., García-Olmo D., González-Aseguinolaza G., Juan M., Martín F., Mata R., Montserrat N., Pérez-Martínez A., Pérez-Simón J.A., Prósper F., Urbano-Ispizua Á., Zapata A.G., Sureda A., Moraleda J.M.. Role of Hospital Exemption in Europe: position paper from the Spanish Advanced Therapy Network (TERAV). Bone Marrow Transplantation. June 2023. DOI: 10.1038/s41409-023-01962-0.

Impacto: 4.5. Cuartil: Q1

Ugalde L., Fañanas S., Torres R., Quintana-Bustamante O., Río P.. CRISPR/Cas9-mediated gene editing. A promising strategy in hematological disorders. Cytotherapy. March 2023. DOI: 10.1016/j.jcyt.2022.11.014.

Impacto: 3.7. Cuartil: Q1

Fox T., Bueren J., Candotti F., Fischer A., Aiuti A., Lankester A., Albert M., Bernardo M.E., Cavazzana M., Ehl S., Neven B., Prevot J., Schambach A., Sevilla J., Thrasher A., Wynn R., Booth C.. Access to gene therapy for rare diseases when commercialization is not fit for purpose. Nature Medicine. March 2023. DOI: 10.1038/s41591-023-02208-8.

Impacto: 58.7. Cuartil: Q1 (1er decil)

Mencattini A., Rizzuto V., Antonelli G., Di Giuseppe D., D'Orazio M., Filippi J., Comes M.C., Casti P., Vives Corrons J.L., Garcia-Bravo M., Segovia J.C., del Mar Mañú-Pereira M., Lopez-Martinez M.J., Samitier J., Martinelli E.. Machine learning microfluidic based platform: Integration of Lab-on-Chip devices and data analysis algorithms for red blood cell plasticity evaluation in Pyruvate Kinase Disease monitoring. Sensors and Actuators A: Physical. 1 March 2023. DOI: 10.1016/j.sna.2023.114187.

Impacto: 4.1. Cuartil: Q1

Hervás-Salcedo R., Fernández-García M., Hernando-Rodríguez M., Suárez-Cabrera C., Bueren J.A., Yáñez R.M.. Improved efficacy of mesenchymal stromal cells stably expressing CXCR4 and IL-10 in a xenogeneic graft versus host disease mouse model. Frontiers in Immunology. 1 February 2023. DOI: 10.3389/fimmu.2023.1062086.

Impacto: 5.7. Cuartil: Q1

Hernández G., Romero-Cortadellas L., Ferrer-Cortès X., Venturi V., Dessy-Rodriguez M., Olivella M., Husami A., de Soto C.P., Morales-Camacho R.M., Villegas A., González-Fernández F.A., Morado M., Kalfa T.A., Quintana-Bustamante O., Pérez-Montero S., Tornador C., Segovia J.C., Sánchez M.. Mutations in the RACGAP1 gene cause autosomal recessive congenital dyserythropoietic anemia type III. Haematologica. February 2023. DOI: 10.3324/haematol.2022.281277.

Impacto: 8.2. Cuartil: Q1

Medina J.P., Bermejo-Álvarez I., Pérez-Baos S., Yáñez R., Fernández-García M., García-Olmo D., Mediero A., Herrero-Beaumont G., Largo R.. Corrigendum: MSC therapy ameliorates experimental gouty arthritis hinting an early COX-2 induction (Front. Immunol., (2023), 14, 1193179, 10.3389/fimmu.2023.1193179). Frontiers in Immunology. 2023. DOI: 10.3389/fimmu.2023.1345777.

Impacto: 5.7. Cuartil: Q1

Medina J.P., Bermejo-Álvarez I., Pérez-Baos S., Yáñez R., Fernández-García M., García-Olmo D., Mediero A., Herrero-Beaumont G., Largo R.. MSC therapy ameliorates experimental gouty arthritis hinting an early COX-2 induction. Frontiers in Immunology. 2023. DOI: 10.3389/fimmu.2023.1193179.

Impacto: 5.7. Cuartil: Q1

Beaverson K.L., Julkowska D., Letinturier M.C.V., Aartsma-Rus A., Austin J., Bueren J., Frost S., Hamamura M., Larkindale J., LaRosa G., Magenheim R., Merico A., Gerdina Pasmooij A.M., Pirard V., Ekow Thomford N., Wada M., Wong-Rieger D., Hartman A.L.. The IRDiRC Chrysalis Task Force: making rare disease research attractive to companies. Therapeutic Advances in Rare Disease. January-December 2023. DOI: 10.1177/26330040231188979.

Fañanas-Baquero S., Morín M., Fernández S., Ojeda-Perez I., Dessy-Rodriguez M., Giurgiu M., Bueren J.A., Moreno-Pelayo M.A., Segovia J.C., Quintana-Bustamante O.. Specific correction of pyruvate kinase deficiency-causing point mutations by CRISPR/Cas9 and single-stranded oligodeoxynucleotides. Frontiers in Genome Editing. 2023. DOI: 10.3389/fgeed.2023.1104666.

