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Journal Articles

Single-Cell-Multiomics Demonstrates Molecular Efficacy of a Clinical Lentiviral Vector for Gene Therapy of RPS19-Deficient Diamond-Blackfan Anemia

Sofia Freiman, Jun Chen, Johan Flygare, Chayenne Kampman, Karin Pike-Overzet, Alexander Mohseny, Teng-Cheong Ha, Mathias Lidgren, Shamit Soneji, Stefan Karlsson, Dagmar Pospíšilová, Frank Staal, Axel Schambach, Johan Flygare

Blood (2023) 142 (Supplement 1): 943.

https://doi.org/10.1182/blood-2023-185802

Published: 2023

Journal Articles

The Serine Threonine Kinase Inhibitor Ots-167 Improves Erythropoiesis through Suppression of Nlk Activity in Diamond Blackfan Anemia Models

Aya Shibuya, Mark C Wilkes, Manuel Serrano, Johan Flygare, Bert Glader, Anupama Narla, Kathleen M. Sakamoto

Blood (2021) 138 (Supplement 1): 2179.

https://doi.org/10.1182/blood-2021-150070

Published: 2021

Journal Articles

Preclinical and Clinical Signs of Efficacy of RVU120 (SEL120), a Specific CDK8/19 Inhibitor in DNMT3A-Mutated AML

Blood (2021) 138 (Supplement 1): 2371.

https://doi.org/10.1182/blood-2021-152273

Published: 2021

Journal Articles

RVU120 (SEL120) CDK8/19 Inhibitor - a Drug Candidate for the Treatment of MDS Can Induce Erythroid Differentiation

Blood (2021) 138 (Supplement 1): 1518.

https://doi.org/10.1182/blood-2021-150662

Published: 2021

Journal Articles

Molecules Involved in the Generation of Definitive Hematopoiesis

Johan Flygare, MD PhD

Blood (2020) 136 (Supplement 1): SCI3.

https://doi.org/10.1182/blood-2020-133080

Published: 2020

Journal Articles

Metformin Upregulates Mir-26a to Improve Erythropoiesis in Preclinical Models of Diamond Blackfan Anemia through Suppression of Nlk Expression

Mark C Wilkes, PhD, Kavitha Siva, PhD, Jacqueline D Mercado, Ethan Patrick Wentworth, Mallika Saxena, Jun Chen, MD PhD, Anupama Narla, MD, Bertil Glader, MD PhD, Shuo Lin, PhD, Manuel Serrano, PhD, Johan Flygare, MD PhD, Kathleen M. Sakamoto, MD PhD

Blood (2020) 136 (Supplement 1): 7.

https://doi.org/10.1182/blood-2020-137170

Published: 2020

Journal Articles

Pharmacological Inhibition of Nemo-like Kinase Rescues mTOR-Mediated Translation and Primes Progenitors for Leucine-Stimulated Erythroid Expansion in Pre-Clinical Models of Diamond Blackfan Anemia

Mark C Wilkes, PhD, Jacqueline D Mercado, Mallika Saxena, Jun Chen, MD PhD, Kavitha Siva, PhD, Hee-Don Chae, PhD, Minyoung Youn, PhD, Ethan Patrick Wentworth, Kevin Jung, Ryan Sathianathen, Hiral Patel, Bertil Glader, MD PhD, Anupama Narla, MD, Claire E Repellin, PhD, Hanna T Gazda, MD PhD, Manuel Serrano, PhD, Johan Flygare, MD PhD, Kathleen M. Sakamoto, MD PhD

Blood (2019) 134 (Supplement_1): 455.

https://doi.org/10.1182/blood-2019-129570

Published: 2019

Journal Articles

CDK8 Inhibitor SEL120-34A Has Therapeutic Efficacy in Murine and Human Acute Myeloid Leukemia Models

Blood (2018) 132 (Supplement 1): 1520.

https://doi.org/10.1182/blood-2018-99-111859

Published: 2018

Journal Articles

Pharmacological Inhibition of Nlk (Nemo-like Kinase) Rescues Erythropoietic Defects in Pre-Clinical Models of Diamond Blackfan Anemia

