Advanced Therapies Group

Summary of activities

The Joint Unit for Advanced Therapies conducts translational research aimed at developing novel gene and cell therapies for congenital and acquired diseases with poor prognoses. These include Fanconi anaemia, paediatric cancers, and pyruvate kinase deficiency, which are currently at advanced stages of clinical development using lentiviral vectors developed within the laboratory, as well as Leukocyte Adhesion Deficiency Type I, for which a gene therapy has recently received FDA approval. Additional gene therapies based on lentiviral vectors and gene-editing technologies for the treatment of haematological and liver diseases are currently being developed at the preclinical stage. Among the cell therapy approaches under investigation, particular emphasis is placed on therapies based on genetically modified mesenchymal stromal cells (MSCs), regulatory T cells (Tregs) and CAR-T cells for the treatment of inflammatory and autoimmune disorders.

The unit is also developing novel combined immunotherapy strategies for the treatment of head and neck squamous cell carcinoma, brain tumours, and other malignancies characterised by specific genetic rearrangements.

Contributions to society

In addition to the development of clinical trials, the results of which have been highly encouraging, the Unit conducts preclinical studies focused on novel therapeutic approaches that have led to the designation of orphan medicinal products and the filing of international patent applications. The Unit also collaborates actively with patient organisations, including the Fanconi Anaemia Foundation, the Fanconi Cancer Foundation, FA Europe, and patient associations for Diamond-Blackfan anaemia and pyruvate kinase deficiency in Spain, France, Germany and the United States, as well as the CRIS Cancer Foundation, among others.

Collaborations

In addition to its participation in the Centre for Biomedical Network Research on Rare Diseases (CIBERER) and the National Network for Advanced Therapies (TERAVplus), the Unit maintains numerous outstanding national and international collaborations with leading academic institution:

Collaborations with National Hospitals for the development of clinical trials: Fundación Jiménez Díaz University Hospital (Dr José Luis López-Lorenzo); Niño Jesús Children’s University Hospital, Madrid (Dr J. Sevilla); Vall d’Hebron University Hospital, Barcelona (Dr C. Díaz de Heredia); Sant Joan de Déu Hospital, Barcelona (Dr A. Catalá); Gregorio Marañón University Hospital, Madrid (Dr C. Beléndez); Clinic University of Navarra (Dr F. Prosper); Virgen de la Arrixaca University Hospital, Murcia (Dr José M. Moraleda, Dr José Rivera); University Hospital of Salamanca Dr Sánchez-Guijo, Dr Muntion); Fundación Jiménez Díaz University Hospital (Dr Pilar Llamas, Dr García-Olmo); Virgen del Rocío University Hospital, Seville (Dr Salvador Payan); and La Paz University Hospital, Madrid (Dr Antonio Pérez Martínez).

Collaborations with International Hospitals for the development of clinical trials: Great Ormond Street Hospital and University College London (UCL), United Kingdom (Dr A. Thrasher); Children's Hospital Los Angeles, USA (Dr D. Cohn); Stanford University School of Medicine and Lucile Packard Children's Hospital Stanford, USA (Dr M.G. Roncarolo); and Ann & Robert H. Lurie Children's Hospital of Chicago, USA (Dr Xiao-Nan Li).

National collaborations for Preclinical Research: Autonomous University of Barcelona and Sant Pau Hospital, Barcelona (Dr J. Surrallés); Centre for Applied Medical Research (CIMA), Pamplona (Dr F. Prosper, Dr Purificación Fortes, Dr Marta Alonso); Alberto Sols Biomedical Research Institute (CSIC-UAM), Madrid (Dr R. Perona and Dr Leandro Sastre); Complutense University of Madrid (Dr Juarranz, Dr A. Vicente); Gregorio Marañón University Hospital, Madrid (Dr R. Correa-Rocha and Dr M. Pion); 12 de Octubre University Hospital, Madrid (Dr Joaquín Martínez-López, Dr Gargini, Dr Luis Álvarez-Vallina); BloodGenetics (Dr Mayka Sánchez); ViveBiotech (Dr Marie Fertin); Pompeu Fabra University (Dr Marc Güell); University Hospital of the Canary Islands, University of La Laguna (Dr Eduardo Salido); University Hospital of Salamanca (Dr Sánchez-Guijo, Dr Muntion); Niño Jesús Children's University Hospital (Dr Manuel Ramírez, Dr África González); IdiPAZ/CNIO Joint Unit of Paediatric Haemato-Oncology (Dr Antonio Pérez Martínez); Carlos III Health Institute (ISCIII), Majadahonda (Dr Javier García Castro, Dr Beatriz Martín-Antonio); University of the Basque Country (Dr J.L. Pedráz); and the University of Murcia/Murcian Institute for Biomedical Research Pascual Parrilla (Dr Javier Corral, Dr María Eugenia de la Morena).