Fernández-Gómez P., Pérez de la Lastra Aranda C., Tosat-Bitrián C., Bueso de Barrio J.A., Thompson S., Sot B., Salas G., Somoza Á., Espinosa A., Castellanos M., Palomo V.. Nanomedical research and development in Spain: improving the treatment of diseases from the nanoscale. Frontiers in Bioengineering and Biotechnology. 2023. DOI: 10.3389/fbioe.2023.1191327.

Impacto: 4.3. Cuartil: Q1

García M., Bonafont J., Martínez-Palacios J., Xu R., Turchiano G., Svensson S., Thrasher A.J., Larcher F., Del Rio M., Hernández-Alcoceba R., Garín M.I., Mencía Á., Murillas R.. Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors. Molecular Therapy Methods and Clinical Development. 8 December 2022. DOI: 10.1016/j.omtm.2022.09.005.

García-Bernal D., Blanquer M., Martínez C.M., García-Guillén A.I., García-Hernández A.M., Carmen Algueró M., Yáñez R., Lamana M.L., Moraleda J.M., Sackstein R.. Enforced mesenchymal stem cell tissue colonization counteracts immunopathology. npj Regenerative Medicine. December 2022. DOI: 10.1038/s41536-022-00258-z.

Albericio G., Aguilar S., Torán J.L., Yañez R., López J.A., Vázquez J., Mora C., Bernad A.. Comparative proteomic analysis of nuclear and cytoplasmic compartments in human cardiac progenitor cells. Scientific Reports. December 2022. DOI: 10.1038/s41598-021-03956-8.

Impacto: 4.6. Cuartil: Q2

Siegner S.M., Ugalde L., Clemens A., Garcia-Garcia L., Bueren J.A., Rio P., Karasu M.E., Corn J.E.. Adenine base editing efficiently restores the function of Fanconi anemia hematopoietic stem and progenitor cells. Nature Communications. December 2022. DOI: 10.1038/s41467-022-34479-z.

Impacto: 16.6. Cuartil: Q1 (1er decil)

Lazcanoiturburu N., García-Sáez J., González-Corralejo C., Roncero C., Sanz J., Martín-Rodríguez C., Valdecantos M.P., Martínez-Palacián A., Almalé L., Bragado P., Calero-Pérez S., Fernández A., García-Bravo M., Guerra C., Montoliu L., Segovia J.C., Valverde Á.M., Fabregat I., Herrera B., Sánchez A.. Lack of EGFR catalytic activity in hepatocytes improves liver regeneration following DDC-induced cholestatic injury by promoting a pro-restorative inflammatory response. Journal of Pathology. November 2022. DOI: 10.1002/path.6002.

Impacto: 7.3. Cuartil: Q1 (1er decil)

Lopez-Santalla M., Conde C., Rodriguez-Trillo A., Garin M.I.. Assessment of mesenchymal stem/stromal cell-based therapy in K/BxN serum transfer-induced arthritis. Frontiers in Immunology. 10 October 2022. DOI: 10.3389/fimmu.2022.943293.

Impacto: 7.3. Cuartil: Q1

Mesa-Núñez C., Damián C., Fernández-García M., Díez B., Rao G., Schwartz J.D., Law K.M., Sevilla J., Río P., Yáñez R., Bueren J.A., Almarza E.. Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I. Molecular Therapy Methods and Clinical Development. 8 September 2022. DOI: 10.1016/j.omtm.2022.07.015.

Valeri A., García-Ortiz A., Castellano E., Córdoba L., Maroto-Martín E., Encinas J., Leivas A., Río P., Martínez-López J.. Overcoming tumor resistance mechanisms in CAR-NK cell therapy. Frontiers in Immunology. 3 August 2022. DOI: 10.3389/fimmu.2022.953849.

Impacto: 7.3. Cuartil: Q1

Chacón-Solano E., León C., Carretero M., García M., Sánchez-Domínguez R., Quero F., Méndez-Jiménez E., Bonafont J., Ruiz-Mezcua B., Escámez M.J., Larcher F., del Río M.. Mechanistic interrogation of mutation-independent disease modulators of RDEB identifies the small leucine-rich proteoglycan PRELP as a TGF-β antagonist and inhibitor of fibrosis. Matrix Biology. August 2022. DOI: 10.1016/j.matbio.2022.06.007.

Impacto: 6.9. Cuartil: Q1

Casado J.A., Valeri A., Sanchez-Dominguez R., Vela P., Lopez A., Navarro S., Alberquilla O., Hanenberg H., Pujol R., Segovia J.C., Minguillon J., Surralles J., De Heredia C.D., Sevilla J., Rio P., Bueren J.A.. Upregulation of NKG2D ligands impairs hematopoietic stem cell function in Fanconi anemia. Journal of Clinical Investigation. 1 August 2022. DOI: 10.1172/JCI142842.

