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Journal Articles

A Pipeline for Comparison and Selection of Clinically Applicable Gene Therapy Approaches for ELANE -Associated Severe Congenital Neutropenia

Malte Ulrich Ritter, Masoud Nasri, Benjamin Dannenmann, Perihan Mir, Benjamin Secker, Cornelia Zeidler, Maksim Klimiankou, Karl Welte, Julia Skokowa

Blood (2023) 142 (Supplement 1): 934.

https://doi.org/10.1182/blood-2023-188707

Published: 2023

Journal Articles

A Universal CRISPR/Cas9n-Mediated Genome Editing Approach for ELANE -Related Severe Congenital Neutropenia

Masoud Nasri, Malte Ulrich Ritter, Perihan Mir, Benjamin Dannenmann, Masako Monika Kaufmann, Yun Xu, Natalia Borbaran-Bravo, Maksim Klimiankou, Claudia Lengerke, Cornelia Zeidler, Toni Cathomen, Karl Welte, Julia Skokowa

Blood (2023) 142 (Supplement 1): 932.

https://doi.org/10.1182/blood-2023-181678

Published: 2023

Journal Articles

Differential Expression of C/Ebpa and Its Stem Cell-Specific Targets in Congenital Neutropenias and Cyclic Neutropenias

Alexander Zeidler, Natalia Borbaran-Bravo, Josef Koenig, Cornelia Zeidler, Julia Skokowa, Karl Welte

Blood (2022) 140 (Supplement 1): 11174.

https://doi.org/10.1182/blood-2022-169231

Published: 2022

Journal Articles

Accumulation of Specific Somatic Leukemia-Associated Mutations in Congenital Neutropenia Precedes Malignant Transformation - New Preconditions for Treatment Decisions

Maksim Klimiankou, Sergey Kandabarau, Cornelia Zeidler, Ingeborg Steiert, Denys Pogozhykh, David C. Dale, Karl Welte, Julia Skokowa

Blood (2022) 140 (Supplement 1): 994–995.

https://doi.org/10.1182/blood-2022-158914

Published: 2022

Journal Articles

CLPB in neutropenia: 1 gene with many faces

Claudia Lengerke, Julia Skokowa

Blood (2022) 139 (5): 649–650.

https://doi.org/10.1182/blood.2021012606

Published: 2022

Journal Articles

Old drug revisited: disulfiram, NETs, and sepsis

Maksim Klimiankou, Julia Skokowa

Blood (2021) 138 (25): 2604–2605.

https://doi.org/10.1182/blood.2021013438

Published: 2021

Journal Articles

SRP54 mutations induce congenital neutropenia via dominant-negative effects on XBP1 splicing

Christoph Schürch, Thorsten Schaefer, Joëlle S. Müller, Pauline Hanns, Marlon Arnone, Alain Dumlin, Jonas Schärer, Irmgard Sinning, Klemens Wild, Julia Skokowa, Karl Welte, Raphael Carapito, Seiamak Bahram, Martina Konantz, Claudia Lengerke

Blood (2021) 137 (10): 1340–1352.

https://doi.org/10.1182/blood.2020008115

Published: 2021

Includes: Supplemental data

Journal Articles

Efficient Correction of HAX1 Mutations in Primary HSPCs of Severe Congenital Neutropenia Patients Using CRISPR/CAS9 GENE-Editing

Malte U Ritter, Benjamin Secker, Masoud Nasri, MSc, Maksim Klimiankou, Benjamin Dannenmann, MSc, Diana Amend, Jeremy Haaf, Msc, Perihan Mir, Regine Bernhard, Ingeborg Steiert, Cornelia Zeidler, Karl Welte, Julia Skokowa, MDPhD

Blood (2020) 136 (Supplement 1): 22.

https://doi.org/10.1182/blood-2020-140260

Published: 2020

Journal Articles

Nicotinamide (NA) Treatment Improves Response to G-CSF in Severe Congenital Neutropenia (SCN) Patients

Ekaterina Deordieva, Julia Skokowa, MDPhD, Anna Shcherbina, Galina Novichkova, MD, Alexey Maschan, MD, Karl Welte

