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Journal Articles

Robust HbF induction and improvement of anemia and hemolysis with base editing in sickle cell disease: Safety and efficacy findings from the ongoing BEACON study

Blood (2025) 146 (Supplement 1): 2532.

https://doi.org/10.1182/blood-2025-2532

Published: 2025

Journal Articles

Improved hematopoietic stem cell mobilization for gene therapy using single agent motixafortide in sickle cell disease

Stephan Grupp, Michael Grimley, Timothy Driscoll, Kris Mahadeo, Alexis Leonard, Yongping Wang, Helen Phillips, David Justus, Leslie Lehmann, Erica Esrick, Julie Kanter, Sarah Nikiforow, Matthew Hsieh, John Tisdale, Akshay Sharma, Zachary Crees, John Manis

Blood (2025) 146 (Supplement 1): 2542.

https://doi.org/10.1182/blood-2025-2542

Published: 2025

Journal Articles

Long-term follow-up demonstrates durable clinical benefits of exagamglogene autotemcel for sickle cell disease with recurrent vaso-occlusive crises: Final results of climb SCD-121

Blood (2025) 146 (Supplement 1): 2537.

https://doi.org/10.1182/blood-2025-2537

Published: 2025

Journal Articles

First results of exagamglogene autotemcel in pediatric patients aged 5-11 years with transfusion-dependent β-thalassemia or sickle cell disease with recurrent severe vaso-occlusive crises

Blood (2025) 146 (Supplement 1): 379.

https://doi.org/10.1182/blood-2025-379

Published: 2025

Journal Articles

Children and adolescents with sickle cell disease demonstrate improved health-related quality of life and low decisional regret after hematopoietic cell transplantation: A sickle cell transplant advocacy and research alliance (STAR) study

Blood (2025) 146 (Supplement 1): 4278.

https://doi.org/10.1182/blood-2025-4278

Published: 2025

Journal Articles

Decisions about gene therapy do not occur in isolation: Co-development of a website about treatment options for sickle cell disease

Liza-Marie Johnson, Yoram Unguru, Yvonne Carroll, Gary Ashwal, Alex Thomas, Lakiea Bailey, Dominique Goodson, Belinda Mandrell, Amanda Young, Ambria Williams, Benjamin Wilfond, Akshay Sharma

Blood (2025) 146 (Supplement 1): 6153.

https://doi.org/10.1182/blood-2025-6153

Published: 2025

Journal Articles

Healthcare costs associated with hematopoietic cell transplant for sickle cell disease in the United States and Canada: A comparative analysis

Jaspreet Gharial, Gregory Guilcher, Eldon Spackman, Akshay Sharma, Staci Arnold

Blood (2025) 146 (Supplement 1): 6151.

https://doi.org/10.1182/blood-2025-6151

Published: 2025

Journal Articles

A longitudinal study on the impact of hematopoietic stem cell transplantation on sickle cardiomyopathy

Parul Rai, Subodh Selukar, Yu Bi, Matthew Wogksch, Clifford Takemoto, Mark Rushing, Micheal Rebolledo, Vijaya Joshi, Cara Morin, Anthony Merlocco, Brandon Triplett, Jason Johnson, Akshay Sharma

Blood (2025) 146 (Supplement 1): 6050.

https://doi.org/10.1182/blood-2025-6050

Published: 2025

Journal Articles

HLA antibodies delay platelet recovery after gene therapy

Ashish O. Gupta, Akshay Sharma

Blood (2025) 145 (22): 2546–2547.

https://doi.org/10.1182/blood.2025028995

Published: 2025

Journal Articles

How I treat sickle cell disease with gene therapy

Akshay Sharma

Blood (2024) 144 (26): 2693–2705.

https://doi.org/10.1182/blood.2024024519

Published: 2024

Journal Articles

Health-Related Quality of Life Post-Hematopoietic Cell Transplantation in Sickle Cell Disease: Differences in Patient Self-Report and Caregiver Report

Jerlym Porter, Kathryn Russell, Kendra Parris, Sean Phipps, Alexis Kristine Leonard, Brandon M Triplett, Stephen Gottschalk, Akshay Sharma

Blood (2024) 144 (Supplement 1): 7676.

https://doi.org/10.1182/blood-2024-211217

Published: 2024

Journal Articles

Motixafortide for Hematopoietic Stem and Progenitor Cell Mobilization and Collection in Sickle Cell Disease

Alexis Kristine Leonard, Akshay Sharma, Yogindra Persaud, Parul Rai, Darla Pickett, Renee Massey, Neha Das Gupta, Subodh Selukar, Salem M. Akel, Leigh Poston, Yan Zheng, Ella Sorani, Irit Gliko Kabir, Mitchell J Weiss, Stephen Gottschalk, Julie Kanter, Matthew Hsieh, John Tisdale

Blood (2024) 144 (Supplement 1): 2100.1.

https://doi.org/10.1182/blood-2024-200330

Published: 2024

Journal Articles

Rheological Comparison of Red Cells from Individuals with SCD Treated with Gene Therapy, Haploidentical Transplantation or Hydroxyurea

Britney Hernandez, Ashwin P. Patel, Erica N Evans, Jordan Zgodny, Stephen Gottschalk, Brandon M Triplett, Serena De Vita, Kirby Wallace, David C Shyr, Elizabeth O Stenger, Suhag Parikh, Akshay Sharma, Vivien Sheehan