Impacto: 15.9. Cuartil: Q1 (1er decil)

Shah A.J., Schwartz J.D., Segovia J.C.. Mitapivat versus Placebo for Pyruvate Kinase Deficiency. New England Journal of Medicine. 30 June 2022. DOI: 10.1056/NEJMc2206275.

Impacto: 158.5. Cuartil: Q1 (1er decil)

Sanchez-Baltasar R., Garcia-Torralba A., Nieto-Romero V., Page A., Molinos-Vicente A., López-Manzaneda S., Ojeda-Pérez I., Ramirez A., Navarro M., Segovia J.C., García-Bravo M.. Efficient and Fast Generation of Relevant Disease Mouse Models by in Vitro and in Vivo Gene Editing of Zygotes. CRISPR Journal. 1 June 2022. DOI: 10.1089/crispr.2022.0013.

Impacto: 3.7. Cuartil: Q2

López-Valls M., Escalona-Noguero C., Rodríguez-Díaz C., Pardo D., Castellanos M., Milán-Rois P., Martínez-Garay C., Coloma R., Abreu M., Cantón R., Galán J.C., Miranda R., Somoza Á., Sot B.. CASCADE: Naked eye-detection of SARS-CoV-2 using Cas13a and gold nanoparticles. Analytica Chimica Acta. 1 May 2022. DOI: 10.1016/j.aca.2022.339749.

Impacto: 6.2. Cuartil: Q1 (1er decil)

Serrano L.J., Garcia‐arranz M., De Pablo‐Moreno J.A., Segovia J.C., Olivera‐salazar R., Garcia‐olmo D., Liras A.. Development and Characterization of a Factor V‐Deficient CRISPR Cell Model for the Correction of Mutations. International Journal of Molecular Sciences. May-2 2022. DOI: 10.3390/ijms23105802.

Impacto: 5.6. Cuartil: Q1

Luque J., Mendes I., Gómez B., Morte B., López de Heredia M., Herreras E., Corrochano V., Bueren J., Gallano P., Artuch R., Fillat C., Pérez-Jurado L.A., Montoliu L., Carracedo Á., Millán J.M., Webb S.M., Palau F., Aguado C., Aguado C., Aguilera P., Albiñana V., Alías L., Almoguera B., Alonso J., Alonso-Ferreira V., Alvarez-Mora M.I., Antiñolo G., Arbones M.L., Arenas J., Arjona E., Armangue T., Armstrong J., Arnedo M., Masó A.A., Avila-Fernandez A., Ayuso C., Badell I., Badenas C., Baeza M.L., Baiget M., Balcells S., Ballesta-Martínez M.J., Barahona M.J., Barros F., Bartoccioni P.C., Pilar Bayona-Bafaluy M., Benito Sanz S., Bernabéu C., Bernal S., Blanco-Kelly F., Blázquez A., Bodoy S., Bogliolo M., Borralleras C., Borrego S., Botella L.M., Bou de Pieri F., Bovolenta P., Bravo-Gil N., Brea A., Bueno-Lozano G., Bueren J., Bustamante A., Caballero T., Camacho-Macorra C., Cámara Y., Camats-Tarruella N., Campos Barros Á., Campuzano V., Cantarero L., Cantó J., Caparrós-Martín J., Cardellach F., Carmona R., Carrera C., Carretero M., Casado M., Casado J., Casasnovas C., Cascón A., Casino P., Castaño L., Castilla-Vallmanya L., Catala A., Luisa Cayuela M., Cediel R., Cervera J., Codina-Solà M., Contreras J., Cormand B., Corominas R., Corral J., Cortés-Rodríguez A., Corton M., Costa-Roger M., Cozar M., Crespo I., Crispi F.. CIBERER: Spanish national network for research on rare diseases: A highly productive collaborative initiative. Clinical Genetics. 01 May-June 2022. DOI: 10.1111/cge.14113.

Impacto: 3.5. Cuartil: Q2

Torra J., Viela F., Megías D., Sot B., Flors C.. Versatile Near-Infrared Super-Resolution Imaging of Amyloid Fibrils with the Fluorogenic Probe CRANAD-2. Chemistry - A European Journal. 1 April 2022. DOI: 10.1002/chem.202200026.

Impacto: 4.3. Cuartil: Q2

Quintana-Bustamante O., Fañanas-Baquero S., Dessy-Rodriguez M., Ojeda-Pérez I., Segovia J.C.. Gene Editing for Inherited Red Blood Cell Diseases. Frontiers in Physiology. 28 March 2022. DOI: 10.3389/fphys.2022.848261.

Impacto: 4.0. Cuartil: Q2

Schwartz J.D., Barcellini W., Grace R.F., Bianchi P., Zanella A., López Lorenzo J.L., Sevilla J., Shah A.J., Glader B., Nicoletti E., Navarro Ordoñez S., Segovia J.C.. Who should be eligible for gene therapy clinical trials in red blood cell pyruvate kinase deficiency (PKD)?: Toward an expanded definition of severe PKD. American Journal of Hematology. 1 March 2022. DOI: 10.1002/ajh.26458.