Blood (2020) 136 (Supplement 1): 38.

https://doi.org/10.1182/blood-2020-142289

Published: 2020

Journal Articles

De Novo Design of Granulopoietic Proteins

Julia Skokowa, MDPhD, Mohammad Elgamacy, PhDMSc,BSc, Patrick Müller

Blood (2020) 136 (Supplement 1): 34–35.

https://doi.org/10.1182/blood-2020-138852

Published: 2020

Journal Articles

Transcriptome Analysis of Severe Congenital Neutropenia Associated Leukemia with Different Types of RUNX1 Mutations in an iPSC-Based Model

Benjamin Dannenmann, MSc, Benedikt Oswald, Malte U Ritter, Masoud Nasri, MSc, Regine Bernhard, Cornelia Zeidler, Karl Welte, Julia Skokowa, MDPhD, Maksim Klimiankou

Blood (2020) 136 (Supplement 1): 23–24.

https://doi.org/10.1182/blood-2020-142524

Published: 2020

Journal Articles

A Zebrafish Model for Severe Congenital Neutropenia with Jagn1b-Deficiency

Larissa Doll, Narges Aghaallaei, Julia Skokowa, MDPhD, Karl Welte, Baubak Bajoghli

Blood (2020) 136 (Supplement 1): 14.

https://doi.org/10.1182/blood-2020-141886

Published: 2020

Journal Articles

Efficient Correction of ELANE mutations in Primary HSPCs of Severe Congenital Neutropenia Patients Using CRISPR/Cas9 and rAVV6 HDR Repair Templates

Malte U Ritter, Benjamin Secker, Maksim Klimiankou, Masoud Nasri, MSc, Narges Aghaallaei, Benjamin Dannenmann, Regine Bernhard, Ingeborg Steiert, Karin Haehnel, Baubak Bajoghli, Cornelia Zeidler, Karl Welte, Julia Skokowa, MD PhD

Blood (2019) 134 (Supplement_1): 1036.

https://doi.org/10.1182/blood-2019-131708

Published: 2019

Journal Articles

102 Patients with Primary Autoimmune Neutropenia : An Analysis of the European Severe Chronic Neutropenia International Registry (SCNIR)

Fabian Frömling, Sabine Mellor-Heineke, Natali Gerschmann, Lena Röbbel, Julia Skokowa, MD PhD, Karl Welte, Cornelia Zeidler

Blood (2019) 134 (Supplement_1): 1039.

https://doi.org/10.1182/blood-2019-130031

Published: 2019

Journal Articles

Disease Modeling of Severe Congenital Neutropenia Using CRISPR/Cas9 Gene Correction or Knockout of ELANE in Patients Derived Induced Pluripotent Stem Cells

Masoud Nasri, MSc, Benjamin Dannenmann, Perihan Mir, Malte U Ritter, Diana Amend, Yun Xu, Maksim Klimiankou, Cornelia Zeidler, Karl Welte, Julia Skokowa, MD PhD

Blood (2019) 134 (Supplement_1): 434.

https://doi.org/10.1182/blood-2019-129991

Published: 2019

Journal Articles

Hax1 Is Indispensable for Neutrophil Development in Zebrafish

Larissa Doll, Narges Aghaallaei, Karl Welte, Julia Skokowa, MD PhD, Baubak Bajoghli

Blood (2019) 134 (Supplement_1): 436.

https://doi.org/10.1182/blood-2019-130226

Published: 2019

Journal Articles

Mutation-Specific Dominant Negative Effects Determine the Phenotype in SRP54 D eficiency

Christoph Schuerch, MSc, Thorsten Schaefer, PhD, Joelle S. Müller, MSc, Pauline Hanns, MSc, Jonas Schärer, BSc, Julia Skokowa, MD PhD, Karl Welte, Raphael Carapito, PhD, Seiamak Bahram, MD, Martina Konantz, PhD, Claudia Lengerke, MD

Blood (2019) 134 (Supplement_1): 437.

