General
International Seminar by
Paula Río
Hematopoietic Innovative Therapies Division, CIEMAT, Madrid, Spain
Hematopoietic stem cell gene therapy:
Overcoming challenges in Fanconi anemia
Thursday, November 28, 2024, 15.00h
Piet Borst Auditorium, Netherlands Cancer Institute (NKI)
Hosted by Roderick Beijersbergen (NKI) and Rodrigo Leite de Oliveira (Amsterdam UMC)
For the occasion of the OOA CRISPR Course 2024
Paula Río, Biologist, PhD (UCM) is Head of the Bone Marrow Aplasia Unit at CIEMAT/CIBERER/IIS-FJD, UAM; Madrid. She has been working for more than 20 years in the field of gene therapy for Fanconi anemia, where she has focused on the design of a lentiviral vector for the correction of hematopoietic stem cells in patients with Fanconi anemia and its application in the clinic, with very promising results for patients. She is currently an associate researcher in two clinical gene therapy trials based on the preclinical studies developed by the group.
Since 2010, her work has focused on the search for new strategies to correct hematopoietic stem cells of 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 was elected President of the Spanish Society of Gene and Cell Therapy and is a member of several scientific advisory boards, including the American Fanconi Cancer Foundation, the Gene Editing Committees of the American Society for Gene Therapy and the European Society for Gene and Cell Therapy, and the Bone Marrow Failure Subcommittee of the American Society of Hematology.