Una nueva manera de editar el genoma con la tecnología CRISPR/Cas9 ha permitido corregir el defecto de las células madre de la sangre de pacientes con anemia de Fanconi en estudios preclínicos, ha sido publicado hoy por la revista Cell Stem CellProponen una innovadora estrategia de edición génica para tratar la anemia de Fanconi en un estudio preclínico
The Division of Hematopoietic Innovative Therapies develops an international translational research, where a significant portion of the funding is received from Spanish and European, public and private institutions.
Our studies are aimed at developing new therapies for difficult to treat diseases. Among the diseases to which we dedicate more effort highlights a hereditary disease that causes aplastic anemia, called Fanconi anemia, in which it is launching the first international clinical trial of gene therapy with a lentiviral vector developed in our laboratory. This vector has received the Orphan drug designation from the European Commission. We also investigate the etiology and develop new therapies for other diseases, such as anemias and congenital immunodeficiencies, rheumatoid arthritis, graft versus host disease, and others.
In support to previous studies, studies of the basic biology of hematopoietic and mesenchymal stem cells are made. In particular, we are developing new strategies for gene therapy in different cell types, including induced pluripotent stem cells (iPSCs) that due to their capacity to generate all tissue cells have a great potential for clinical use. Based on the ability of mesenchymal cells to facilitate tissue repair and control the inflammation associated with various diseases, our laboratory is also interested in developing new cellular drugs based on the administration of these cells.
We intend to demonstrate the impact of our work by means of the publication of scientific articles in prestigious international journals, the development of new cellular drugs, and the collaboration for the implementation of new clinical trials of gene and cell therapy to demonstrate the efficacy and safety of advanced therapies in certain diseases difficult to treat.