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Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I
Authors: Mesa-Núñez, Cristina et al
Journal: Molecular Therapy - Methods & Clinical Development. 2022 Sep
DOI: 10.1016/j.omtm.2022.07.015

Upregulation of NKG2D ligands impairs hematopoietic stem cell function in Fanconi anemia
Authors: Casado, J. A. et al
Journal: J Clin Invest. 2022 Aug
DOI: 10.1172/JCI142842

CIBERER: Spanish national network for research on rare diseases: A highly productive collaborative initiative
Authors: Luque, J. et al
Journal: Clin Genet. 2022 May
DOI: 10.1111/cge.14113.

Gene Editing for Inherited Red Blood Cell Diseases
Authors: Quintana-Bustamante, O. et al
Journal: Frontiers in Physiology. 2022 Mar
DOI: 10.3389/fphys.2022.848261

Comparative proteomic analysis of nuclear and cytoplasmic compartments in human cardiac progenitor cells
Authors: Albericio G. et al
Journal: Sci Rep. 2022 Jan
DOI: 10.1038/s41598-021-03956-8.

Translational research for bone marrow failure patients
Authors: Malouf,C. et al
Journal:  Exp Hematol. 2022 Jan
DOI: 10.1016/j.exphem.2021.11.004.

Generation of NKX2.5(GFP) Reporter Human iPSCs and Differentiation Into Functional Cardiac Fibroblasts
Authors: Lopez-Muneta, L.  et al
Journal: Front Cell Dev Biol. 2022 Jan
DOI: 10.3389/fcell.2021.797927.