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- 2022
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Mark
Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34+ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector
(
- Contribution to journal › Article
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Mark
Targeting elevated heme levels to treat a mouse model for Diamond-Blackfan Anemia
(
- Contribution to journal › Article
- 2021
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Mark
Decreased PGC1beta expression results in disrupted human erythroid differentiation, impaired hemoglobinization and cell cycle exit
(
- Contribution to journal › Article
- 2020
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Mark
Lysine-specific demethylase 1A (LSD1) restricts ex vivo propagation of human HSCs and is a target of UM171
(
- Contribution to journal › Article
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Mark
Diamond Blackfan anemia is mediated by hyperactive Nemo-like kinase
(
- Contribution to journal › Article
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Mark
Metformin-induced suppression of Nemo-like kinase improves erythropoiesis in preclinical models of Diamond–Blackfan anemia through induction of miR-26a
(
- Contribution to journal › Article
- 2019
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Mark
A Phenotypic Screening Assay Identifies Modulators of Diamond Blackfan Anemia
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- Contribution to journal › Article
- 2018
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Mark
Small Molecule Screens Identify CDK8-Inhibitors As Candidate Diamond-Blackfan Anemia Drugs
(
- Contribution to journal › Published meeting abstract
- 2017
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Mark
Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia
(
- Contribution to journal › Article
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Mark
Hepatic Leukemia Factor Maintains Quiescence of Hematopoietic Stem Cells and Protects the Stem Cell Pool during Regeneration
(
- Contribution to journal › Article