Impacto: 12.8. Cuartil: Q1 (1er decil)

López-Muneta L., Linares J., Casis O., Martínez-Ibáñez L., González Miqueo A., Bezunartea J., Sanchez de la Nava A.M., Gallego M., Fernández-Santos M.E., Rodriguez-Madoz J.R., Aranguren X.L., Fernández-Avilés F., Segovia J.C., Prósper F., Carvajal-Vergara X.. Generation of NKX2.5<sup>GFP</sup> Reporter Human iPSCs and Differentiation Into Functional Cardiac Fibroblasts. Frontiers in Cell and Developmental Biology. 21 January 2022. DOI: 10.3389/fcell.2021.797927.

Impacto: 5.5. Cuartil: Q1

Rubio C., Avendaño-Ortiz J., Ruiz-Palomares R., Karaivanova V., Alberquilla O., Sánchez-Domínguez R., Casalvilla-Dueñas J.C., Montalbán-Hernández K., Lodewijk I., Rodríguez-Izquierdo M., Munera-Maravilla E., Nunes S.P., Suárez-Cabrera C., Pérez-Crespo M., Martínez V.G., Morales L., Pérez-Escavy M., Alonso-Sánchez M., Lozano-Rodríguez R., Cueto F.J., Aguirre L.A., Guerrero-Ramos F., Paramio J.M., López-Collazo E., Dueñas M.. Toward Tumor Fight and Tumor Microenvironment Remodeling: PBA Induces Cell Cycle Arrest and Reduces Tumor Hybrid Cells’ Pluripotency in Bladder Cancer. Cancers. January-2 2022. DOI: 10.3390/cancers14020287.

Impacto: 5.2. Cuartil: Q2

Malouf C., Loughran S.J., Wilkinson A.C., Shimamura A., Río P.. Translational research for bone marrow failure patients. Experimental Hematology. January 2022. DOI: 10.1016/j.exphem.2021.11.004.

Impacto: 2.6. Cuartil: Q3

Carrascoso-Rubio C., Zittersteijn H.A., Pintado-Berninches L., Fernández-Varas B., Lozano M.L., Manguan-Garcia C., Sastre L., Bueren J.A., Perona R., Guenechea G.. Generation of dyskeratosis congenita-like hematopoietic stem cells through the stable inhibition of DKC1. Stem Cell Research and Therapy. December 2021. DOI: 10.1186/s13287-021-02145-8.

Impacto: 8.079. Cuartil: Q1

Soria-Juan B., Garcia-Arranz M., Llanos Jiménez L., Aparicio C., Gonzalez A., Mahillo Fernandez I., Riera del Moral L., Grochowicz L., Andreu E.J., Marin P., Castellanos G., Moraleda J.M., García-Hernández A.M., Lozano F.S., Sanchez-Guijo F., Villarón E.M., Parra M.L., Yañez R.M., de la Cuesta Diaz A., Tejedo J.R., Bedoya F.J., Martin F., Miralles M., del Rio Sola L., Fernández-Santos M.E., Ligero J.M., Morant F., Hernández-Blasco L., Andreu E., Hmadcha A., Garcia-Olmo D., Soria B.. Efficacy and safety of intramuscular administration of allogeneic adipose tissue derived and expanded mesenchymal stromal cells in diabetic patients with critical limb ischemia with no possibility of revascularization: study protocol for a randomized controlled double-blind phase II clinical trial (The NOMA Trial). Trials. December 2021. DOI: 10.1186/s13063-021-05430-2.

Impacto: 2.728. Cuartil: Q4

Solanes-Casado S., Cebrián A., Rodríguez-Remírez M., Mahíllo I., García-García L., Río-Vilariño A., Baños N., de Cárcer G., Monfort-Vengut A., Castellano V., Fernández-Aceñero M.J., García-Foncillas J., del Puerto-Nevado L.. Overcoming PLK1 inhibitor resistance by targeting mevalonate pathway to impair AXL-TWIST axis in colorectal cancer. Biomedicine and Pharmacotherapy. December 2021. DOI: 10.1016/j.biopha.2021.112347.

Impacto: 7.419. Cuartil: Q1 (1er decil)

Hervás-Salcedo R., Fernández-García M., Hernando-Rodríguez M., Quintana-Bustamante O., Segovia J.C., Alvarez-Silva M., García-Arranz M., Minguez P., del Pozo V., de Alba M.R., García-Olmo D., Ayuso C., Lamana M.L., Bueren J.A., Yañez R.M.. Enhanced anti-inflammatory effects of mesenchymal stromal cells mediated by the transient ectopic expression of CXCR4 and IL10. Stem Cell Research and Therapy. December 2021. DOI: 10.1186/s13287-021-02193-0.