Blood (2024) 144 (Supplement 1): 4956.

https://doi.org/10.1182/blood-2024-210578

Published: 2024

Journal Articles

Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Disease

Blood (2024) 144 (Supplement 1): 4954.

https://doi.org/10.1182/blood-2024-204001

Published: 2024

Journal Articles

Cerebral Hemodynamics Improve in Adults with Sickle Cell Disease after Gene Therapy

Akshay Sharma, Jane S Hankins, Jaap Jan Boelens, Maria Cancio, Radhika Peddinti, Lawrence Rispoli, Sarah Sloan, Sarah DiDominick Costa, Sharon Peled, Andrea Wiethoff, James LaBelle

Blood (2024) 144 (Supplement 1): 4953.

https://doi.org/10.1182/blood-2024-203184

Published: 2024

Journal Articles

Clinician Adherence to Screening and Preventive Practices for Non-Malignant Late Effects Among Allogeneic Hematopoietic Cell Transplant Survivors in the United States

Blood (2024) 144 (Supplement 1): 2170.

https://doi.org/10.1182/blood-2024-210263

Published: 2024

Journal Articles

Real-World Clinician Adherence to Screening and Preventive Practices Guidelines for Subsequent Neoplasms Among Allogeneic Hematopoietic Cell Transplant Survivors in the United States

Blood (2024) 144 (Supplement 1): 4921.

https://doi.org/10.1182/blood-2024-199289

Published: 2024

Journal Articles

Kidney Outcomes after Hematopoietic Cell Transplantation in Children and Young Adults with Sickle Cell Disease

Rima Zahr, Yu Bi, Subodh Selukar, Stephen Gottschalk, Brandon M Triplett, Alexis Kristine Leonard, Akshay Sharma

Blood (2024) 144 (Supplement 1): 5067.

https://doi.org/10.1182/blood-2024-209896

Published: 2024

Journal Articles

Memory T-Cell Add Back and Prophylactic blinatumomab Post Tcrαβ Depleted Haploidentical Transplantation Results in Promising Outcomes in Pediatric Patients with High-Risk Acute Leukemia

Blood (2024) 144 (Supplement 1): 383.

https://doi.org/10.1182/blood-2024-208547

Published: 2024

Journal Articles

Health-Related Quality-of-Life Improvements after Exagamglogene Autotemcel in Patients with Severe Sickle Cell Disease

Akshay Sharma, Haydar Frangoul, Franco Locatelli, Kevin H.M. Kuo, Monica Bhatia, Markus Y Mapara, Michael J. Eckrich, Suzan Imren, Nanxin Li, Jaime Rubin, Siyu Zhang, Tina Liu, William Hobbs, Stephan A. Grupp

Blood (2024) 144 (Supplement 1): 7453.

https://doi.org/10.1182/blood-2024-204112

Published: 2024

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Robust HbF induction and improvement of anemia and hemolysis with base editing in sickle cell disease: Safety and efficacy findings from the ongoing BEACON study

Improved hematopoietic stem cell mobilization for gene therapy using single agent motixafortide in sickle cell disease

Long-term follow-up demonstrates durable clinical benefits of exagamglogene autotemcel for sickle cell disease with recurrent vaso-occlusive crises: Final results of climb SCD-121

First results of exagamglogene autotemcel in pediatric patients aged 5-11 years with transfusion-dependent β-thalassemia or sickle cell disease with recurrent severe vaso-occlusive crises

Children and adolescents with sickle cell disease demonstrate improved health-related quality of life and low decisional regret after hematopoietic cell transplantation: A sickle cell transplant advocacy and research alliance (STAR) study

Decisions about gene therapy do not occur in isolation: Co-development of a website about treatment options for sickle cell disease

Healthcare costs associated with hematopoietic cell transplant for sickle cell disease in the United States and Canada: A comparative analysis

A longitudinal study on the impact of hematopoietic stem cell transplantation on sickle cardiomyopathy

HLA antibodies delay platelet recovery after gene therapy

How I treat sickle cell disease with gene therapy

Health-Related Quality of Life Post-Hematopoietic Cell Transplantation in Sickle Cell Disease: Differences in Patient Self-Report and Caregiver Report

Motixafortide for Hematopoietic Stem and Progenitor Cell Mobilization and Collection in Sickle Cell Disease

Rheological Comparison of Red Cells from Individuals with SCD Treated with Gene Therapy, Haploidentical Transplantation or Hydroxyurea

Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Disease

Cerebral Hemodynamics Improve in Adults with Sickle Cell Disease after Gene Therapy

Clinician Adherence to Screening and Preventive Practices for Non-Malignant Late Effects Among Allogeneic Hematopoietic Cell Transplant Survivors in the United States

Real-World Clinician Adherence to Screening and Preventive Practices Guidelines for Subsequent Neoplasms Among Allogeneic Hematopoietic Cell Transplant Survivors in the United States

Kidney Outcomes after Hematopoietic Cell Transplantation in Children and Young Adults with Sickle Cell Disease

Memory T-Cell Add Back and Prophylactic blinatumomab Post Tcrαβ Depleted Haploidentical Transplantation Results in Promising Outcomes in Pediatric Patients with High-Risk Acute Leukemia

Health-Related Quality-of-Life Improvements after Exagamglogene Autotemcel in Patients with Severe Sickle Cell Disease

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