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Author Details

Mitchell J Weiss
1972
210
71
PMIDPaper TitleJournal TitlePublished Year
37522715ETV6 represses inflammatory response genes and regulates HSPC function during stress hematopoiesis in mice.Blood Adv2023
36413407An RPS19-edited model for Diamond-Blackfan anemia reveals TP53-dependent impairment of hematopoietic stem cell activity.JCI Insight2023
36907613Gene Therapy and Gene Editing for β-Thalassemia.2023
37339583Loss of miR-144/451 alleviates β-thalassemia by stimulating ULK1-mediated autophagy of free α-globin.2023
37400614Potent and uniform fetal hemoglobin induction via base editing.Nat Genet2023
37069266Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice.2023
36865281Multiplex Base Editing to Protect from CD33-Directed Therapy: Implications for Immune and Gene Therapy.2023
36993312Genetic regulation of fetal hemoglobin across global populations.medRxiv2023
36852088Forced enhancer-promoter rewiring to alter gene expression in animal models.2023
35052663Redox-Regulation of α-Globin in Vascular Physiology.Antioxidants2022
36302779Endothelial alpha globin is a nitrite reductase.Nat Commun2022
35650318Publisher Correction: Dual function NFI factors control fetal hemoglobin silencing in adult erythroid cells.Nature Genetics2022
35830837Recent advances in "sickle and niche" research - Tribute to Dr. Paul S Frenette.Stem Cell Reports2022
36224385Activation of γ-globin expression by hypoxia-inducible factor 1α.Nature2022
35934593Effective therapies for sickle cell disease: are we there yet?Trends in Genetics2022
35737751Optimizing haematopoietic stem and progenitor cell apheresis collection from plerixafor-mobilized patients with sickle cell disease.Br J Haematol2022
35618846Dual function NFI factors control fetal hemoglobin silencing in adult erythroid cells.Nature Genetics2022
35793591Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations.Dis Model Mech2022
36459484Modular UBE2H-CTLH E2-E3 complexes regulate erythroid maturation.Elife2022
35021089SCP4-STK35/PDIK1L complex is a dual phospho-catalytic signaling dependency in acute myeloid leukemia.Cell Reports2022
34486392The role of globins in cardiovascular physiology.Physiological Reviews2022
35090172Disrupting the adult globin promoter alleviates promoter competition and reactivates fetal globin gene expression.Blood2022
33301730ZNF410 Uniquely Activates the NuRD Component CHD4 to Silence Fetal Hemoglobin Expression.Molecular Cell2021
34180713RNA-Binding Proteins PCBP1 and PCBP2 Are Critical Determinants of Murine Erythropoiesis.Molecular and Cellular Biology2021
34079130Base editing of haematopoietic stem cells rescues sickle cell disease in mice.Nature2021
33855970Genetic therapies for the first molecular disease.Journal of Clinical Investigation2021
33855784Eltrombopag in children with severe aplastic anemia.Pediatr Blood Cancer2021
34283174A polygenic score for acute vaso-occlusive pain in pediatric sickle cell disease.Blood Adv2021
33958780Single-nucleotide-level mapping of DNA regulatory elements that control fetal hemoglobin expression.Nature Genetics2021
33956057Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease.Blood Adv2021
33846636Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing.Nature Genetics2021
33512425Dynamic CTCF binding directly mediates interactions among cis-regulatory elements essential for hematopoiesis.Blood2021
34137022Generalization of a genetic risk score for time to first albuminuria in children with sickle cell anaemia: SCCRIP cohort study results.Br J Haematol2021
34341563Activation of γ-globin gene expression by GATA1 and NF-Y in hereditary persistence of fetal hemoglobin.Nat Genet2021
33156908FBXO11-mediated proteolysis of BAHD1 relieves PRC2-dependent transcriptional repression in erythropoiesis.Blood2021
32897878BCL11A enhancer-edited hematopoietic stem cells persist in rhesus monkeys without toxicity.J Clin Invest2020
31769130Systematic integration of GATA transcription factors and epigenomes via IDEAS paints the regulatory landscape of hematopoietic cells.IUBMB Life2020
33259127Integrative proteomics reveals principles of dynamic phosphosignaling networks in human erythropoiesis.Mol Syst Biol2020
32983414Erythropoietin regulation of red blood cell production: from bench to bedside and back.F1000Res2020
32915977A novel algorithm comprehensively characterizes human RH genes using whole-genome sequencing data.Blood Adv2020
30995372Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.N Engl J Med2019
31698466Genome editing of HBG1 and HBG2 to induce fetal hemoglobin.Blood Adv2019
29884903Mutation-specific signaling profiles and kinase inhibitor sensitivities of juvenile myelomonocytic leukemia revealed by induced pluripotent stem cells.Leukemia2019
31303423Mechanisms of Progression of Myeloid Preleukemia to Transformed Myeloid Leukemia in Children with Down Syndrome.Cancer Cell2019
31526763Mechanisms of Progression of Myeloid Preleukemia to Transformed Myeloid Leukemia in Children with Down Syndrome.Cancer Cell2019
30971389Regulation of gene expression by miR-144/451 during mouse erythropoiesis.Blood2019
30890594Children with sickle cell anemia and APOL1 genetic variants develop albuminuria early in life.Haematologica2019
29797644Sickle Cell Clinical Research and Intervention Program (SCCRIP): A lifespan cohort study for sickle cell disease progression from the pediatric stage into adulthood.Pediatr Blood Cancer2018
30559313Complexities of genetic diagnosis illustrated by an atypical case of congenital hypoplastic anemia.Cold Spring Harb Mol Case Stud2018
29884740Metformin induces FOXO3-dependent fetal hemoglobin production in human primary erythroid cells.Blood2018
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Boston Children's Hospital and Dana-Farber Cancer Institute, Harvard Medical School
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St. Jude Children's Research Hospital
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St. Jude Children's Research Hospital.
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Johns Hopkins University
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University of Washington
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St. Jude Children's Research Hospital
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St. Jude Children's Research Hospital
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The Wistar Institute
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University of Washington
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Altius Institute for Biomedical Sciences
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Max-Planck-Institute for Molecular Genetics
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Emory University
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