International collaborations for Preclinical Research: Rocket Pharma, USA (Dr Gaurav Shah); Kiji Therapeutics, France-Spain (Dr Miguel Forte); DanausGT Biotechnology (Dr Michael Wang); ETH Zurich (Dr Jacob Corn); Fanconi Cancer Foundation (FCF, USA); H. San Louis (Dr Jean Soulier); Hospital Robert-Debre (Dr Thierry Leblanc); University Medical Centre Freiburg (Dr Charlotte Niemeyer); SR-TIGET de Milán (Dr Luigi Naldini; University of Freiburg, Institute for Transfusion Medicine and Gene Therapy (Dr Toni Cathomen); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlínico (Dr Paola Bianchi); Harvard University (Dr David R. Liu); University of Minnesota (Dr Branden Moriarity); Fred Hutch Cancer Centre (Hans-Peter Kiem); St. Vincent's Institute Fitzroy (Dr Andrew Deans); Stanford University (Dr Matthew Porteus); Utrecht University Hospital (Dr Richard van Wijk); Revertium Therapeutics (Dr Nidhi Jyotsana and Vishal Suri), Vagelos College – Columbia (Maksim Mamonkin).

PhD Theses

Terapias avanzadas-02

List of publications

Outcomes of two distinct T-cell depletion strategies in haploidentical vs unrelated donor pediatric HSCT: A single-center retrospective analysis.

Sisinni L, León-Triana O, Artazcoz MF, Guariento M, Real de Asua IF, Del Castillo YM, Del Campo PL, Sanchez DB, Durán CM, Pedreño JM, Kabat MG, Pérez-Martínez A.

Leuk Res. 2025 Dec 23.160:108150.

PMID: 41447850

FI: 2,2

http://dx.doi.org/10.1016/j.leukres.2025.108150

Selective genome editing of amplified oncogenes triggers immunogenic cell death and tumor remodeling.

Nieto-Sanchez A, Martinez-Lage M, Puig-Serra P, Carpintero S, Alonso-Yanez A, Ojeda-Walczuk P, Ibañez-Navarro M, Pita G, Moya FJ, Moreno C, Martin MC, Alonso R, Nuñez-Torres R, Sanchez-Arevalo Lobo VJ, Alonso-Guirado L, Malats N, Gonzalez-Neira A, Fernandez L, Roda-Navarro P, Torres-Ruiz R, Rodriguez-Perales S.

Mol Cancer. 2025 Dec 22.

PMID: 41430241

FI: 33,9

http://dx.doi.org/10.1186/s12943-025-02542-0

Harnessing DNA barcoding to enhance and sustain polyclonality in gene-edited hematopoietic stem cells.

Ojeda-Perez I, Bustos A, Selvaraj S, Fañanas-Baquero S, Alberquilla-Fernandez O, Serrano LJ, Bonafont J, Garcia-Torralba A, Torres-Ruiz R, Rodriguez-Perales S, Lang V, Trigueros C, Mayo-Garcia R, Quintana-Bustamante O, Porteus M, Sánchez-Dominguez R, Segovia JC.

Mol Ther Methods Clin Dev. 2025 Dec 11.33(4):101615.

PMID: 41245217

FI: 4,7

http://dx.doi.org/10.1016/j.omtm.2025.101615

The repressor Capicua is a barrier to lung tumor development driven by Kras/Trp53 mutations.

