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CRISPR/Cas9 Genome Editing of Human-Induced Pluripotent Stem Cells Followed by Granulocytic Differentiation

Methods in molecular biology (Clifton, N.J.), 2020-01, Vol.2115, p.471

2020

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Expanding the genetic landscape of congenital neutropenia: CXCR2 mutations in three families revealed through whole exome sequencing

Haematologica (Roma), 2024-08

2024

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Generation, expansion, and drug treatment of hematopoietic progenitor cells derived from human iPSCs

STAR protocols, 2022-06, Vol.3 (2), p.101400-101400, Article 101400

2022

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Circumventing Mutation to Nix Neutropenia

The New England journal of medicine, 2021-05, Vol.384 (20), p.1956-1958

2021

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Gene Knockout in Hematopoietic Stem and Progenitor Cells Followed by Granulocytic Differentiation

Methods in molecular biology (Clifton, N.J.), 2020-01, Vol.2115, p.455

2020

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Differential transcriptional control of hematopoiesis in congenital and cyclic neutropenia patients harboring ELANE mutations

Haematologica (Roma), 2024-05, Vol.109 (5), p.1393

2024

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Case report: Granulocyte-macrophage colony-stimulating factor sargramostim did not rescue the neutrophil phenotype in two patients with JAGN1-mutant severe congenital neutropenia

Frontiers in immunology, 2024-09, Vol.15, p.1373495

2024

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A unique role of p53 haploinsufficiency or loss in the development of acute myeloid leukemia with FLT3-ITD mutation

Leukemia, 2022-03, Vol.36 (3), p.675-686

2022

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A zebrafish model for HAX1-associated congenital neutropenia

Haematologica (Roma), 2021-05, Vol.106 (5), p.1311-1320

2021

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Cooperating, congenital neutropenia–associated Csf3r and Runx1 mutations activate pro-inflammatory signaling and inhibit myeloid differentiation of mouse HSPCs

Annals of hematology, 2020-10, Vol.99 (10), p.2329-2338

2020

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Lentiviral gene therapy and vitamin B3 treatment enable granulocytic differentiation of G6PC3-deficient induced pluripotent stem cells

Gene therapy, 2020-06, Vol.27 (6), p.297-306

2020

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Large-Scale Hematopoietic Differentiation of Human Induced Pluripotent Stem Cells Provides Granulocytes or Macrophages for Cell Replacement Therapies

Stem cell reports, 2015-02, Vol.4 (2), p.282-296

2015

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CRISPR/Cas9-mediated ELANE knockout enables neutrophilic maturation of primary hematopoietic stem and progenitor cells and induced pluripotent stem cells of severe congenital neutropenia patients

Haematologica (Roma), 2020-03, Vol.105 (3), p.598-609

2020

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The diversity of mutations and clinical outcomes for ELANE-associated neutropenia

Current opinion in hematology, 2015-01, Vol.22 (1), p.3-11

2015

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The role of sirtuin 2 activation by nicotinamide phosphoribosyltransferase in the aberrant proliferation and survival of myeloid leukemia cells

Haematologica (Roma), 2012-04, Vol.97 (4), p.551-559

2012

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NAMPT is essential for the G-CSF-induced myeloid differentiation via a NAD + -sirtuin-1-dependent pathway

Nature medicine, 2009-02, Vol.15 (2), p.151-158

2009

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Correction: NAMPT/SIRT2-mediated inhibition of the p53-p21 signaling pathway is indispensable for maintenance and hematopoietic differentiation of human iPS cells

Stem cell research & therapy, 2023-10, Vol.14 (1), p.1-299, Article 299

2023

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A topological refactoring design strategy yields highly stable granulopoietic proteins

Nature communications, 2022-05, Vol.13 (1), p.2948-2948, Article 2948

2022

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P458: HIPSC DISEASE MODELING TO STUDY LEUKEMIA DEVELOPMENT IN HAX1 VS ELANE ASSOCIATED CONGENITAL NEUTROPENIA

