Paula Rı́o

This study shows gene therapy can restore the transcriptional program of hematopoietic stem cells in Fanconi Anemia patients, making them resemble healthy donor cells.

This study demonstrates that adenine base editing can restore FANCA function in FA patient cells without causing DNA breaks, showing promise for therapeutic applications.

This paper demonstrates efficient gene editing in FA mouse models using TALENs and a donor template at a safe harbor locus, achieving phenotypic correction of FA cells.