Ballesteros-González I, Hernández-Navas I, Brehey O, Lechuga CG, Salmón M, Scotece M, Velasco-Vicente R, Flores-Gómez AA, Cebriá A, Simón-Carrasco L, Jiménez G, Musteanu M, Guerra C, Domínguez O, Caleiras E, Blanco-Aparicio C, Pons T, Ferrer I, Paz-Ares L, Torres-Ruiz R, Rodríguez-Perales S, Barbacid M, Drosten M.

EMBO Mol Med. 2025 Dec.17(12):3377-3406.

PMID: 41219537

FI: 8,3

http://dx.doi.org/10.1038/s44321-025-00326-z

Lentiviral-mediated panErbB CAR-T cell therapy against head and neck squamous cell carcinomas for patients with Fanconi anemia.

López A, Charbonnier D, Vela P, Díez B, Río P, Sánchez R, Alberquilla O, Martín-Antonio B, Minguillón J, Esquinas E, García-Escudero R, Errazquin R, Del Marro S, Pascual A, Lorz C, Juarranz Á, Barahona A, Balmaña J, Maher J, Bueren JA, Casado JA.

Mol Ther Oncol. 2025 Dec 18.33(4):201060.

PMID: 41141364

FI: 5,3

http://dx.doi.org/10.1016/j.omton.2025.201060

Case Report: Eltrombopag in mosaic and gene therapy-treated patients with Fanconi anemia.

Zubicaray J, Iriondo J, Sebastián E, Sanz A, Rio P, Soulier J, San Román S, Uriz JJ, Navarro S, Nicoletti E, Bueren JA, Schwartz JD, Sevilla J.

Front Pediatr. 2025.13:1625751.

PMID: 40861050

FI: 2

http://dx.doi.org/10.3389/fped.2025.1625751

Excessive glycosylation drives thoracic aortic aneurysm formation through integrated stress response.

Rochano-Ortiz A, San Sebastián-Jaraba I, Zamora C, Simó C, García-Cañas V, Martínez-Albaladejo S, Fernández-Gómez MJ, Marcos-Ríos D, Martínez-Núñez P, Martín-Lorenzo M, Velho TR, Ruíz-Rodríguez MJ, Leal-Zafra A, Gabandé-Rodríguez E, Martínez-Martínez S, Guala A, Lorenzo Ó, Blanco-Colio LM, Martín-Ventura JL, Álvarez-Llamas G, Aldamiz-Echevarría G, Teixidó-Tura G, Forteza A, Nistal JF, Redondo JM, Méndez-Barbero N, Mittelbrunn M, Oller J.

Eur Heart J. 2025 Dec 01.46(45):4988-5005.

PMID: 40720766

FI: 35,7

http://dx.doi.org/10.1093/eurheartj/ehaf556

Improving the therapeutic profile of MSCs: Cytokine priming reduces donor-dependent heterogeneity and enhances their immunomodulatory capacity.

Valencia J, Yáñez RM, Muntión S, Fernández-García M, Martín-Rufino JD, Zapata AG, Bueren JA, Vicente Á, Sánchez-Guijo F.

Front Immunol. 2025.16:1473788.

PMID: 40034706

FI: 5,9

http://dx.doi.org/10.3389/fimmu.2025.1473788

Fate and function of exogenously administered mesenchymal stromal cells: current insights and future directions.

Shokoohmand A, Patel NM, Braid L, Dominici M, Heng TSP, Ankrum JA, Barua J, Caicedo A, Creane M, Davies L, Dos Santos CC, Enes SR, English K, Farge D, Fernández-García M, Galipeau J, Kadri N, Khoury M, Kilfeather S, Krampera M, Krasnodembskaya A, Lalu M, Blanc KL, Moll G, Nolta J, O'Kane C, Rocco PRM, Shi Y, Weiss DJ, Viswanathan S.

Cytotherapy. 2025 Nov 15:102007.

PMID: 41420629

FI: 3,2

http://dx.doi.org/10.1016/j.jcyt.2025.102007

GMP-compliant manufacturing of allogeneic peripheral blood CAR-NK cells for the treatment of acute myeloid leukemia.