HemaSphere, 2023-08, Vol.7 (S3), p.e39781cd-n/a

2023

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Design of novel granulopoietic proteins by topological rescaffolding

PLoS biology, 2020-12, Vol.18 (12), p.e3000919-e3000919

2020

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Neutropenie im Kindesalter

Monatsschrift Kinderheilkunde, 2021-07, Vol.169 (7), p.659-669

2021

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Old drug revisited: disulfiram, NETs, and sepsis

Blood, 2021-12, Vol.138 (25), p.2604-2605

2021

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NAMPT/SIRT2-mediated inhibition of the p53-p21 signaling pathway is indispensable for maintenance and hematopoietic differentiation of human iPS cells

Stem cell research & therapy, 2021-02, Vol.12 (1), p.112-112, Article 112

2021

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Neutropenie im Kindesalter

Best practice onkologie, 2021-11, Vol.16 (11), p.532-543

2021

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CRISPR/Cas9 Genome Editing of Human-Induced Pluripotent Stem Cells Followed by Granulocytic Differentiation

Expanding the genetic landscape of congenital neutropenia: CXCR2 mutations in three families revealed through whole exome sequencing

Generation, expansion, and drug treatment of hematopoietic progenitor cells derived from human iPSCs

Circumventing Mutation to Nix Neutropenia

Gene Knockout in Hematopoietic Stem and Progenitor Cells Followed by Granulocytic Differentiation

Differential transcriptional control of hematopoiesis in congenital and cyclic neutropenia patients harboring ELANE mutations

Case report: Granulocyte-macrophage colony-stimulating factor sargramostim did not rescue the neutrophil phenotype in two patients with JAGN1-mutant severe congenital neutropenia

A unique role of p53 haploinsufficiency or loss in the development of acute myeloid leukemia with FLT3-ITD mutation

A zebrafish model for HAX1-associated congenital neutropenia

Cooperating, congenital neutropenia–associated Csf3r and Runx1 mutations activate pro-inflammatory signaling and inhibit myeloid differentiation of mouse HSPCs

Lentiviral gene therapy and vitamin B3 treatment enable granulocytic differentiation of G6PC3-deficient induced pluripotent stem cells

Cooperativity of RUNX1 and CSF3R mutations in severe congenital neutropenia: a unique pathway in myeloid leukemogenesis

Large-Scale Hematopoietic Differentiation of Human Induced Pluripotent Stem Cells Provides Granulocytes or Macrophages for Cell Replacement Therapies

CRISPR/Cas9-mediated ELANE knockout enables neutrophilic maturation of primary hematopoietic stem and progenitor cells and induced pluripotent stem cells of severe congenital neutropenia patients

The diversity of mutations and clinical outcomes for ELANE-associated neutropenia

The role of sirtuin 2 activation by nicotinamide phosphoribosyltransferase in the aberrant proliferation and survival of myeloid leukemia cells

NAMPT is essential for the G-CSF-induced myeloid differentiation via a NAD + -sirtuin-1-dependent pathway

Correction: NAMPT/SIRT2-mediated inhibition of the p53-p21 signaling pathway is indispensable for maintenance and hematopoietic differentiation of human iPS cells

A topological refactoring design strategy yields highly stable granulopoietic proteins

P458: HIPSC DISEASE MODELING TO STUDY LEUKEMIA DEVELOPMENT IN HAX1 VS ELANE ASSOCIATED CONGENITAL NEUTROPENIA

Design of novel granulopoietic proteins by topological rescaffolding

Neutropenie im Kindesalter

Old drug revisited: disulfiram, NETs, and sepsis

NAMPT/SIRT2-mediated inhibition of the p53-p21 signaling pathway is indispensable for maintenance and hematopoietic differentiation of human iPS cells

Neutropenie im Kindesalter

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