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Journal Articles

Impact of disease genotype and severity of anemia on myocardial extracellular volume fraction in sickle cell disease (SCD) and non-SCD hemolytic anemias

Omar Niss, Cara Morin, Sassan Hashemi, Tarek Alsaied, Sean Lang, MacKenzie Tasset, Michael Taylor, Punam Malik, Charles Quinn

Blood (2025) 146 (Supplement 1): 2957.

https://doi.org/10.1182/blood-2025-2957

Published: 2025

Journal Articles

Renin-angiotensin-aldosterone system inhibitors exacerbate anemia in sickle cell disease

Emma Pappano, Najmeh Eskandari, Maria Armila Ruiz, Guohui Ren, Xu Zhang, Jin Han, Zalaya Ivy, Franklin Njoku, Marwah Farooqui, Robert Molokie, Victor Gordeuk, Mark Gladwin, Punam Malik, Charles Quinn, Santosh Saraf

Blood (2025) 146 (Supplement 1): 297.

https://doi.org/10.1182/blood-2025-297

Published: 2025

Journal Articles

Replicative stress-induced aging of hematopoietic stem progenitor cells increases oncogenic mutation burden and incidence of myelodysplasia in sickle cell disease mice

Mengna Chi, Fabienne Lucas, Ammar Husami, Tahereh Setayesh, Zhenqi Zhu, Harsimran Kaur, Anifat Tijani, Shristi Khanal, Jing Chen, Wenying Zhang, Punam Malik

Blood (2025) 146 (Supplement 1): 11.

https://doi.org/10.1182/blood-2025-11

Published: 2025

Journal Articles

Oxidative stress and hemoglobin-c denaturation drive hemoglobin SC pathophysiology and can be ameliorated by antioxidants and hydroxyurea

Tahereh Setayesh, Anifat Tijani, Harsimran Kaur, Shristi Khanal, Zhenqi Zhu, Mengna Chi, Abigail Kincaid, Russell Ware, Punam Malik

Blood (2025) 146 (Supplement 1): 7.

https://doi.org/10.1182/blood-2025-7

Published: 2025

Journal Articles

A novel mouse model of hemoglobin SC disease reveals mechanisms underlying beneficial effects of hydroxyurea

Tahereh Setayesh, Mengna Chi, Zachery Oestreicher, Masahide Sakabe, Katie Seu, Zhenqi Zhu, Harsimran Kaur, Anifat Tijani, Mei Xin, Amy Shova, Kenneth D. Greis, Tim M. Townes, József Balla, Katherine VandenHeuvel, Yueh-Chiang Hu, Punam Malik

Blood (2025) 146 (1): 13–28.

https://doi.org/10.1182/blood.2024028136

Published: 2025

Includes: Supplemental data

Journal Articles

Splenic Ossification in Sickle Cell Anemia Mice

Mengna Chi, Katherine Vandenheuvel, Punam Malik

Blood (2024) 144 (Supplement 1): 5283.

https://doi.org/10.1182/blood-2024-209046

Published: 2024

Journal Articles

Blockade of Angiotensin II Signaling Ameliorates Cardiac Fibrosis and Improves Diastolic Dysfunction in Mice with Sickle Cell Anemia

Keara Little, Zhenqi Zhu, Oluwabukola Gbotosho, Punam Malik

Blood (2024) 144 (Supplement 1): 2480.

https://doi.org/10.1182/blood-2024-208670

Published: 2024

Journal Articles

Underutilization of Disease-Modifying Therapies in Sickle Cell Disease: A Real-World Analysis from the ASH Research Collaborative Data Hub

Omar Niss, Matthew Fenchel, Erin Kingsley, Teresa S. Latham, Punam Malik, Charles T. Quinn

Blood (2024) 144 (Supplement 1): 2312.

https://doi.org/10.1182/blood-2024-206015

Published: 2024

Journal Articles

Longitudinal Assessment of Myocardial Fibrosis in Sickle Cell Disease

Omar Niss, Cara Morin, Sean M Lang, Tarek Alsaied, MacKenzie Tasset, Punam Malik, Charles T. Quinn

Blood (2024) 144 (Supplement 1): 799.

https://doi.org/10.1182/blood-2024-200951

Published: 2024

Journal Articles

A Novel Mouse Model of Hemoglobin-SC Disease: Bridging the Knowledge Gap in Sickle Cell Disease

Tahereh Setayesh, Mengna Chi, Zachery Oestreicher, Masahide Sakabe, Xin Mei, Katherine Vandenheuvel, Tim M. Townes, Yueh-Chiang Hu, Punam Malik

Blood (2024) 144 (Supplement 1): 620.