Impacto: 8.079. Cuartil: Q1

Lozano-Pedraza C., Plaza-Mayoral E., Espinosa A., Sot B., Serrano A., Salas G., Blanco-Andujar C., Cotin G., Felder-Flesch D., Begin-Colin S., Teran F.J.. Assessing the parameters modulating optical losses of iron oxide nanoparticles under near infrared irradiation. Nanoscale Advances. 21 November 2021. DOI: 10.1039/d1na00601k.

Impacto: 5.598. Cuartil: Q2

García-García L., Fernández-Tabanera E., Cervera S.T., de Mera R.M.M.F., Josa S., González-González L., Rodríguez-Martín C., Grünewald T.G.P., Alonso J.. The transcription factor fezf1, a direct target of ewsr1-fli1 in ewing sarcoma cells, regulates the expression of neural-specific genes. Cancers. November-2 2021. DOI: 10.3390/cancers13225668.

Impacto: 6.575. Cuartil: Q1

Lopez-Santalla M., Garin M.I.. Improving the efficacy of mesenchymal stem/stromal-based therapy for treatment of inflammatory bowel diseases. Biomedicines. November 2021. DOI: 10.3390/biomedicines9111507.

Impacto: 4.757. Cuartil: Q2

Büning H., Wilson E., Bueren J., Schambach A., Auricchio A.. The European Society of Gene and Cell Therapy: A Nearly 30-Year Endeavor to Make Gene Therapy a Clinical Reality. Human Gene Therapy. October 2021. DOI: 10.1089/hum.2021.29183.hbu.

Impacto: 4.793. Cuartil: Q1

Navarro S., Quintana-Bustamante O., Sanchez-Dominguez R., Lopez-Manzaneda S., Ojeda-Perez I., Garcia-Torralba A., Alberquilla O., Law K., Beard B.C., Bastone A., Rothe M., Villanueva M., Ramirez J.C., Fañanas-Baquero S., Nieto-Romero V., Molinos-Vicente A., Gutierrez S., Nicoletti E., García-Bravo M., Bueren J.A., Schwartz J.D., Segovia J.C.. Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment. Molecular Therapy Methods and Clinical Development. 10 September 2021. DOI: 10.1016/j.omtm.2021.07.006.

Impacto: 5.849. Cuartil: Q2

Sevilla J., Navarro S., Rio P., Sánchez-Domínguez R., Zubicaray J., Gálvez E., Merino E., Sebastián E., Azqueta C., Casado J.A., Segovia J.C., Alberquilla O., Bogliolo M., Román-Rodríguez F.J., Giménez Y., Larcher L., Salgado R., Pujol R.M., Hladun R., Castillo A., Soulier J., Querol S., Fernández J., Schwartz J., García de Andoín N., López R., Catalá A., Surralles J., Díaz-de-Heredia C., Bueren J.A.. Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes. Molecular Therapy Methods and Clinical Development. 10 September 2021. DOI: 10.1016/j.omtm.2021.06.001.

Impacto: 5.849. Cuartil: Q2

Fañanas-Baquero S., Quintana-Bustamante O., Dever D.P., Alberquilla O., Sanchez-Dominguez R., Camarena J., Ojeda-Perez I., Dessy-Rodriguez M., Turk R., Schubert M.S., Lattanzi A., Xu L., Lopez-Lorenzo J.L., Bianchi P., Bueren J.A., Behlke M.A., Porteus M., Segovia J.C.. Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency. Molecular Therapy Methods and Clinical Development. 10 September 2021. DOI: 10.1016/j.omtm.2021.05.001.

Impacto: 5.849. Cuartil: Q2

Leivas A., Valeri A., Córdoba L., García-Ortiz A., Ortiz A., Sánchez-Vega L., Graña-Castro O., Fernández L., Carreño-Tarragona G., Pérez M., Megías D., Paciello M.L., Sánchez-Pina J., Pérez-Martínez A., Lee D.A., Powell D.J., Río P., Martínez-López J.. NKG2D-CAR-transduced natural killer cells efficiently target multiple myeloma. Blood Cancer Journal. August 2021. DOI: 10.1038/s41408-021-00537-w.

Impacto: 9.812. Cuartil: Q1

Ramírez M.J., Pujol R., Trujillo-Quintero J.P., Minguillón J., Bogliolo M., Río P., Navarro S., Casado J.A., Badell I., Carrasco E., Balmaña J., Català A., Sevilla J., Beléndez C., Argilés B., López M., Díaz de Heredia C., Rao G., Nicoletti E., Schwartz J.D., Bueren J.A., Surrallés J.. Natural gene therapy by reverse mosaicism leads to improved hematology in Fanconi anemia patients. American Journal of Hematology. 1 August 2021. DOI: 10.1002/ajh.26234.

Impacto: 13.265. Cuartil: Q1 (1er decil)

Cervera S.T., Rodríguez-Martín C., Fernández-Tabanera E., Melero-Fernández de Mera R.M., Morin M., Fernández-Peñalver S., Iranzo-Martínez M., Amhih-Cardenas J., García-García L., González-González L., Moreno-Pelayo M.A., Alonso J.. Therapeutic potential of ewsr1–fli1 inactivation by crispr/cas9 in ewing sarcoma. Cancers. 1 August 2021. DOI: 10.3390/cancers13153783.