Córdoba-Espejo L, Sánchez-Vega L, García-Ortiz A, Castellano E, Oliva R, Ortiz-Ruiz A, Gil-Alós D, Del Moral P, Fernández A, Sanjurjo D, López-García S, Perpiña U, Canals JM, Lee DA, Powell DJ, Alonso R, Río P, Paciello ML, Valeri A, Leivas A, Martínez-López J.

Cytotherapy. 2025 Nov 16.28(2):101968.

PMID: 41386018

FI: 3,2

http://dx.doi.org/10.1016/j.jcyt.2025.07.009

Metabolic Rigidity as a Mechanical Barrier to Malaria: Flickering Loss in PKLR-Deficient Erythrocytes.

Hernando-Ospina N, Calero M, Solís G, Azcárate IG, Herráez-Aguilar D, Caselli N, Moleiro LH, Segovia JC, Bautista JM, Monroy F.

FASEB J. 2025 Nov 30.39(22):e71234.

PMID: 41258954

FI: 4,2

http://dx.doi.org/10.1096/fj.202502505R

Base edited skin equivalents with clinical potential for ex vivo correction of recessive dystrophic epidermolysis bullosa.

Bassons-Bascuñana A, Duarte B, Méndez-Jiménez E, Ugalde L, Sanabria-Betrián P, Aussel C, Peña-Gutiérrez I, Olalla-Sastre B, Ammann S, Cathomen T, Del Rio M, Río P, Larcher F, López-Manzaneda S.

Mol Ther. 2025 Nov 17.

PMID: 41254941

FI: 12

http://dx.doi.org/10.1016/j.ymthe.2025.11.023

A presumed mouse parvovirus with overlooked high toxicity for human primitive CD34+ hematopoietic precursors in vitro and in bone marrow-humanized mice.

Segovia JC, Ramírez JC, Río P, Lotufo CM, López-Bueno A, Calvo-López T, Lamana ML, Sandonís V, Balas A, Bautista JM, Abad P, Azcárate IG, Lopez Lorenzo JL, Jalilian FA, Amini R, Kinnunen PM, Jokelainen P, Mohanraj U, Söderlund-Venermo M, Almendral JM.

Microbiol Spectr. 2025 Oct 27.13(12):e0233925.

PMID: 41143413

FI: 3,8

http://dx.doi.org/10.1128/spectrum.02339-25

GPR41 and GPR43 modulate rodent pancreatic α-cell function and growth.

Sánchez-Roncero A, Fernández-Marcelo T, Pérez-Serna AA, Martínez-Oca P, Alberquilla-Fernández O, Sánchez-Domínguez R, Segovia JC, Escrivá F, Gálvez BG, Álvarez C, Marroqui L, Fernández-Millán E.

Life Sci. 2025 Oct 15.379:123913.

PMID: 40818660

FI: 5,1

http://dx.doi.org/10.1016/j.lfs.2025.123913

New insights into thiopurine toxicity: The contribution of rare XDH variants to myelotoxicity.

Tejera-Pérez H, Velasco-Ruiz A, Chaparro M, Núñez-Torres R, Pita G, Alonso MR, Herráez B, Álvarez N, Cortina-Martin C, Puig-Serra P, Torres-Ruiz R, Rodriguez-Perales S, Ricart E, Mañosa M, Márquez L, Martín-Arranz MD, Zabana Y, Guardiola J, Giordano A, Piqueras M, Iglesias E, Calvet X, Lucendo AJ, Busquets D, Ordás I, Calafat M, Domènech E, Gisbert JP, Gonzalez-Neira A, ENEIDA Project of GETECCU.

Biomed Pharmacother. 2025 Oct.191:118446.

PMID: 40812208

FI: 7,5

http://dx.doi.org/10.1016/j.biopha.2025.118446

HSC engraftment is enhanced by combining mobilization with anti-C-Kit and Anti-CD47-based conditioning in hematopoietic transplant.