https://doi.org/10.1182/blood-2024-206241

Published: 2024

Journal Articles

CRISPR/CAS9 Gene Editing of MIR155HG in Primary Human T Cells to Prevent Acute Graft-Versus-Host Disease

Shrijan Khanal, Lotus Neidemire-Colley, Kara M Braunreiter, Katiri Snyder, Yandi Gao, Sonu Kalyan, Meisam Naeimi Kararoudi, Molly Erin Duszynski, Mengna Chi, Punam Malik, Hannah Choe, Ramiro Garzon, Parvathi Ranganathan

Blood (2022) 140 (Supplement 1): 10200–10201.

https://doi.org/10.1182/blood-2022-159599

Published: 2022

Journal Articles

Early initiation of disease-modifying therapy can impede or prevent diffuse myocardial fibrosis in sickle cell anemia

Omar Niss, Jon Detterich, John C. Wood, Thomas D. Coates, Punam Malik, Michael D. Taylor, Charles T. Quinn

Blood (2022) 140 (11): 1322–1324.

https://doi.org/10.1182/blood.2021015303

Published: 2022

Includes: Supplemental data

Journal Articles

Safety and Efficacy of Aru-1801 in Patients with Sickle Cell Disease: Early Results from the Phase 1/2 Momentum Study of a Modified Gamma Globin Gene Therapy and Reduced Intensity Conditioning

Michael Grimley, Monika Asnani, Archana Shrestha, Sydney Felker, Carolyn Lutzko, Paritha I. Arumugam, Scott Witting, Jennifer Knight-Madden, Omar Niss, Charles T. Quinn, Chris Lo, Courtney R. Little, Alisa Dong, Punam Malik

Blood (2021) 138 (Supplement 1): 3970.

https://doi.org/10.1182/blood-2021-147469

Published: 2021

Journal Articles

Bone Marrow (BM) Delivery of Genetically-Modified (gm) Adult CD34+ Hematopoietic Stem and Progenitor Cells (HSPC) Improves Homing and Engraftment of Short-Term Progenitors over Long-Term Repopulating Hematopoietic Stem Cells

Sydney Felker, Archana Shrestha, Punam Malik, MD

Blood (2020) 136 (Supplement 1): 22–23.

https://doi.org/10.1182/blood-2020-138529

Published: 2020

Journal Articles

FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Demonstrates Proof of Mechanism and Proof of Concept after a Single Dose and after Multiple Daily Doses in a Phase 1 Study of Patients with Sickle Cell Disease

R. Clark Brown, MD PhD, Kimberly Cruz, MD MS, Theodosia A. Kalfa, MD PhD, Frans A. Kuypers, PhD, Santosh L. Saraf, MD, Jeremie H. Estepp, MD, Luke R. Smart, MD, Punam Malik, MD, Mark Lerman, MD, Renae Mayer, MD, Maria D. Ribadeneira, PhD, Sanjeev Forsyth, MS, Patricia Schroeder, PhD, Eric Wu, PhD, Patrick Kelly, MD, Marilyn J. Telen, MD

Blood (2020) 136 (Supplement 1): 19–20.

https://doi.org/10.1182/blood-2020-134269

Published: 2020

Journal Articles

Early Results from a Phase 1/2 Study of Aru-1801 Gene Therapy for Sickle Cell Disease (SCD): Manufacturing Process Enhancements Improve Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant

Michael Grimley, MD, Monika Asnani, PhD, Archana Shrestha, Sydney Felker, Carolyn Lutzko, PhD, Paritha I. Arumugam, PhD, Scott Witting, PhD, Jennifer Knight-Madden, MBBS, PhD, Omar Niss, MD, Charles T. Quinn, MD, Chris Lo, BS, Courtney R Little, BSN, Joseph W. McIntosh, MD, Punam Malik, MD

Blood (2020) 136 (Supplement 1): 20–21.

https://doi.org/10.1182/blood-2020-140963

Published: 2020

Journal Articles

Angiotensin Signaling Is Essential for Stress Erythropoiesis but Results in Retention of Dysfunctional Mitochondria in Erythrocytes That Generate Excessive Reactive Oxygen Species

Parul Rai, MDMBBS, Swarnava Roy, PhD, Diamantis G. Konstantinidis, PhD BSc, Sithara Raju Ponny, MS, Marthe-sandrine Eiymo Mwa Mpollo, PhD, Archana Shrestha, Theodosia A. Kalfa, MD PhD, Punam Malik, MD

Blood (2020) 136 (Supplement 1): 31–32.

https://doi.org/10.1182/blood-2020-139980

Published: 2020

Journal Articles

Increased Hydroxyurea Prescribing Practices over Ten Years with Improved Clinical Outcomes in Children with Sickle Cell Anemia: A Single Center's Experience

