Paula Rio | AIChE

Paula Rio

Head of the Bone Marrow Aplasias Unit

She has been working in the field of Fanconi anemia (FA) gene therapy for more than 20 years. Her studies in gene therapy for Fanconi anemia include the design of a new lentiviral vector that received the Orphan Drug Designation both by the EMA and FDA and the first demonstration that a short transduction protocol allows the engraftment of corrected cells from FA patients in immunodeficient mice. Now she is deeply involved as associated investigator in two different clinical trials focused on the correction of HSCs from Fanconi anemia A patients (NCT03157804 and NCT04248439).

Since 2010 she has been also working in gene editing, first by targeting HSCs from FA patients by Homologous recombination and nowadays by Non-homologous End Joining using the CRISPR/Cas9 system and new editing tools. Nowadays she is the coordinator of a European Joint Program for Rare diseases focused on gene editing strategies for Fanconi anemia (FANEDIT).

She has published 54 articles (H index: 22) and received 6 International Awards including the Young Investigator Award by the European Society of Gene and Cell Therapy (ESGCT) in 2019.

Paula has been a Board Member of the Spanish Society of Cell and Gene Therapy (SETGYC) for the last four years and during the last two years she is the Treasurer of the SETGYC. Since 2020 she is a member of the Immuno-Gene Therapy Committee from the International Society of Cell Therapy (ISCT). She has been recently nominated as a Board member of the Fanconi Anemia Research Fundation (FARF) and actively participates in the Fanconi anemia Gene Therapy working group. Since 2021 she is also member of the Gene Editing committee of the American Society of Gene and Cell Therapy (ASGCT) and member of the subcommittee on Emerging Gene and Cell Therapies of the American Society of Hematology (ASH).