biostudies-literatureMartelli FAssociazione Italiana per la Ricerca sul CancroNHLBI NIH HHSNational Heart, Lung, and Blood InstituteNational Cancer InstituteNCI NIH HHS720552https://www.ebi.ac.uk/biostudies/studies/S-EPMC8542976biostudies-literatureUnknown12The phenotype of mice carrying the <i>Gata1</i> ^<i>low</i> mutation that decreases expression of <i>Gata1</i> in erythroid cells and megakaryocytes, includes anemia, thrombocytopenia, hematopoietic failure in bone marrow and development of extramedullary hematopoiesis in spleen. With age, these mice develop myelofibrosis, a disease sustained by alterations in stem/progenitor cells and megakaryocytes. This study analyzed the capacity of <i>hGATA1</i> driven by a <i>μLCR</i>/<i>β-globin</i> promoter to rescue the phenotype induced by the <i>Gata1</i> ^<i>low</i> mutation in mice. Double <i>hGATA1</i>/<i>Gata1</i> ^<i>low/0</i> mice were viable at birth with hematocrits greater than those of their <i>Gata1</i> ^<i>low/0</i> littermates but platelet couFrontiers in genetics<i>hGATA1</i> Under the Control of a <i>μLCR/β-Globin</i> Promoter Rescues the Erythroid but Not the Megakaryocytic Phenotype Induced by the <i>Gata1</i> ^<i>low</i> Mutation in Mice.PMC8542976IG17608 AIRC5×1000 project #21267, Metastatic disease: the key unmet need in oncology” to MYNERVA, MYeloidNEoplasms Research Venture AIRC Accelerator Award Project funded through a partnership between Cancer Research UK, Fondazione AIRC and FundacionCientifica de la Asociacion Espanola Contra el CancerP01 CA108671R01 HL134684P01-CA1086711R01-HL134684Verachi PLonetti AMazzarini MVannucchi AMZingariello MMartelli FSarli GMigliaccio ARBacci Bfalse<i>hGATA1</i> Under the Control of a <i>μLCR/β-Globin</i> Promoter Rescues the Erythroid but Not the Megakaryocytic Phenotype Induced by the <i>Gata1</i> ^<i>low</i> Mutation in Mice.The phenotype of mice carrying the <i>Gata1</i> ^<i>low</i> mutation that decreases expression of <i>Gata1</i> in erythroid cells and megakaryocytes, includes anemia, thrombocytopenia, hematopoietic failure in bone marrow and development of extramedullary hematopoiesis in spleen. With age, these mice develop myelofibrosis, a disease sustained by alterations in stem/progenitor cells and megakaryocytes. This study analyzed the capacity of <i>hGATA1</i> driven by a <i>μLCR</i>/<i>β-globin</i> promoter to rescue the phenotype induced by the <i>Gata1</i> ^<i>low</i> mutation in mice. Double <i>hGATA1</i>/<i>Gata1</i> ^<i>low/0</i> mice were viable at birth with hematocrits greater than those of their <i>Gata1</i> ^<i>low/0</i> littermates but platelet cou2021-01-01T00:00:00Z20212025-05-29T20:55:28.098Z2025-05-29T20:55:28.098ZS-EPMC85429763470764010.3389/fgene.2021.720552