Kristine Anne Karkoska, MD, Kevin E Todd, Jr., MD, Kelly Clapp, NP, Lynette Fenchel, NP, Theodosia A. Kalfa, MD PhD, Punam Malik, MD, Omar Niss, MD, Charles T. Quinn, MD, Russell E. Ware, MD PhD, Patrick T. McGann, MD MS

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

https://doi.org/10.1182/blood-2020-138754

Published: 2020

Journal Articles

Oral Administration of FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Has Potent Anti-Sickling Effects in a Sickle Cell Anemia (SCA) Mouse Model, Resulting in Improved RBC Survival and Hemoglobin Levels

Archana Shrestha, Mengna Chi, PhD, Kimberly Wagner, Adam Drake, Keertik Fulzele, PhD, Sylvie Guichard, Punam Malik, MD

Blood (2020) 136 (Supplement 1): 21–22.

https://doi.org/10.1182/blood-2020-140875

Published: 2020

Journal Articles

Rapid and Automated Quantitation of Dense Red Blood Cells: A Robust Biomarker of Therapeutic Response to Early Initiation of Hydroxyurea in Young Children with Sickle Cell Anemia

Alina Sadaf, MD, Charles T. Quinn, MD, Jennifer B Korpik, Amanda Pfeiffer, BS, MA, Mary Reynaud, MA, MT(ASCP), Omar Niss, MD, Punam Malik, MD, Russell E. Ware, MD PhD, Theodosia A. Kalfa, MD PhD, Patrick T. McGann, MD MS

Blood (2020) 136 (Supplement 1): 16–17.

https://doi.org/10.1182/blood-2020-137070

Published: 2020

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Impact of disease genotype and severity of anemia on myocardial extracellular volume fraction in sickle cell disease (SCD) and non-SCD hemolytic anemias

Renin-angiotensin-aldosterone system inhibitors exacerbate anemia in sickle cell disease

Replicative stress-induced aging of hematopoietic stem progenitor cells increases oncogenic mutation burden and incidence of myelodysplasia in sickle cell disease mice

Oxidative stress and hemoglobin-c denaturation drive hemoglobin SC pathophysiology and can be ameliorated by antioxidants and hydroxyurea

A novel mouse model of hemoglobin SC disease reveals mechanisms underlying beneficial effects of hydroxyurea

Splenic Ossification in Sickle Cell Anemia Mice

Blockade of Angiotensin II Signaling Ameliorates Cardiac Fibrosis and Improves Diastolic Dysfunction in Mice with Sickle Cell Anemia

Underutilization of Disease-Modifying Therapies in Sickle Cell Disease: A Real-World Analysis from the ASH Research Collaborative Data Hub

Longitudinal Assessment of Myocardial Fibrosis in Sickle Cell Disease

A Novel Mouse Model of Hemoglobin-SC Disease: Bridging the Knowledge Gap in Sickle Cell Disease

CRISPR/CAS9 Gene Editing of MIR155HG in Primary Human T Cells to Prevent Acute Graft-Versus-Host Disease

Early initiation of disease-modifying therapy can impede or prevent diffuse myocardial fibrosis in sickle cell anemia

Safety and Efficacy of Aru-1801 in Patients with Sickle Cell Disease: Early Results from the Phase 1/2 Momentum Study of a Modified Gamma Globin Gene Therapy and Reduced Intensity Conditioning

Bone Marrow (BM) Delivery of Genetically-Modified (gm) Adult CD34+ Hematopoietic Stem and Progenitor Cells (HSPC) Improves Homing and Engraftment of Short-Term Progenitors over Long-Term Repopulating Hematopoietic Stem Cells

FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Demonstrates Proof of Mechanism and Proof of Concept after a Single Dose and after Multiple Daily Doses in a Phase 1 Study of Patients with Sickle Cell Disease

Early Results from a Phase 1/2 Study of Aru-1801 Gene Therapy for Sickle Cell Disease (SCD): Manufacturing Process Enhancements Improve Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant

Angiotensin Signaling Is Essential for Stress Erythropoiesis but Results in Retention of Dysfunctional Mitochondria in Erythrocytes That Generate Excessive Reactive Oxygen Species

Increased Hydroxyurea Prescribing Practices over Ten Years with Improved Clinical Outcomes in Children with Sickle Cell Anemia: A Single Center's Experience

Oral Administration of FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Has Potent Anti-Sickling Effects in a Sickle Cell Anemia (SCA) Mouse Model, Resulting in Improved RBC Survival and Hemoglobin Levels

Rapid and Automated Quantitation of Dense Red Blood Cells: A Robust Biomarker of Therapeutic Response to Early Initiation of Hydroxyurea in Young Children with Sickle Cell Anemia

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