Ojeda-Perez I, Alberquilla-Fernandez O, García-Torralba A, Lopez-Santalla M, Sánchez-Domínguez R, Segovia JC.

Mol Ther. 2025 Oct 01.33(10):5044-5060.

PMID: 40676835

FI: 12

http://dx.doi.org/10.1016/j.ymthe.2025.07.012

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

Iriondo J, Zubicaray J, Río P, Català 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, Sevilla J.

Eur J Haematol. 2025 Oct.115(4):403-412.

PMID: 40665878

FI: 2,3

http://dx.doi.org/10.1111/ejh.70007

A Novel Early Memory-Enriched Allogeneic NKG2D CAR-T Cell Therapy Based on CRISPR/Cas9 Technology for Solid Tumors.

Aparicio C, Queipo M, Belver M, Espeso F, Serna-Pérez J, Enríquez-Rodríguez L, Acebal C, Martín-Muñoz Á, Valeri A, Leivas A, Río P, Powell DJ, Lobo-Valentín R, Arrabal D, Martínez-López J, Sánchez A, de la Fuente MÁ, González-Vallinas M.

Cancers (Basel). 2025 Sep 30.17(19).

PMID: 41097713

FI: 4,4

http://dx.doi.org/10.3390/cancers17193186

Advancements in Nonviral Gene Editing Strategies for Rare Diseases.

Pérez-Maroto J, Sepp-Lorenzino L, Castaño-Esteban D, Palacios D, Sot B.

Hum Gene Ther. 2025 Sep.36(17-18):1118-1137.

PMID: 40887819

FI: 4

http://dx.doi.org/10.1177/10430342251372056

Risk and Benefit Assessment of Gene Therapy with Lentiviral Vectors and Hematopoietic Stem Cells: The Skysona Case.

Puig-Serra P, Hinckley-Boned A, Tristán-Manzano M, Rio P, Torres-Ruiz R, Rodriguez-Perales S, Martín F.

Hum Gene Ther. 2025 Sep.36(17-18):1159-1172.

PMID: 40887817

FI: 4

http://dx.doi.org/10.1177/10430342251372474

Regulatory Strategies for Accelerating the Translation of Gene Therapies to Clinical Practice: Focus on GMO Considerations.

Delgadillo J, Martin F, Carmona G, Canals JM, Rio P, Eguizabal C, Prosper F, Avendaño-Solá C.

Hum Gene Ther. 2025 Sep.36(17-18):1154-1158.

PMID: 40838843

FI: 4

http://dx.doi.org/10.1177/10430342251372063

Targeting olfactory receptor OR2AT4: An innovative aptamer-based treatment for hair growth promotion.

Mataix M, Illera N, Hidalgo I, Arriba MDC, Martín E, Fernández G, González C, Larcher F, González VM, Rio MD, Jiménez F, Carretero M.

Mol Ther Nucleic Acids. 2025 Sep 09.36(3):102608.

PMID: 40686857

FI: 6,1

http://dx.doi.org/10.1016/j.omtn.2025.102608

Tandem CD19/CD22 CAR T-cells as potential therapy for children and young adults with high-risk r/r B-ALL.

González-Martínez B, Galán-Gómez V, Navarro-Zapata A, Mirones-Aguilar I, Cobo M, Pernas-Sánchez A, Vallejo S, Sánchez-Zapardiel E, León-Triana O, Echecopar C, Martínez-Romera I, Guerra-García P, San Román-Pacheco S, Escudero A, Izquierdo E, Izquierdo M, Naharro S, Martín-Ayuso A, Bareke H, París-Muñoz A, Hu P, Schneider D, Orentas RJ, Minguillón J, Pérez-Martínez A.

EBioMedicine. 2025 Aug.118:105872.

PMID: 40753722

FI: 10,8

http://dx.doi.org/10.1016/j.ebiom.2025.105872

CAR-T cells targeting CCR9 and CD1a for the treatment of T cell acute lymphoblastic leukemia.