Impacto: 6.575. Cuartil: Q1

Leceta J., Garin M.I., Conde C.. Mechanism of Immunoregulatory Properties of Vasoactive Intestinal Peptide in the K/BxN Mice Model of Autoimmune Arthritis. Frontiers in Immunology. 16 July 2021. DOI: 10.3389/fimmu.2021.701862.

Impacto: 8.786. Cuartil: Q1

Lopez-Santalla M., Bueren J.A., Garin M.I.. Mesenchymal stem/stromal cell-based therapy for the treatment of rheumatoid arthritis: An update on preclinical studies. EBioMedicine. July 2021. DOI: 10.1016/j.ebiom.2021.103427.

Impacto: 11.205. Cuartil: Q1 (1er decil)

Fernandez-Perez R., Lopez-Santalla M., Sánchez-Domínguez R., Alberquilla O., Gutiérrez-Cañas I., Juarranz Y., Bueren J.A., Garin M.I.. Enhanced Susceptibility of Galectin-1 Deficient Mice to Experimental Colitis. Frontiers in Immunology. 28 June 2021. DOI: 10.3389/fimmu.2021.687443.

Impacto: 8.786. Cuartil: Q1

Bonafont J., Mencía A., Chacón-Solano E., Srifa W., Vaidyanathan S., Romano R., Garcia M., Hervás-Salcedo R., Ugalde L., Duarte B., Porteus M.H., Del Rio M., Larcher F., Murillas R.. Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing. Molecular Therapy. 2 June 2021. DOI: 10.1016/j.ymthe.2021.02.019.

Impacto: 12.91. Cuartil: Q1 (1er decil)

Fañanas-Baquero S., Orman I., Aparicio F.B., de Miguel S.B., Merino J.G., Yañez R., Sainz Y.F., Sánchez R., Dessy-Rodríguez M., Alberquilla O., Alfaro D., Zapata A., Bueren J.A., Segovia J.C., Quintana-Bustamante O.. Natural estrogens enhance the engraftment of human hematopoietic stem and progenitor cells in immunodeficient mice. Haematologica. June 2021. DOI: 10.3324/haematol.2019.233924.

Impacto: 11.047. Cuartil: Q1 (1er decil)

Gálvez E., Vallespín E., Arias-Salgado E.G., Sánchez-Valdepeñas C., Giménez Y., Navarro S., Río P., Bogliolo M., Pujol R., Peiró M., Nevado J., Zubicaray J., Sebastián E., Catalá A., Beléndez C., Díaz De Heredia C., Galera A., Badell I., Madero L., Perona R., Sastre L., Surrallés J., Bueren J., Lapunzina P., Sevilla J.. Next-generation Sequencing in Bone Marrow Failure Syndromes and Isolated Cytopenias: Experience of the Spanish Network on Bone Marrow Failure Syndromes. HemaSphere. 9 April 2021. DOI: 10.1097/HS9.0000000000000539.

Impacto: 8.3. Cuartil: Q1

Escalona-Noguero C., López-Valls M., Sot B.. CRISPR/Cas technology as a promising weapon to combat viral infections. BioEssays. April 2021. DOI: 10.1002/bies.202000315.

Impacto: 4.653. Cuartil: Q1

Errazquin R., Sieiro E., Moreno P., Ramirez M.J., Lorz C., Peral J., Ortiz J., Casado J.A., Roman-Rodriguez F.J., Hanenberg H., Río P., Surralles J., Segrelles C., Garcia-Escudero R.. Generating new fanca-deficient hnscc cell lines by genomic editing recapitulates the cellular phenotypes of fanconi anemia. Genes. April 2021. DOI: 10.3390/genes12040548.

Impacto: 4.141. Cuartil: Q2

García-Bernal D., García-Arranz M., Yáñez R.M., Hervás-Salcedo R., Cortés A., Fernández-García M., Hernando-Rodríguez M., Quintana-Bustamante Ó., Bueren J.A., García-Olmo D., Moraleda J.M., Segovia J.C., Zapata A.G.. The Current Status of Mesenchymal Stromal Cells: Controversies, Unresolved Issues and Some Promising Solutions to Improve Their Therapeutic Efficacy. Frontiers in Cell and Developmental Biology. 16 March 2021. DOI: 10.3389/fcell.2021.650664.

Impacto: 6.081. Cuartil: Q1

Pintado-Berninches L., Montes-Worboys A., Manguan-García C., Arias-Salgado E.G., Serrano A., Fernandez-Varas B., Guerrero-López R., Iarriccio L., Planas L., Guenechea G., Egusquiaguirre S.P., Hernandez R.M., Igartua M., Luis Pedraz J., Cortijo J., Sastre L., Molina-Molina M., Perona R.. GSE4-loaded nanoparticles a potential therapy for lung fibrosis that enhances pneumocyte growth, reduces apoptosis and DNA damage. FASEB Journal. March 2021. DOI: 10.1096/fj.202001160RR.

