Page 58 - CIBERER2016-ENG
P. 58
leaD reSearcHer
Bueren, Juan Antonio
Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT)
Avda. Complutense, 40 Edificio, 70a
28040 Madrid
(+34) 91 346 65 18 [email protected] group website
GROUP MEMBERS
Staff members: Damian Burgoa, Carlos | Hernando Rodríguez, Miriam | Sánchez Domínguez, Rebeca Associated members: Aldea García, Montserrat | Almarza Novoa, Elena | Álvarez Ramos, Lara | Casado Olea, José Antonio | Cerrato Carrasco, Laura | Fernández García, Raquel María | García Bravo, María | Garín Ferreira, Marina Inmaculada | Güenechea Amurrio, Guillermo | Lamana Luzuriaga, María Luisa | López Santalla, Mercedes | Lozano Vinagre, María de la Luz | Navarro Ordoñez, Susana | Orman Bernal, Israel | Quintana Bustamante, Oscar | Río Galdo, Paula | Rodríguez Fornes, Fátima | Román Rodríguez, Francisco José | Segovia Sanz, José Carlos | Yañez González, Rosa María
Main lines of research
The main research lines of our group are summarized as follows:
1. Investigation of the molecular and genetic basis of rare diseases affecting the hematopoietic system.
2. Gene Therapy of rare diseases affecting blood cells.
3. Therapy with mesenchymal stromal cells (MSCs) for the treatment of autoimmune and inflammatory pathologies.
Our work is focused on the development of innovative therapies for rare diseases that primarily affect blood cells. These include congenital bone marrow failures, as well as congenital anemias and immunodeficiencies. In particular, along 2016 we have worked in the following research areas:
• Gene therapy in congenital blood cell syndromes
- Lentiviral gene therapy of patients with Fanconi anemia (FA).
- Lentiviral gene therapy in erythrocyte pyruvate kinase deficiency anemia (PKD).
- Gene therapy in immunodeficiency by leukocyte adhesion deficiency type 1 (LAD-I).
- Gene therapy by homologous recombination in Fanconi anemia.
- Gene therapy by homologous recombination of the erythrocyte pyruvate kinase gene.
- Gene therapy in dyskeratosis congenital.
- Advanced therapies in Blackfan Diamond Anemia.
- Reprogramming and gene therapy of cells from patients with primary hyperoxaluria type 1 (HP1).
• Therapy with stromal mesenchymal cells (MSC) in inflammatory and autoimmune pathologies
- Cell therapy for rheumatoid arthritis.
- Cell therapy of inflammatory bowel disease and acute inflammation.
58
PROGRAMMES
Inherited Cancer, Haematological and Dermatological Diseases