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Animal models of Diamond-Blackfan anemia: updates and challenges
Haematologica (Roma), 2023-05, Vol.108 (5), p.1222
2023
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Accurate Identification of Hemoglobin Variants By Top-Down Protein Analysis Using Capillary Electrophoresis-High-Resolution Mass Spectrometry
Blood, 2022-11, Vol.140 (Supplement 1), p.5384-5386
2022
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The Serine Threonine Kinase Inhibitor Ots-167 Improves Erythropoiesis through Suppression of Nlk Activity in Diamond Blackfan Anemia Models
Blood, 2021-11, Vol.138 (Supplement 1), p.2179-2179
2021
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Nutritional Supplements, Ginseng and Leucine, Increase Erythropoiesis in Diamond Blackfan Anemia Models through Inhibition of Nemo-like Kinase
Blood, 2021-11, Vol.138 (Supplement 1), p.1129-1129
2021
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The Launch of Two Sub-Studies of the Peak Registry, a Global, Longitudinal Study of Pyruvate Kinase Deficiency
Blood, 2022-11, Vol.140 (Supplement 1), p.11041-11042
2022
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Comorbidities and Complications in Pediatric Patients with Pyruvate Kinase Deficiency Enrolled in the Peak Registry
Blood, 2022-11, Vol.140 (Supplement 1), p.5316-5318
2022
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Age of Onset of Complications in Patients with Pyruvate Kinase Deficiency: Analysis from the Peak Registry
Blood, 2022-11, Vol.140 (Supplement 1), p.5323-5325
2022
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Downregulation of SATB1 by miRNAs reduces megakaryocyte/erythroid progenitor expansion in preclinical models of Diamond–Blackfan anemia
Experimental hematology, 2022-07, Vol.111, p.66-78
2022
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Characterizing Iron Overload By Age in Patients Diagnosed with Pyruvate Kinase Deficiency - a Descriptive Analysis from the Peak Registry
Blood, 2021-11, Vol.138 (Supplement 1), p.3074-3074
2021
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SATB1 Chromatin Loops Regulate Megakaryocyte/Erythroid Progenitor Expansion by Facilitating HSP70 and GATA1 Induction
Stem cells (Dayton, Ohio), 2023-06, Vol.41 (6), p.560-569
2023
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Lentiviral-mediated Gene Therapy for Adults and Children with Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study
Blood, 2022-11, Vol.140 (Supplement 1), p.4902-4903
2022
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Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Interim Results of a Global Phase 1 Study for Adult and Pediatric Patients
Blood, 2021-11, Vol.138 (Supplement 1), p.563-563
2021
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Long‐term hematologic and clinical outcomes of splenectomy in children with hereditary spherocytosis and sickle cell disease
Pediatric blood & cancer, 2020-08, Vol.67 (8), p.n/a
2020
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Lentiviral-Mediated Gene Therapy for Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study
Transplantation and cellular therapy, 2023-02, Vol.29 (2), p.S225-S226
2023
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293 - Lentiviral-Mediated Gene Therapy for Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study
Transplantation and cellular therapy, 2023-02, Vol.29 (2), p.S225-S226
2023
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Adrenal and renal corticomedullary junction iron deposition in red cell aplasia
Pediatric radiology, 2010-12, Vol.40 (12), p.1955-1957
2010
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Comparative Effectiveness of Different Types of Splenectomy for Children with Congenital Hemolytic Anemias
The Journal of pediatrics, 2012-04, Vol.160 (4), p.684-689.e13
2012
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