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Journal Articles

Initial Results from the BEACON Clinical Study: A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease with Severe Vaso-Occlusive Crises

Blood (2024) 144 (Supplement 1): 513.

https://doi.org/10.1182/blood-2024-204888

Published: 2024

Journal Articles

SAGES1: Clinical Translation of CRISPR Genome Editing Strategy to Induce Fetal Hemoglobin to Treat Sickle Cell Disease

Blood (2024) 144 (Supplement 1): 146.

https://doi.org/10.1182/blood-2024-211182

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

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

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

Antibiotic Resistance Genes Peak before Hematopoietic Stem Cell Transplant in Pediatric Blood Cancer Patients

Marygrace S Duggar, Davide Leardini, Edoardo Muratore, Akshay Sharma, Riccardo Masetti, Ellie S Margolis

Blood (2024) 144 (Supplement 1): 3499.

https://doi.org/10.1182/blood-2024-198671

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

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

Trial in Progress: Cureaa - a Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia, Blood and Marrow Transplant Clinical Trials Network (BMT CTN) #2207

Blood (2024) 144 (Supplement 1): 2134.1.

https://doi.org/10.1182/blood-2024-207952

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

Impact of Extramedullary Disease on the Outcomes after Allogeneic Hematopoietic Cell Transplantation in Children and Young Adults with Acute Myeloid Leukemia - a CIBMTR Analysis

Hemalatha Rangarajan, Deepak Chellapandian, Rasha Atshan, Kwang Wooahn, Akshay Sharma, Kirk R. Schultz, Parinda A. Mehta, Christine L. Phillips, Larisa Broglie, Prakash Satwani

Blood (2024) 144 (Supplement 1): 2412.

https://doi.org/10.1182/blood-2024-201848

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

How I Treat Sickle Cell Disease with Gene Therapy

Akshay Sharma

Blood blood.2024024519.

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

Published: 2024

Journal Articles

Impact of hematopoietic cell transplantation on myocardial fibrosis in young patients with sickle cell disease

Akshay Sharma, Subodh Selukar, Yu Bi, Anthony Merlocco, Cara E. Morin, Chris Goode, Parul Rai, Jeffrey A. Towbin, Jane S. Hankins, Stephen Gottschalk, Brandon Triplett, Jason N. Johnson

Blood (2024) 144 (6): 672–675.

https://doi.org/10.1182/blood.2023023028

Published: 2024

Includes: Supplemental data

Journal Articles

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

Akshay Sharma, Haydar Frangoul, Markus Mapara, Suzan Imren, Nanxin Li, Tina Liu, Jaime Rubin, Daoyuan Shi, William Hobbs, Stephan Grupp

Blood (2023) 142 (Supplement 1): 4999.

https://doi.org/10.1182/blood-2023-190203

Published: 2023

Journal Articles

Exagamglogene Autotemcel for Severe Sickle Cell Disease

Blood (2023) 142 (Supplement 1): 1052.

https://doi.org/10.1182/blood-2023-190139

Published: 2023

Journal Articles

Hematopoietic Stem Cells from Mice and Individuals with Sickle Cell Disease Display Premature Senescence and Loss of Function That Is Targetable By Senolytic Therapy

Aditya Barve, Mahmoud Dabbah, Emilia Kooienga, Esther A. Obeng, Jacquelyn Myers, Akshay Sharma, Shannon McKinney-Freeman

Blood (2023) 142 (Supplement 1): 33.

https://doi.org/10.1182/blood-2023-190264

Published: 2023

NARROW

Initial Results from the BEACON Clinical Study: A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease with Severe Vaso-Occlusive Crises

SAGES1: Clinical Translation of CRISPR Genome Editing Strategy to Induce Fetal Hemoglobin to Treat Sickle Cell Disease

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

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

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

Antibiotic Resistance Genes Peak before Hematopoietic Stem Cell Transplant in Pediatric Blood Cancer Patients

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

Trial in Progress: Cureaa - a Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia, Blood and Marrow Transplant Clinical Trials Network (BMT CTN) #2207

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

Impact of Extramedullary Disease on the Outcomes after Allogeneic Hematopoietic Cell Transplantation in Children and Young Adults with Acute Myeloid Leukemia - a CIBMTR Analysis

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

How I Treat Sickle Cell Disease with Gene Therapy

Impact of hematopoietic cell transplantation on myocardial fibrosis in young patients with sickle cell disease

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

Exagamglogene Autotemcel for Severe Sickle Cell Disease

Hematopoietic Stem Cells from Mice and Individuals with Sickle Cell Disease Display Premature Senescence and Loss of Function That Is Targetable By Senolytic Therapy

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American Society of Hematology

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American Society of Hematology

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