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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

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

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

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

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

Journal Articles

Understanding the Role of CSF3R and Runx1 Runt Homology Domain Missense Mutations in Leukemic Transformation of Hematopoietic Stem Cells

Malte U Ritter, Olga Klimenkova, Maksim Klimiankou, Amy E Schmidt, MDPhD, Carol Stocking, PhD, Lothar Kanz, Cornelia Zeidler, Daniel C. Link, MD, Karl Welte, Julia Skokowa, MDPhD

Blood (2018) 132 (Supplement 1): 1101.

https://doi.org/10.1182/blood-2018-99-118644

Published: 2018

Journal Articles

A Method to Fluorescently Label the CRISPR/Cas9-gRNA RNP Complexes Enables Enrichment of Clinical-Grade Gene-Edited Primary Hematopoietic Stem Cells and iPSCs

Masoud Nasri, Perihan Mir, Benjamin Dannenmann, Diana Amend, Yun Xu, Anna Solovyeva, Sylwia Stefanczyk, Maksim Klimiankou, Cornelia Zeidler, Lothar Kanz, Karl Welte, Julia Skokowa, MDPhD

Blood (2018) 132 (Supplement 1): 1108.

https://doi.org/10.1182/blood-2018-99-114844

Published: 2018

Journal Articles

Mechanism of BAALC-Mediated Leukemogenesis Downstream of RUNX1 -Mutations in Severe Congenital Neutropenia

Benjamin Dannenmann, Maksim Klimiankou, Benedikt Oswald, Malte U Ritter, Masoud Nasri, Anna Solovyeva, Lothar Kanz, Cornelia Zeidler, Karl Welte, Julia Skokowa, MDPhD

Blood (2018) 132 (Supplement 1): 13.

https://doi.org/10.1182/blood-2018-99-118558

Published: 2018

Journal Articles

GADD45b Plays an Essential Role in the G-CSF Triggered Granulocytic Differentiation of Human Hematopoietic Cells

Perihan Mir, Masoud Nasri, Benjamin Dannenmann, Maksim Klimiankou, Betuel Findik, Regine Bernhard, Michael A. Rieger, Cornelia Zeidler, Lothar Kanz, Karl Welte, Julia Skokowa, MDPhD

Blood (2018) 132 (Supplement 1): 17.

https://doi.org/10.1182/blood-2018-99-114875

Published: 2018

Journal Articles

HAX1 Associated Congenital Neutropenia: A 25 Year Long-Term Analysis of the European Scnir Cohort

Cornelia Zeidler, Sabine Mellor-Heineke, MD, Maksim Klimiankou, Julia Skokowa, Karl Welte, MD PhD

Blood (2017) 130 (Supplement 1): 3566.

https://doi.org/10.1182/blood.V130.Suppl_1.3566.3566

Published: 2017

Journal Articles

Establishment of the Safe and Efficient CRISPR/Cas9-RNP Based Gene-Correction Platform of ELANE Mutations in iPSCs of Severe Congenital Neutropenia (CN) Patients with No Response to G-CSF and High Risk to Develop Leukemia

Masoud Nasri, MSc, Benjamin Dannenmann, Perihan Mir, Tessa Skroblyn, Anna Solovjeva, Maksim Klimiankou, Cornelia Zeidler, Lothar Kanz, Karl Welte, Julia Skokowa

Blood (2017) 130 (Supplement 1): 542.

https://doi.org/10.1182/blood.V130.Suppl_1.542.542

Published: 2017

Journal Articles

BAALC Is a Key Mediator of Leukemia Development in Congenital Neutropenia

Benjamin Dannenmann, Maksim Klimiankou, Anna Solovyeva, Azadeh Zahabi, MSc, Perihan Mir, Masoud Nasri, MSc, Regine Bernhard, Christian Lindner, Tatsuya Morishima, MD PhD, Doris Steinemann, PhD, Cornelia Zeidler, Lothar Kanz, Karl Welte, MD PhD, Julia Skokowa

Blood (2017) 130 (Supplement 1): 541.

https://doi.org/10.1182/blood.V130.Suppl_1.541.541

Published: 2017

Journal Articles

Effects of CSF3R mutations on Myeloid Differentiation and Proliferation of Hematopoietic Cells of Congenital Neutropenia Patients

Maksim Klimiankou, Benjamin Dannenmann, Anna Solovyeva, Regine Bernhard, Diana Amend, Cornelia Zeidler, Sabine Mellor-Heineke, MD, Lothar Kanz, Julia Skokowa, Karl Welte, MD PhD

Blood (2017) 130 (Supplement 1): 2278.

https://doi.org/10.1182/blood.V130.Suppl_1.2278.2278

Published: 2017

Journal Articles

Long-Term Outcomes for G-CSF Treatment of Patients with Glycogen-Storage Disease Type Ib

David Dale, MD, Audrey Anna Bolyard, RN, BS, Tracy M. Marrero, David A. Weinstein, MD MMSc, Cornelia Zeidler, Karl Welte

Blood (2017) 130 (Supplement 1): 996.

https://doi.org/10.1182/blood.V130.Suppl_1.996.996

Published: 2017

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

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

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

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

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

Understanding the Role of CSF3R and Runx1 Runt Homology Domain Missense Mutations in Leukemic Transformation of Hematopoietic Stem Cells

A Method to Fluorescently Label the CRISPR/Cas9-gRNA RNP Complexes Enables Enrichment of Clinical-Grade Gene-Edited Primary Hematopoietic Stem Cells and iPSCs

Mechanism of BAALC-Mediated Leukemogenesis Downstream of RUNX1 -Mutations in Severe Congenital Neutropenia

GADD45b Plays an Essential Role in the G-CSF Triggered Granulocytic Differentiation of Human Hematopoietic Cells

HAX1 Associated Congenital Neutropenia: A 25 Year Long-Term Analysis of the European Scnir Cohort

Establishment of the Safe and Efficient CRISPR/Cas9-RNP Based Gene-Correction Platform of ELANE Mutations in iPSCs of Severe Congenital Neutropenia (CN) Patients with No Response to G-CSF and High Risk to Develop Leukemia

BAALC Is a Key Mediator of Leukemia Development in Congenital Neutropenia

Effects of CSF3R mutations on Myeloid Differentiation and Proliferation of Hematopoietic Cells of Congenital Neutropenia Patients

Long-Term Outcomes for G-CSF Treatment of Patients with Glycogen-Storage Disease Type Ib

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American Society of Hematology

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ASH Publications

American Society of Hematology

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