Impacto: 5.834. Cuartil: Q1

ElMaghloob Y., Sot B., McIlwraith M.J., Garcia E., Yelland T., Ismail S., Pfeffer S.R.. ARL3 activation requires the co-GEF BART and effector-mediated turnover. eLife. January 2021. DOI: 10.7554/eLife.64624.

Impacto: 8.713. Cuartil: Q1 (1er decil)

López-Manzaneda S., Ojeda-Pérez I., Zabaleta N., García-Torralba A., Alberquilla O., Torres R., Sánchez-Domínguez R., Torella L., Olivier E., Mountford J., Ramírez J.C., Bueren J.A., González-Aseguinolaza G., Segovia J.C.. In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9. Molecular Therapy Methods and Clinical Development. 11 December 2020. DOI: 10.1016/j.omtm.2020.10.007.

Impacto: 6.698. Cuartil: Q1

Rodríguez-Remírez M., Del Puerto-Nevado L., Fernández-Aceñero M.J., Cruz-Ramos M., García-García L., Solanes S., Molina-Roldán E., García-Foncillas J., Cebrián A.. Targeting Galectin-1 by Aflibercept Strongly Enhances Its Antitumor Effect in Neuroendocrine Carcinomas. Neuroendocrinology. December 2020. DOI: 10.1159/000506163.

Impacto: 4.914. Cuartil: Q2

Pino-Barrio M.J., Giménez Y., Villanueva M., Hildenbeutel M., Sánchez-Dominguez R., Rodríguez-Perales S., Pujol R., Surrallés J., Río P., Cathomen T., Mussolino C., Bueren J.A., Navarro S.. TALEN mediated gene editing in a mouse model of Fanconi anemia. Scientific Reports. 1 December 2020. DOI: 10.1038/s41598-020-63971-z.

Impacto: 4.379. Cuartil: Q1

Martinez-Lage M., Torres-Ruiz R., Puig-Serra P., Moreno-Gaona P., Martin M.C., Moya F.J., Quintana-Bustamante O., Garcia-Silva S., Carcaboso A.M., Petazzi P., Bueno C., Mora J., Peinado H., Segovia J.C., Menendez P., Rodriguez-Perales S.. In vivo CRISPR/Cas9 targeting of fusion oncogenes for selective elimination of cancer cells. Nature Communications. 1 December 2020. DOI: 10.1038/s41467-020-18875-x.

Impacto: 14.919. Cuartil: Q1 (1er decil)

Vázquez A., Fernández-Sevilla L.M., Jiménez E., Pérez-Cabrera D., Yañez R., Subiza J.L., Varas A., Valencia J., Vicente A.. Involvement of Mesenchymal Stem Cells in Oral Mucosal Bacterial Immunotherapy. Frontiers in Immunology. 19 November 2020. DOI: 10.3389/fimmu.2020.567391.

Impacto: 7.561. Cuartil: Q1

Luengo Y., Sot B., Salas G.. Combining Ag and γ-Fe<inf>2</inf>O<inf>3</inf> properties to produce effective antibacterial nanocomposites. Colloids and Surfaces B: Biointerfaces. October 2020. DOI: 10.1016/j.colsurfb.2020.111178.

Impacto: 5.268. Cuartil: Q1

Lopez-Santalla M., Hervas-Salcedo R., Fernandez-Garcia M., Bueren J.A., Garin M.I.. Cell Therapy with Mesenchymal Stem Cells Induces an Innate Immune Memory Response That Attenuates Experimental Colitis in the Long Term. Journal of Crohn's and Colitis. 1 October 2020. DOI: 10.1093/ecco-jcc/jjaa079.

Impacto: 9.071. Cuartil: Q1

Rodriguez-Fornes F., Quintana-Bustamante O., Lozano M.L., Segovia J.C., Bueren J.A., Guenechea G.. Targeted gene therapy into a safe harbor site in human hematopoietic progenitor cells. Gene Therapy. 1 September 2020. DOI: 10.1038/s41434-020-0144-x.

Impacto: 5.25. Cuartil: Q1

Lopez-Santalla M., Fernandez-Perez R., Garin M.I.. Mesenchymal stem/stromal cells for rheumatoid arthritis treatment: An update on clinical applications. Cells. August 2020. DOI: 10.3390/cells9081852.

Impacto: 6.6. Cuartil: Q2

Torra J., Bondia P., Gutierrez-Erlandsson S., Sot B., Flors C.. Long-term STED imaging of amyloid fibers with exchangeable Thioflavin T. Nanoscale. 28 July 2020. DOI: 10.1039/d0nr02961k.