Tirado N, Fidyt K, Mansilla MJ, Garcia-Perez A, Martínez-Moreno A, Vinyoles M, Alcain J, García-Peydró M, Roca-Ho H, Fernandez-Fuentes N, Guerrero-Murillo M, Falgàs A, Velasco-Hernandez T, Bueno C, Panelli P, Mulens-Arias V, Apostolov A, Engel P, González EA, Vick B, Jeremias I, Caye-Eude A, Baruchel A, Cavé H, Genescà E, Ribera J, Díaz-Beyá M, Martínez-Sánchez MV, Fuster JL, Escudero López A, Minguillón J, Pérez-Martínez A, Ramírez-Orellana M, Torrebadell M, Díaz VM, Toribio ML, Sánchez-Martínez D, Menéndez P.

J Hematol Oncol. 2025 Jul 01.18(1):69.

PMID: 40598348

FI: 40,4

http://dx.doi.org/10.1186/s13045-025-01715-0

A heterozygous CEBPA mutation disrupting the bZIP domain in a RUNX1 and SRSF2 mutational background causes MDS disease progression.

Almaghrabi R, Alyahyawi Y, Keane P, Mian SA, Habel K, Atkinson A, Ward C, Bayley R, Sargas C, Menendez P, Murphy GJ, Sobahy T, Baghdadi MA, Flemban AF, Kabrah SM, Torres-Ruiz R, Papapetrou EP, Akerman I, Raghavan M, Barragan E, Bonnet D, Bonifer C, Garcia P.

Nat Commun. 2025 Jul 01.16(1):5489.

PMID: 40593493

FI: 15,7

http://dx.doi.org/10.1038/s41467-025-60192-8

An unbiased genomewide screen uncovers 7 genes that drive hematopoietic stem cell fate from mouse embryonic stem cells.

Palma LG, Kartha GM, Maqueda M, Barrero M, Canton E, Iglesias A, González J, Herrero-Molinero P, Torres-Ruiz R, Payer B, Bueno C, Menéndez P, Espinosa L, Bigas A.

Blood. 2025 Jul 17.146(3):328-340.

PMID: 40209065

FI: 23,1

http://dx.doi.org/10.1182/blood.2024027742

The tumor suppressor HNRNPK induces p53-dependent nucleolar stress to drive ribosomopathies.

Aguilar-Garrido P, Velasco-Estévez M, Navarro-Aguadero MÁ, Otero-Sobrino Á, Ibáñez-Navarro M, Marugal MÁ, Hernández-Sánchez M, Malaney P, Rodriguez A, Benitez O, Zhang X, Aitken MJ, Ortiz-Ruiz A, Megías D, Pérez M, Mata G, Gomez J, Lafarga M, Domínguez O, Graña-Castro O, Caleiras E, Ximénez-Embun P, Isasa M, de Andres PJ, Rodríguez-Perales S, Torres-Ruiz R, Revilla E, García-Martín RM, Azorín D, Zubicaray J, Sevilla J, Sirozh O, Lafarga V, Martínez-López J, Post SM, Gallardo M.

J Clin Invest. 2025 Jun 16.135(12).

PMID: 40338663

FI: 13,6

http://dx.doi.org/10.1172/JCI183697

In vivo haemopoietic stem cell gene therapy enabled by postnatal trafficking.

Milani M, Fabiano A, Perez-Rodriguez M, Hernandez RJ, Zecchillo A, Zonari E, Ottonello S, Basso-Ricci L, Canepari C, Volpin M, Iannello V, Capo V, Quaranta P, Seffin L, Russo F, Biffi M, Ormoli L, Brombin C, Carlucci F, Forlino A, Filibian M, Montini E, Scala S, Villa A, Bueren JA, Rio P, Aiuti A, Cantore A, Naldini L.

Nature. 2025 May 28.

PMID: 40437086

FI: 48,5

http://dx.doi.org/10.1038/s41586-025-09070-3

Safe and Efficacious Permanent Removal of Large COL7A1 Exons for Gene Reframing as a Reliable Therapeutic Strategy for Recessive Dystrophic Epidermolysis Bullosa.

López-Manzaneda S, Mencía Á, Bonafont J, Bassons-Bascuñana A, García M, Nyström A, Duarte B, Llames S, Murillas R, Modamio-Hoybjor S, Morín M, Soletto L, Escamez MJ, Moreno-Pelayo MA, Rio MD, Larcher F.

