Terapias Avanzadas

Responsable

• Juan Bueren (CIEMAT)

IPs

• Marina Inmaculada Garín Ferreira
• José Carlos Segovia Sanz
• Rosa María Yáñez González

Investigación

• Básica
• Clínica

Producción científica

Resumen 2019

Publicaciones 2019

Humanized mice are precious tools for evaluation of hematopoietic gene therapies and preclinical modeling to move towards a clinical trial.

Brendel C, Rio P, Verhoeyen E.

Biochemical pharmacology 2019 Nov 11:113711.

PMID: 31726047

FI: 4,825

Elongated Flexuous Plant Virus-Derived Nanoparticles Functionalized for Autoantibody Detection.

Yuste-Calvo C, López-Santalla M, Zurita L, Cruz-Fernández CF, Sánchez F, Garín MI, Ponz F.

Nanomaterials 2019 Oct 10;9(10). pii: E1438.

PMID: 31658770

FI: 4,034

Gene editing of PKLR gene in human hematopoietic progenitors through 5' and 3' UTR modified TALEN mRNA.

Quintana-Bustamante O, Fañanas-Baquero S, Orman I, Torres R, Duchateau P, Poirot L, Gouble A, Bueren JA, Segovia JC.

PLoS One 2019 Oct 16;14(10):e0223775.

PMID: 31618280

FI: 2,776

NHEJ-Mediated Repair of CRISPR-Cas9-Induced DNA Breaks Efficiently Corrects Mutations in HSPCs from Patients with Fanconi Anemia.

Román-Rodríguez FJ, Ugalde L, Álvarez L, Díez B, Ramírez MJ, Risueño C, Cortón M, Bogliolo M, Bernal S, March F, Ayuso C, Hanenberg H, Sevilla J, Rodríguez-Perales S, Torres-Ruiz R, Surrallés J, Bueren JA, Río P.

Cell Stem Cell Epub 2019 Sep 19. 2019 Nov 7;25(5):607-621.e7.

PMID: 31543367

FI: 21,464

Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia.

Río P, Navarro S, Wang W, Sánchez-Domínguez R, Pujol RM, Segovia JC, Bogliolo M, Merino E, Wu N, Salgado R, Lamana ML, Yañez RM, Casado JA, Giménez Y, Román-Rodríguez FJ, Álvarez L, Alberquilla O, Raimbault A, Guenechea G, Lozano ML, Cerrato L, Hernando M, Gálvez E, Hladun R, Giralt I, Barquinero J, Galy A, García de Andoín N, López R, Catalá A, Schwartz JD, Surrallés J, Soulier J, Schmidt M, Díaz de Heredia C, Sevilla J, Bueren JA.

Nature medicine Epub 2019 Sep 9. 2019 Sep;25(9):1396-1401.

PMID: 31501599

FI: 30,641

Inhibition of a G9a/DNMT network triggers immune-mediated bladder cancer regression.

Segovia C, San José-Enériz E, Munera-Maravilla E, Martínez-Fernández M, Garate L, Miranda E, Vilas-Zornoza A, Lodewijk I, Rubio C, Segrelles C, Valcárcel LV, Rabal O, Casares N, Bernardini A, Suarez-Cabrera C, López-Calderón FF, Fortes P, Casado JA, Dueñas M, Villacampa F, Lasarte JJ, Guerrero-Ramos F, de Velasco G, Oyarzabal J, Castellano D, Agirre X, Prósper F, Paramio JM.

Nature medicine Epub 2019 Jul 3. 2019 Jul;25(7):1073-1081.

PMID: 31270502

FI: 30,641

c-Met Signaling Is Essential for Mouse Adult Liver Progenitor Cells Expansion After Transforming Growth Factor-β-Induced Epithelial-Mesenchymal Transition and Regulates Cell Phenotypic Switch.

Almalé L, García-Álvaro M, Martínez-Palacián A, García-Bravo M, Lazcanoiturburu N, Addante A, Roncero C, Sanz J, de la O López M, Bragado P, Mikulits W, Factor VM, Thorgeirsson SS, Casal JI, Segovia JC, Rial E, Fabregat I, Herrera B, Sánchez A.

Stem cells Epub 2019 Jun 18. 2019 Aug;37(8):1108-1118.

PMID: 31108004

FI: 6,614

Helios modulates the maturation of a CA1 neuronal subpopulation required for spatial memory formation.

Giralt A, Brito V, Pardo M, Rubio SE, Marion-Poll L, Martín-Ibáñez R, Zamora-Moratalla A, Bosch C, Ballesteros JJ, Blasco E, García-Torralba A, Pascual M, Pumarola M, Alberch J, Ginés S, Martín ED, Segovia J, Soriano E, Canals JM.

Experimental neurology Epub 2019 Nov 8. 2020 Jan;323:113095.

PMID: 31712124

FI: 4,562

Advances in the gene therapy of monogenic blood cell diseases.

Bueren JA, Quintana-Bustamante O, Almarza E, Navarro S, Río P, Segovia JC, Guenechea G.

Clinical genetics Epub 2019 Jul 11. 2020 Jan;97(1):89-102.

PMID: 31231794

FI: 4,104

GSE4 peptide suppresses oxidative and telomere deficiencies in ataxia telangiectasia patient cells.

Pintado-Berninches L, Fernandez-Varas B, Benitez-Buelga C, Manguan-Garcia C, Serrano-Benitez A, Iarriccio L, Carrillo J, Guenechea G, Egusquiaguirre SP, Pedraz JL, Hernández RM, Igartua M, Arias-Salgado EG, Cortés-Ledesma F, Sastre L, Perona R.

Cell death and differentiation Epub 2019 Jan 22. 2019 Oct;26(10):1998-2014.

PMID: 30670828

FI: 8,086

Premature aging and cancer development in transgenic mice lacking functional CYLD.

Alameda JP, Ramírez Á, García-Fernández RA, Navarro M, Page A, Segovia JC, Sanchez R, Suárez-Cabrera C, Paramio JM, Bravo A, Fernández-Aceñero MJ, Casanova ML.

Aging-US 2019 Jan 10;11(1):127-159.

PMID: 30631004

FI: 5,515

Proyectos, Contratos y EOs vigentes 2019

Fuentes de financiación públicas de proyectos que se están desarrollando en el IIS-FJD:

Imagen

Patentes

1. Título: "Improvements for performing and facilitating the recovery after hematopoietic stem cell transplantation"

Nº solicitud: EP18382779.9 y PCT/EP2019/079945

Entidades titulares: CIEMAT (80%); CIBER (12%); FIIS-FJD (8%)

Inventores: Oscar Quintana; José Antonio Bueren; José Carlos Segovia.

2. Título: "Pyruvate kinase deficiency (PKD) gene editing treatment method"

Nº solicitud: EP20382568.2

Entidades titulares: CIEMAT (63%), CIBER (12,9 %), IISFJD (14,1 %) Y UNIV. STANFORD (10%)

Inventores: Jose Carlos Segovia, Oscar Quintana, Sara Fañanas, Matthew Porteus.