Impacto: 7.79. Cuartil: Q1

Montanuy H., Martínez-Barriocanal A., Casado J.A., Rovirosa L., Ramírez M.J., Nieto R., Carrascoso-Rubio C., Riera P., Gonzalez A., Lerma E., Lasa A., Carreras-Puigvert J., Helleday T., Bueren J.A., Arango D., Minguillon J., Surralles J.. Geﬁtinib and afatinib show potential efﬁcacy for fanconi anemia-related head and neck cancer. Clinical Cancer Research. 15 June 2020. DOI: 10.1158/1078-0432.CCR-19-1625.

Impacto: 12.531. Cuartil: Q1 (1er decil)

Comella-Bolla A., Orlandi J.G., Miguez A., Straccia M., García-Bravo M., Bombau G., Galofré M., Sanders P., Carrere J., Segovia J.C., Blasi J., Allen N.D., Alberch J., Soriano J., Canals J.M.. Human Pluripotent Stem Cell-Derived Neurons Are Functionally Mature In Vitro and Integrate into the Mouse Striatum Following Transplantation. Molecular Neurobiology. 1 June 2020. DOI: 10.1007/s12035-020-01907-4.

Impacto: 5.59. Cuartil: Q1

Segovia C., Jose-Eneriz E.S., Munera-Maravilla E., Martínez-Fernandez M., Garate L., Miranda E., Vilas-Zornoza A., Lodewijk I., Rubio C., Segrelles C., Valcarcel L.V., Rabal O., Casares N., Bernardini A., Suarez-Cabrera C., Lopez-Calderon F.F., Fortes P., Casado J.A., Duenas M., Villacampa F., Lasarte J.J., Guerrero-Ramos F., Velasco G.D., Oyarzabal J., Castellano D., Agirre X., Prosper F., Paramio J.M.. Re: Inhibition of a G9a/DNMT Network Triggers Immune-Mediated Bladder Cancer Regression. Journal of Urology. 1 May 2020. DOI: 10.1097/JU.0000000000000781.01.

Impacto: 7.45. Cuartil: Q1

Nicoletti E., Rao G., Bueren J.A., Río P., Navarro S., Surrallés J., Choi G., Schwartz J.D.. Mosaicism in Fanconi anemia: concise review and evaluation of published cases with focus on clinical course of blood count normalization. Annals of Hematology. 1 May 2020. DOI: 10.1007/s00277-020-03954-2.

Impacto: 3.673. Cuartil: Q2

Mesa-Núñez C., Leon-Rico D., Aldea M., Damián C., Sanchez-Baltasar R., Sanchez R., Alberquilla O., Segovia J.C., Bueren J.A., Almarza E.. The downregulated membrane expression of CD18 in CD34<sup>+</sup> cells defines a primitive population of human hematopoietic stem cells. Stem Cell Research and Therapy. 28 April 2020. DOI: 10.1186/s13287-020-01672-0.

Impacto: 6.832. Cuartil: Q1

Bogliolo M., Pujol R., Aza-Carmona M., Muñoz-Subirana N., Rodriguez-Santiago B., Casado J.A., Rio P., Bauser C., Reina-Castillón J., Lopez-Sanchez M., Gonzalez-Quereda L., Gallano P., Catalá A., Ruiz-Llobet A., Badell I., DIaz-Heredia C., Hladun R., Senent L., Argiles B., Bergua Burgues J.M., Bañez F., Arrizabalaga B., López Almaraz R., Lopez M., Figuera Á., Molinés A., Pérez De Soto I., Hernando I., Muñoz J.A., Del Rosario Marin M., Balmaña J., Stjepanovic N., Carrasco E., Cuesta I., Cosuelo J.M., Regueiro A., Moraleda Jimenez J., Galera-Miñarro A.M., Rosiñol L., Carrió A., Beléndez-Bieler C., Escudero Soto A., Cela E., De La Mata G., Fernández-Delgado R., Garcia-Pardos M.C., Sáez-Villaverde R., Barragaño M., Portugal R., Lendinez F., Hernadez I., Vagace J.M., Tapia M., Nieto J., Garcia M., Gonzalez M., Vicho C., Galvez E., Valiente A., Antelo M.L., Ancliff P., Garcia F., Dopazo J., Sevilla J., Paprotka T., Pérez-Jurado L.A., Bueren J., Surralles J.. Optimised molecular genetic diagnostics of Fanconi anaemia by whole exome sequencing and functional studies. Journal of Medical Genetics. 1 April 2020. DOI: 10.1136/jmedgenet-2019-106249.

Impacto: 6.318. Cuartil: Q1

Brendel C., Rio P., Verhoeyen E.. Humanized mice are precious tools for evaluation of hematopoietic gene therapies and preclinical modeling to move towards a clinical trial. Biochemical Pharmacology. April 2020. DOI: 10.1016/j.bcp.2019.113711.

Impacto: 5.858. Cuartil: Q1

Gallardo J., Escalona-Noguero C., Sot B.. Role of α-Synuclein Regions in Nucleation and Elongation of Amyloid Fiber Assembly. ACS Chemical Neuroscience. 18 March 2020. DOI: 10.1021/acschemneuro.9b00527.

Impacto: 4.418. Cuartil: Q2