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Research Groups
Group U710
Programme: Inherited Cancer, Haematological & Dermatological Diseases Lead Researcher: Bueren, Juan Antonio
Group members
STAFF MEMBERS: Hernando Rodríguez, Miriam | Sánchez Domínguez, Rebeca.
ASSOCIATED MEMBERS: Aldea García, Montserrat | Almarza Novoa, Elena | Álvarez Ramos, Lara | Casado Olea, José Antonio | Cerrato Carrasco, Laura | Chinchón Córdoba, Raquel | Díez Cabezas, Begoña | Fernández García, Raquel Ma| García Bravo, María | Garín Ferreira, Marina Inmaculada | Güenechea Amurrio, Guillermo | Lamana Luzuriaga, Ma Luisa | León Rico, Diego | López Santalla, Mercedes | Lozano Vinagre, Ma de la Luz | Moleiro San Emeterio, Victoria | Navarro Ordoñez, Susana | Orman Bernal, Israel | Quintana Bustamante, Oscar | Río Galdo, Paula | Rodríguez Fornes, Fatima | Román Rodríguez, Fco. José | Segovia Sanz, José Carlos | Yañez González, Rosa Ma.
Main lines of research
• Gene and cell therapy for rare diseases.
• Research and applications of stem cells.
• Investigation of the molecular and genetic basis of rare diseases affecting the hematopoietic system.
• Biology hematopoietic transplantation.
Our work is focused on the development of innova- tive therapies for rare diseases that primarily affect blood cells. These include congenital bone marrow failures, as well as congenital anemias and immu- nodeficiencies. In particular, along 2015 we have worked in the following research areas:
• Gene therapy, gene editing and cell reprogramming in bone marrow failure sundromes, mainly Fanconi anemia.
• Gene therapy, gene editing and cell reprogramming 58 I Annual report 2015 I CIBERER
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in anemias associated with a deficit in erythrocyte pyruvate kinase (PKD).
Lentiviral gene therapy in the congenital immunodefi- ciency, leukocyte adhesion deficiency type 1 (LAD-1).
Applications of mesenchymal stromal cells in the treatment of rare diseases.
During 2015 our activity has been focused on the de-
velopment of research projects of the National Plan for Research and the 7th Framework Programme of the EU (EUROFANCOLEN Project). Also we have participated in the Transatlantic Gene Therapy Con- sortium, and have established collaborations with biotech companies, with which we hope to achieve important cooperation agreements. Our interests in conducting translational biomedical research is re- flected in the launch of the first National GMP labo-