Hum Gene Ther. 2025 May 28.

PMID: 40432605

FI: 4

http://dx.doi.org/10.1089/hum.2024.238

Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1.

Booth C, Sevilla J, Almarza E, Kuo CY, Zubicaray J, Terrazas D, O'Toole G, Chitty-Lopez M, Choi G, Nicoletti E, Long-Boyle J, Fernandes A, Chetty K, De Oliveira S, Banuelos C, Xu-Bayford J, Bastone AL, John-Neek P, Jackson C, Moore TB, Gilmour K, Schambach A, Rothe M, Kasbekar S, Rao GR, Patel K, Shah G, Thrasher AJ, Bueren JA, Schwartz JD, Kohn DB.

N Engl J Med. 2025 May 01.392(17):1698-1709.

PMID: 40305711

FI: 78,5

http://dx.doi.org/10.1056/NEJMoa2407376

CD19 CAR-T cell therapy in a pediatric patient with MDA5+ dermatomyositis and rapidly progressive interstitial lung disease.

París-Muñoz A, Alcobendas-Rueda RM, Verdú-Sánchez C, Udaondo C, Galán-Gómez V, González-Martínez B, Menéndez JJ, Martínez-Romera I, Minguillón J, Pertíñez L, de Manuel-Gómez C, de la Cruz-Benito A, Sanz-Rupérez A, Remesal A, Cámara C, Sánchez-Zapardiel E, Del Pino-Molina L, Gómez-Zamora A, Serrano-Olmedo MG, Español-Rego M, Ruiz de Valbuena M, Climent F, Dorao P, Ríos-Blanco JJ, Andrade JD, Ruiz-Zurita G, Fernández-García MA, Pérez-Martínez A.

Med. 2025 Apr 25:100676.

PMID: 40306284

FI: 11,8

http://dx.doi.org/10.1016/j.medj.2025.100676

Donor insertion into CX3CR1 allows epigenetic modulation of a constitutive promoter on hematopoietic stem cells and its activation upon myeloid differentiation.

Ramos-Hernández I, Fuster-García C, Aguilar-González A, Lozano-Vinagre ML, Guenechea-Amurrio G, Sanchez-Luque FJ, Gonçalves MAFV, Cathomen T, Muñoz P, Molina-Estévez FJ, Martín F.

Nucleic Acids Res. 2025 Apr 22.53(8).

PMID: 40298109

FI: 13,1

http://dx.doi.org/10.1093/nar/gkaf344

Siltuximab for the treatment of early complications after chimeric antigen receptor T-cell therapy for acute lymphoblastic leukemia in children, adolescents, and young adults.

Galán-Gómez V, González-Martínez B, Alonso-Saladrigues A, Rives S, Herrero B, Kwon M, Sánchez-Pina J, Minguillón J, Martínez-Romera I, Mirones Aguilar I, Mestre-Durán C, Casado G, Sánchez-Martín M, Echecopar C, González-Pérez C, León-Triana O, Aguirre-Portolés C, Molinos-Quintana Á, Barba P, Balsalobre P, Pérez-Martínez A.

Exp Hematol Oncol. 2025 Apr 02.14(1):49.

PMID: 40176077

FI: 13,5

http://dx.doi.org/10.1186/s40164-025-00638-3

The impact of a high fat diet and platelet activation on pre-metastatic niche formation.

Hergueta-Redondo M, Sánchez-Redondo S, Hurtado B, Santos V, Pérez-Martínez M, Ximénez-Embún P, McDowell SAC, Mazariegos MS, Mata G, Torres-Ruiz R, Rodríguez-Perales S, Martínez L, Graña-Castro O, Megias D, Quail D, Quintela-Fandino M, Peinado H.

Nat Commun. 2025 Apr 02.16(1):2897.

PMID: 40175356

FI: 15,7

http://dx.doi.org/10.1038/s41467-025-57938-9

Elucidating Immune Cell Changes in Celiac Disease: Revealing New Insights from Spectral Flow Cytometry.