Mark C Wilkes, Jun Chen, MD PhD, Kavitha Siva, PhD, Gianluca Veretti, PhD, Daniel P. Dever, PhD, Minyoung Youn, PhD, Hee-Don Chae, PhD, Jacqueline D Mercado, Mallika Saxena, Anupama Narla, MD, Bertil Glader, MD, Matthew Porteus, MD PhD, Claire E Repellin, PhD, Hanna T Gazda, MD PhD, Manuel Serrano, PhD, Johan Flygare, MD PhD, Kathleen M. Sakamoto, MD PhD

Blood (2018) 132 (Supplement 1): 754.

https://doi.org/10.1182/blood-2018-99-119954

Published: 2018

Journal Articles

Small Molecule Screens Identify CDK8-Inhibitors As Candidate Diamond-Blackfan Anemia Drugs

Blood (2018) 132 (Supplement 1): 753.

https://doi.org/10.1182/blood-2018-99-114570

Published: 2018

Journal Articles

A High-Throughput Screening Approach to Identify Therapeutics for the Treatment of Diamond-Blackfan Anaemia

Lorena Nunez Villacis, Sheren J Al-Obaidi, Piyush Madhamshettiwar, PhD, Nadine Hein, PhD, Jun Chen, MD PhD, Priscilla Soo, Perlita Poh, Megan Pavy, Kaylene J Simpson, PhD, Thomas John Gonda, PhD BSc, Richard B Pearson, PhD, Johan Flygare, MD PhD, Katherine M Hannan, PhD, Amee J George, PhD, Ross Hannan, PhD

Blood (2018) 132 (Supplement 1): 3859.

https://doi.org/10.1182/blood-2018-99-114303

Published: 2018

Journal Articles

Calmodulin Inhibition Rescues DBA Models with Ribosomal Protein Deficiency through Reduction of RSK Signaling

Elizabeth R Macari, PhD, Alison M Taylor, PhD, David Raiser, PhD, Kavitha Siva, PhD, Katherine McGrath, Jessica M Humphries, Johan Flygare, MDPhD, Benjamin L. Ebert, MDPhD, Leonard I. Zon, MD

Blood (2016) 128 (22): 332.

https://doi.org/10.1182/blood.V128.22.332.332

Published: 2016

Journal Articles

Calmodulin Inhibition Rescues the Effects of Ribosomal Protein Deficiency in in Vitro and In Vivo Diamond Blackfan Anemia Models

Elizabeth R Macari, PhD, Alison Taylor, PhD, David Raiser, PhD, Kavitha Siva, PhD, Katherine McGrath, Jessica M Humphries, Johan Flygare, MD PhD, Benjamin L Ebert, MD PhD, Leonard I. Zon, MD

Blood (2015) 126 (23): 672.

https://doi.org/10.1182/blood.V126.23.672.672

Published: 2015

Journal Articles

Direct Lineage Reprogramming of Murine Fibroblasts to Erythroid Progenitor Cells By Defined Factors

Sandra Capellera Garcia, Kishori Dhulipala, MD, Kavitha Siva, PhD, Violeta Rayon Estrada, Evelyn Wang, Gregory Hyde, PhD, Sofie Singbrant, PhD, Carl Walkley, PhD BPharm, Shamit Soneji, PhD, Vijay G. Sankaran, MD PhD, Johan Flygare, MD PhD

Blood (2014) 124 (21): 246.

https://doi.org/10.1182/blood.V124.21.246.246

Published: 2014

Journal Articles

Glucocorticoids Reduce Expression Of p53 Target Genes and Reverse Anemia In a Mouse Model Of Diamond-Blackfan Anemia

Sara Sjögren, Pekka Jaako, PhD, Stefan Karlsson, MD, PhD, Johan Flygare, MD, PhD

Blood (2013) 122 (21): 3703.

https://doi.org/10.1182/blood.V122.21.3703.3703

Published: 2013

Journal Articles

A Robust Screening Assay For Diamond Blackfan Anemia Candidate Drugs

Kavitha Siva, Fredrik Ek, Hanna Axelsson, Abdul Ghani Alattar, Svetlana Soboleva, Roger Olsson, Thomas Lundback, Johan Flygare, MD, PhD

Blood (2013) 122 (21): 2472.

https://doi.org/10.1182/blood.V122.21.2472.2472

Published: 2013

Journal Articles

Gene Therapy Corrects the Lethal Bone Marrow Failure in a Mouse Model for RPS19-Deficient Diamond-Blackfan Anemia

Pekka Jaako, Shubhranshu Debnath, Karin Olsson, Axel Schambach, MD, PhD, Christopher Baum, MD, Johan Flygare, MD, PhD, Stefan Karlsson, MD, PhD

