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Haematologica|February 29, 2020
NCOA4-mediated ferritinophagy in macrophages is crucial to sustain erythropoiesis in miceAntonella Nai, Maria Rosa Lidonnici, Giorgia Federico, et al.
Proceedings of the National Academy of Sciences of the United States of America|July 25, 2008
In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of beta-thalassemiaAnnarita Miccio, Rossano Cesari, Francesco Lotti, et al.
Cell Stem Cell|May 5, 2023
Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapySamuele Ferrari, Erika Valeri, Anastasia Conti, et al.
Blood|April 29, 2020
Hematopoietic stem cell function in β-thalassemia is impaired and is rescued by targeting the bone marrow nicheAnnamaria Aprile, Alessandro Gulino, Mariangela Storto, et al.
Science Translational Medicine|May 31, 2023
Inhibition of FGF23 is a therapeutic strategy to target hematopoietic stem cell niche defects in β-thalassemiaAnnamaria Aprile, Laura Raggi, Simona Bolamperti, et al.
Stem Cells (Dayton, Ohio)|November 20, 2009
Mesenchymal cells appearing in pancreatic tissue culture are bone marrow-derived stem cells with the capacity to improve transplanted islet functionValeria Sordi, Raffaella Melzi, Alessia Mercalli, et al.
Molecular and Cellular Biology|July 19, 2006
Hypomorphic mutation of the TALE gene Prep1 (pKnox1) causes a major reduction of Pbx and Meis proteins and a pleiotropic embryonic phenotypeElisabetta Ferretti, J Carlos Villaescusa, Patrizia Di Rosa, et al.
Nucleic Acids Research|September 21, 2010
A highly conserved SOX6 double binding site mediates SOX6 gene downregulation in erythroid cellsClaudio Cantu', Vito Grande, Ilaria Alborelli, et al.
American Journal of Hematology|September 8, 2022
Transferrin receptor 2 (Tfr2) genetic deletion makes transfusion-independent a murine model of transfusion-dependent β-thalassemiaSimona Maria Di Modica, Emanuele Tanzi, Violante Olivari, et al.
Molecular Therapy. Nucleic Acids|April 24, 2023
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategiesMégane Brusson, Anne Chalumeau, Pierre Martinucci, et al.
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