Page 82 - MEMOCiberer014-ENG

RESEARCH GROUPS
Group U710
Programme: Hereditary Cancer and Related Syndromes
Lead Researcher: Bueren, Juan Antonio Group Members
STAFF MEMBERS: Hernando Rodríguez, Miriam | Sánchez Domínguez, Rebeca
ASSOCIATED MEMBERS: Aldea García, Montserrat | Álvarez Ramos, Lara | Casado Olea, José Antonio | Ce- rrato Carrasco, Laura | Chinchón Córdoba, Raquel | Díez Cabezas, Begoña | Fernández García, Raquel María | Garín Ferreira, Marina Inmaculada | Güenechea Amurrio, Guillermo | Lamana Luzuriaga, María Luisa | León Rico, Diego | López Santalla, Mercedes | Lozano Vinagre, María de la Luz | Moleiro San Emeterio, Victoria | Navarro Ordoñez, Susana | Orman Bernal, Israel | Quintana Bustamante, Oscar | Rio Galdo, Paula | Rodríguez Fornes, Fatima | Román Rodríguez, Francisco José | Segovia Sanz, José Carlos | Yañez González, Rosa María
Main lines of research
General research lines of our group are: • Cell and gene therapy for rare diseases. • Stem cell research and applications. • Research of the molecular
and genetic bases of rare diseases affecting the hematopoietic system. • Biology of hematopoietic trans- plantation .
During 2014, our work has focused on developing innovative therapies for aplasias, anemias and congenital immunodeficiencies . Specifically we have worked in the following research areas:
• Gene therapy of patients with Fanconi anemia subtype A. • Applications of cellular reprogramming and directed gene therapy in Fanconi anemia (FA) and erythrocyte pyruvate kinase deficiency (PKD). • Develo- pment of an orphan drug for the treatment of erythrocyte pyruvate kinase deficiency (PKD). • - Preclinical studies for the gene therapy of the leukocyte adhesion deficiency type 1 (LAD-1) .
Our research has been conducted in the context of the National Research Plan and the EU Framework Programme FP7 (PERSIST Project; REGENERATE and EUROFANCOLEN) . For the same purpose, we participate in the International Consortium “Transatlantic Gene Therapy Consortium” promoted by Drs Williams (Harvard Medical School), Thrasher (UCL, London) and Baum (Hannover Medical School) . The interest in conducting translational biomedical research has resulted in the signing of a 5 year period agreement with the Health Research Institute-Fundación Jiménez Díaz (January 2014-December 2017), in which we created a Joint Unit for Advanced Therapies CIEMAT / IIS-Fundación Jiménez Díaz to give clinical applicability to our gene and cell therapy studies .
82 CIBERER » Annual report 2014