Department of Molecular Medicine and Gene Therapy
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Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis.
Blood Feb, 2003 | Pubmed ID: 12393514
Deficiency of ribosomal protein S19 in CD34+ cells generated by siRNA blocks erythroid development and mimics defects seen in Diamond-Blackfan anemia.
Blood Jun, 2005 | Pubmed ID: 15626736
Development of cellular models for ribosomal protein S19 (RPS19)-deficient diamond-blackfan anemia using inducible expression of siRNA against RPS19.
Molecular therapy : the journal of the American Society of Gene Therapy Apr, 2005 | Pubmed ID: 15771965
Human RPS19, the gene mutated in Diamond-Blackfan anemia, encodes a ribosomal protein required for the maturation of 40S ribosomal subunits.
Blood Feb, 2007 | Pubmed ID: 16990592
Diamond-Blackfan anemia: erythropoiesis lost in translation.
Blood Apr, 2007 | Pubmed ID: 17164339
Endoglin is not critical for hematopoietic stem cell engraftment and reconstitution but regulates adult erythroid development.
Stem cells (Dayton, Ohio) Nov, 2007 | Pubmed ID: 17673527
Ribosomal protein S19 deficiency leads to reduced proliferation and increased apoptosis but does not affect terminal erythroid differentiation in a cell line model of Diamond-Blackfan anemia.
Stem cells (Dayton, Ohio) Feb, 2008 | Pubmed ID: 17962699
Gene therapy of Diamond Blackfan anemia CD34(+) cells leads to improved erythroid development and engraftment following transplantation.
Experimental hematology Nov, 2008 | Pubmed ID: 18715690
Mice with ribosomal protein S19 deficiency develop bone marrow failure and symptoms like patients with Diamond-Blackfan anemia.
Blood Dec, 2011 | Pubmed ID: 21989989
From stem cell to red cell: regulation of erythropoiesis at multiple levels by multiple proteins, RNAs, and chromatin modifications.
Blood Dec, 2011 | Pubmed ID: 21998215
Progress towards mechanism-based treatment for Diamond-Blackfan anemia.
TheScientificWorldJournal , 2012 | Pubmed ID: 22619618
Dietary L-leucine improves the anemia in a mouse model for Diamond-Blackfan anemia.
Blood Sep, 2012 | Pubmed ID: 22791294
Fetal hepatic progenitors support long-term expansion of hematopoietic stem cells.
Experimental hematology May, 2013 | Pubmed ID: 23415675
Gene therapy cures the anemia and lethal bone marrow failure in a mouse model of RPS19-deficient Diamond-Blackfan anemia.
Haematologica Dec, 2014 | Pubmed ID: 25216681
Glucocorticoids improve erythroid progenitor maintenance and dampen Trp53 response in a mouse model of Diamond-Blackfan anaemia.
British journal of haematology Nov, 2015 | Pubmed ID: 26305041
Defining the Minimal Factors Required for Erythropoiesis through Direct Lineage Conversion.
Cell reports 06, 2016 | Pubmed ID: 27264182
Direct Conversion of Fibroblasts to Megakaryocyte Progenitors.
Cell reports 10, 2016 | Pubmed ID: 27732845
Direct lineage reprogramming: a useful addition to the blood cell research toolbox.
Expert review of hematology 02, 2017 | Pubmed ID: 27967256
Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia.
Molecular therapy : the journal of the American Society of Gene Therapy 08, 2017 | Pubmed ID: 28434866
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