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Blood|August 13, 2017
Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+ cells from Fanconi anemia patientsPaula Río, Susana Navarro, Guillermo Guenechea, et al.Nature|June 2, 2009
Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cellsAngel Raya, Ignasi Rodríguez-Pizà, Guillermo Guenechea, et al.Cell Death and Differentiation|January 24, 2019
GSE4 peptide suppresses oxidative and telomere deficiencies in ataxia telangiectasia patient cellsLaura Pintado-Berninches, Beatriz Fernandez-Varas, Carlos Benitez-Buelga, et al.FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology|February 27, 2021
GSE4-loaded nanoparticles a potential therapy for lung fibrosis that enhances pneumocyte growth, reduces apoptosis and DNA damageLaura Pintado-Berninches, Ana Montes-Worboys, Cristina Manguan-García, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|March 6, 2018
Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon VectorsMarta Holstein, Cristina Mesa-Nuñez, Csaba Miskey, et al.Stem Cell Reports|November 10, 2015
Generation of a High Number of Healthy Erythroid Cells from Gene-Edited Pyruvate Kinase Deficiency Patient-Specific Induced Pluripotent Stem CellsZita Garate, Oscar Quintana-Bustamante, Ana M Crane, et al.Nature Medicine|September 11, 2019
Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemiaPaula Río, Susana Navarro, Wei Wang, et al.Pageof 3