Author
Centre for Biomedical Network Research on Rare DiseasesORCID 0000-0002-6753-546XUnclaimed ORCID profile · follow to get notified of new preprints
Mutual co-authors in cluster
3 papers
Recent·Most cited
Juan M. Lozano Gil, Lola Rodríguez‐Ruiz, Manuel Palacios…·Víctoriano Mulero
This study shows that tyrosine kinase inhibitors can restore erythropoiesis in Diamond-Blackfan Anemia by inhibiting NLRP1 inflammasome activation.
Miren Lasaga, Paula Rı́o, Amaia Vilas‐Zornoza…·Juan Antonio Bueren
This study shows gene therapy can restore the transcriptional program of hematopoietic stem cells in Fanconi Anemia patients, making them resemble healthy donor cells.
María José Pino-Barrio, Yari A. Gimenez, M Villanueva…·Susana Navarro
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.