https://doi.org/10.1182/blood-2019-127007

Published: 2019

Journal Articles

Heterozygous Mutations of Clpb As a Newly Identified and Frequent Cause of Severe Congenital Neutropenia

Julia T Warren, MD PhD, Peeradol Wattanasirakul, David H Spencer, MD, Adam Locke, PhD, Vahagn Makaryan, MD, Audrey Anna Bolyard, RN, BS, Karl Welte, Julia Skokowa, MD PhD, David C. Dale, MD, Daniel C. Link, MD

Blood (2019) 134 (Supplement_1): 433.

https://doi.org/10.1182/blood-2019-129635

Published: 2019

Journal Articles

Active DNA Demethylation Mediated By GADD45β Is Essential during G-CSF Triggered Granulocytic Differentiation

Perihan Mir, Maksim Klimiankou, Narges Aghaallaei, Betuel Findik, Frederic Thalheimer, Susanne Wingert, Michael A. Rieger, Thomas Thumberger, Cornelia Zeidler, Baubak Bajoghli, Karl Welte, Julia Skokowa, MD PhD

Blood (2019) 134 (Supplement_1): 211.

https://doi.org/10.1182/blood-2019-128508

Published: 2019

Journal Articles

Response to High Dose G-CSF Treatment (20µg/kg/d or Higher) of Patients with Congenital Neutropenia: An Analysis By the Scnir in Europe

Cornelia Zeidler, Natali Gerschmann, Sabine Mellor-Heineke, Fabian Frömling, Julia Skokowa, MD PhD, Karl Welte

Blood (2019) 134 (Supplement_1): 1225.

https://doi.org/10.1182/blood-2019-129844

Published: 2019

NARROW

A Pipeline for Comparison and Selection of Clinically Applicable Gene Therapy Approaches for ELANE -Associated Severe Congenital Neutropenia

A Universal CRISPR/Cas9n-Mediated Genome Editing Approach for ELANE -Related Severe Congenital Neutropenia

Differential Expression of C/Ebpa and Its Stem Cell-Specific Targets in Congenital Neutropenias and Cyclic Neutropenias

Accumulation of Specific Somatic Leukemia-Associated Mutations in Congenital Neutropenia Precedes Malignant Transformation - New Preconditions for Treatment Decisions

CLPB in neutropenia: 1 gene with many faces

Old drug revisited: disulfiram, NETs, and sepsis

SRP54 mutations induce congenital neutropenia via dominant-negative effects on XBP1 splicing

Efficient Correction of HAX1 Mutations in Primary HSPCs of Severe Congenital Neutropenia Patients Using CRISPR/CAS9 GENE-Editing

Nicotinamide (NA) Treatment Improves Response to G-CSF in Severe Congenital Neutropenia (SCN) Patients

De Novo Design of Granulopoietic Proteins

Transcriptome Analysis of Severe Congenital Neutropenia Associated Leukemia with Different Types of RUNX1 Mutations in an iPSC-Based Model

A Zebrafish Model for Severe Congenital Neutropenia with Jagn1b-Deficiency

Efficient Correction of ELANE mutations in Primary HSPCs of Severe Congenital Neutropenia Patients Using CRISPR/Cas9 and rAVV6 HDR Repair Templates

102 Patients with Primary Autoimmune Neutropenia : An Analysis of the European Severe Chronic Neutropenia International Registry (SCNIR)

Disease Modeling of Severe Congenital Neutropenia Using CRISPR/Cas9 Gene Correction or Knockout of ELANE in Patients Derived Induced Pluripotent Stem Cells

Hax1 Is Indispensable for Neutrophil Development in Zebrafish

Mutation-Specific Dominant Negative Effects Determine the Phenotype in SRP54 D eficiency

Heterozygous Mutations of Clpb As a Newly Identified and Frequent Cause of Severe Congenital Neutropenia

Active DNA Demethylation Mediated By GADD45β Is Essential during G-CSF Triggered Granulocytic Differentiation

Response to High Dose G-CSF Treatment (20µg/kg/d or Higher) of Patients with Congenital Neutropenia: An Analysis By the Scnir in Europe

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American Society of Hematology

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American Society of Hematology

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