Dr. Paula Río, Division of Hematopoietic Innovative Therapies, CIEMAT/CIBERER/ IIS-FJD, UAM (Spain)

Paula Río, Biologist, PhD (UCM) is the head of Bone Marrow Failure Unit at CIEMAT/CIBERER/IIS-FJD, UAM; Madrid, Spain. She has been working in the field of gene therapy of Fanconi anaemia (FA) for more than 20 years, where she has focused on the design of a lentiviral vector for the correction of haematopoietic stem cells in patients with FA and its application in the clinic, with very promising results for patients. She is currently an associate researcher in two gene therapy clinical trials based on the pre-clinical studies developed by the group.

Since 2010 her work has focused on the search for new strategies to correct haematopoietic stem cells from FA patients by gene editing, a field in which she is coordinator of a European project and participates as principal investigator in other national and European projects.

She has published more than 55 papers in the field of gene therapy. She is elected president of the Spanish Society of Gene and Cell Therapy and member of several scientific advisory committees, including the American Fanconi Anemia Research Foundation, the Gene Editing committees of the American Society for Gene Therapy and European Society for Gene and Cell Therapy and the Emerging Gene and Cell Therapies subcommittee from the American Society of Hematology.