Gómez-Aguililla S, Farrais S, Senosiain C, López-Palacios N, Arau B, Ruiz-Carnicer Á, Sánchez-Domínguez R, Corzo M, Casado I, Pujals M, Bodas A, Sousa C, Núñez C.

Int J Mol Sci. 2025 Mar 21.26(7).

PMID: 40243487

FI: 4,9

http://dx.doi.org/10.3390/ijms26072877

Generation and GMP scale-up of human CAR-T cells using non-viral Sleeping Beauty transposons for B cell malignances.

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 JA, Rodríguez-Madoz JR, Prosper F.

Mol Ther Methods Clin Dev. 2025 Mar 13.33(1):101425.

PMID: 40034423

FI: 4,7

http://dx.doi.org/10.1016/j.omtm.2025.101425

Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group.

Cavazza A, Molina-Estévez FJ, Reyes ÁP, 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 EM, 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 RM, Mazini L, Aseguinolaza GG, Miccio A, Rio P, Desviat LR, Gonçalves MAFV, Peng L, Jiménez-Mallebrera C, Molina FM, Gupta D, Lainšček D, Luo Y, Benabdellah K.

Mol Ther Nucleic Acids. 2025 Mar 11.36(1):102457.

PMID: 39991472

FI: 6,1

http://dx.doi.org/10.1016/j.omtn.2025.102457

Illustrated capsules from the Advanced Course in Platelet Research.

Di Buduo CA, Abbonante V, Malara A, Balduini A, Waller AK, Watson SP, Martin EM, Bridge L, Gibbins J, Hers I, Masson C, Eckly A, Poulter NS, Martínez-García B, Aguila S, Gresele P, Momi S, Amstrong P, Rondina M, Troitiño S, García Á, Bastida JM, Quilez AM, Fuentes AS, Rivera J, Torres-Ruiz R, Ojeda-Walczuk P, Morgan NV, Morena B, Ramaekers K, Tran M, De Wispelaere K, Freson K, Bergmeier W, Greinacher A, He F, Oh S, Di Paola J, Semple JW, Lozano ML, Llamas P, Ward C.

Res Pract Thromb Haemost. 2025 Feb.9(2):102715.

PMID: 40177221

FI: 3,4

http://dx.doi.org/10.1016/j.rpth.2025.102715

Premature ageing of lung alveoli and bone marrow cells from Terc deficient mice with different telomere lengths.

Guerrero-López R, Manguán-García C, Carrascoso-Rubio C, Lozano ML, Toldos-Torres M, García-Castro L, Sánchez-Dominguez R, Alberquilla O, Sánchez-Pérez I, Molina-Molina M, Bueren JA, Guenechea G, Perona R, Sastre L.

Sci Rep. 2025 Feb 19.15(1):6102.

PMID: 39971959

FI: 3,9

http://dx.doi.org/10.1038/s41598-025-90246-2

Targeting ELOVL6 to disrupt c-MYC driven lipid metabolism in pancreatic cancer enhances chemosensitivity.

García García A, Ferrer Aporta M, Vallejo Palma G, Giráldez Trujillo A, Castillo-González R, Calzón Lozano D, Mora Perdiguero A, Muñoz Velasco R, Colina Castro M, de Simone Benito E, Torres-Ruiz R, Rodriguez-Perales S, Dehairs J, Swinnen JV, Garcia-Cañaveras JC, Lahoz A, Montalvo Quirós S, Del Pozo-Rojas C, Luque Rioja C, Monroy F, Herráez-Aguilar D, Alonso Riaño M, Rodríguez Peralto JL, Sánchez-Arévalo Lobo VJ.

Nat Commun. 2025 Feb 16.16(1):1694.

PMID: 39956817

FI: 15,7

http://dx.doi.org/10.1038/s41467-025-56894-8

All-in-one gene therapy alternative for DBAS.

Navarro S, Río P.

Cell Stem Cell. 2025 Jan 02.32(1):4-6.

PMID: 39753111

FI: 20,4

http://dx.doi.org/10.1016/j.stem.2024.12.003