Blood (2012) 120 (21): 513.

https://doi.org/10.1182/blood.V120.21.513.513

Published: 2012

Journal Articles

Dietary L-leucine improves the anemia in a mouse model for Diamond-Blackfan anemia

Pekka Jaako, Shubhranshu Debnath, Karin Olsson, David Bryder, Johan Flygare, Stefan Karlsson

Blood (2012) 120 (11): 2225–2228.

https://doi.org/10.1182/blood-2012-05-431437

Published: 2012

Includes: Supplemental data

Journal Articles

From stem cell to red cell: regulation of erythropoiesis at multiple levels by multiple proteins, RNAs, and chromatin modifications

Shilpa M. Hattangadi, Piu Wong, Lingbo Zhang, Johan Flygare, Harvey F. Lodish

Blood (2011) 118 (24): 6258–6268.

https://doi.org/10.1182/blood-2011-07-356006

Published: 2011

Journal Articles

Mice with ribosomal protein S19 deficiency develop bone marrow failure and symptoms like patients with Diamond-Blackfan anemia

Pekka Jaako, Johan Flygare, Karin Olsson, Ronan Quere, Mats Ehinger, Adrianna Henson, Steven Ellis, Axel Schambach, Christopher Baum, Johan Richter, Jonas Larsson, David Bryder, Stefan Karlsson

Blood (2011) 118 (23): 6087–6096.

https://doi.org/10.1182/blood-2011-08-371963

Published: 2011

Includes: Supplemental data

NARROW

Single-Cell-Multiomics Demonstrates Molecular Efficacy of a Clinical Lentiviral Vector for Gene Therapy of RPS19-Deficient Diamond-Blackfan Anemia

The Serine Threonine Kinase Inhibitor Ots-167 Improves Erythropoiesis through Suppression of Nlk Activity in Diamond Blackfan Anemia Models

Preclinical and Clinical Signs of Efficacy of RVU120 (SEL120), a Specific CDK8/19 Inhibitor in DNMT3A-Mutated AML

RVU120 (SEL120) CDK8/19 Inhibitor - a Drug Candidate for the Treatment of MDS Can Induce Erythroid Differentiation

Molecules Involved in the Generation of Definitive Hematopoiesis

Metformin Upregulates Mir-26a to Improve Erythropoiesis in Preclinical Models of Diamond Blackfan Anemia through Suppression of Nlk Expression

Pharmacological Inhibition of Nemo-like Kinase Rescues mTOR-Mediated Translation and Primes Progenitors for Leucine-Stimulated Erythroid Expansion in Pre-Clinical Models of Diamond Blackfan Anemia

CDK8 Inhibitor SEL120-34A Has Therapeutic Efficacy in Murine and Human Acute Myeloid Leukemia Models

Pharmacological Inhibition of Nlk (Nemo-like Kinase) Rescues Erythropoietic Defects in Pre-Clinical Models of Diamond Blackfan Anemia

Small Molecule Screens Identify CDK8-Inhibitors As Candidate Diamond-Blackfan Anemia Drugs

A High-Throughput Screening Approach to Identify Therapeutics for the Treatment of Diamond-Blackfan Anaemia

Calmodulin Inhibition Rescues DBA Models with Ribosomal Protein Deficiency through Reduction of RSK Signaling

Calmodulin Inhibition Rescues the Effects of Ribosomal Protein Deficiency in in Vitro and In Vivo Diamond Blackfan Anemia Models

Direct Lineage Reprogramming of Murine Fibroblasts to Erythroid Progenitor Cells By Defined Factors

Glucocorticoids Reduce Expression Of p53 Target Genes and Reverse Anemia In a Mouse Model Of Diamond-Blackfan Anemia

A Robust Screening Assay For Diamond Blackfan Anemia Candidate Drugs

Gene Therapy Corrects the Lethal Bone Marrow Failure in a Mouse Model for RPS19-Deficient Diamond-Blackfan Anemia

Dietary L-leucine improves the anemia in a mouse model for Diamond-Blackfan anemia

From stem cell to red cell: regulation of erythropoiesis at multiple levels by multiple proteins, RNAs, and chromatin modifications

Mice with ribosomal protein S19 deficiency develop bone marrow failure and symptoms like patients with Diamond-Blackfan anemia

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American Society of Hematology

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ASH Publications

American Society of Hematology

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