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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

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

Journal Articles

Myocardial Fibrosis Improves in Young Patients with Sickle Cell Disease after Hematopoietic Cell Transplantation

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

Blood (2023) 142 (Supplement 1): 2518.

https://doi.org/10.1182/blood-2023-178267

Published: 2023

Journal Articles

Cerebral Oxygen Metabolism Improves Following Hematopoietic Cell Transplantation in Children with Sickle Cell Disease

Chih-Chiang Chang, Matthew A. Scoggins, Ping Zou, Sydney Somogyi, Soniya Pinto, Kathleen Helton, Jane S Hankins, Brandon M Triplett, Akshay Sharma, Ranganatha Sitaram

Blood (2023) 142 (Supplement 1): 2519.

https://doi.org/10.1182/blood-2023-179363

Published: 2023

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

Impact of Publicly Reported Center Specific Analysis on Patient Selection Practices for Hematopoietic Stem Cell Transplantation

Christopher Strouse, Mark Juckett, Brent R. Logan, Noel Estrada-Merly, Jaime M. Preussler, Tony Truong, Jesse Troy, Nandita Khera, William Wood, Hemalatha G. Rangarajan, Luke Akard, Neel S. Bhatt, Akshay Sharma, Wael Saber

Blood (2023) 142 (Supplement 1): 373.

https://doi.org/10.1182/blood-2023-182013

Published: 2023

Journal Articles

Risk-Adapted Donor Lymphocyte Infusion after Hematopoietic Cell Transplant in Pediatric and Young Adult Patients Is Associated with Low Risk of Graft Versus Host Disease

Shruthi Suryaprakash, Yilun Sun, Li Tang, Dinesh Keerthi, Amr Qudeimat, Aimee C. Talleur, Ewelina K. Mamcarz, Ashok Srinivasan, Madden Renee, Rebecca Epperly, Swati Naik, Stephen Gottschalk, Brandon M Triplett, Akshay Sharma

Blood (2023) 142 (Supplement 1): 7042.

https://doi.org/10.1182/blood-2023-182639

Published: 2023

Journal Articles

Allogeneic Hematopoietic Cell Transplantation for Acute Megakaryocytic Leukemia: A Single Center Study

Shruthi Suryaprakash, Yu Bi, Subodh Selukar, Dinesh Keerthi, Amr Qudeimat, Aimee C. Talleur, Ewelina K. Mamcarz, Ashok Srinivasan, Madden Renee, Rebecca Epperly, Swati Naik, Stephen Gottschalk, Brandon M Triplett, Akshay Sharma

Blood (2023) 142 (Supplement 1): 7087.

https://doi.org/10.1182/blood-2023-179853

Published: 2023

Journal Articles

Diffuse myocardial fibrosis occurs in young patients with sickle cell anemia despite early disease-modifying therapy

Cara E. Morin, Akshay Sharma, Subodh Selukar, Gary Beasley, Anthony Merlocco, Chris Goode, Parul Rai, Jeffrey A. Towbin, Jane S. Hankins, Jason N. Johnson

Blood (2023) 141 (11): 1358–1362.

https://doi.org/10.1182/blood.2022018209

Published: 2023

Includes: Supplemental data

Journal Articles

A Phase 2, Open-Label Study to Evaluate the Efficacy and Safety of Mgta-145 in Combination with Plerixafor for the Mobilization of Hematopoietic Stem Cells in Patients with Sickle Cell Anemia

Akshay Sharma, John P Manis, David G. Justus, David A. Williams, Sergey Efuni, Ji Hyun Lee, Erin Conlin, Shawn Rose, Jeffrey Humphrey, Kirk Bertelsen, Nicole Henry, June Li, Donald Pardy, David Santos, Christian Zdybowicz, Alexis Leonard, John Tisdale

Blood (2022) 140 (Supplement 1): 8263–8264.

https://doi.org/10.1182/blood-2022-159728

Published: 2022

Journal Articles

Impact of Public Reporting of Center-Specific Analysis Scores on Hematopoietic Cell Transplant Center Volumes

Akshay Sharma, Brent Logan, Noel Estrada-Merly, Leslie E. Lehmann, Hemalatha G. Rangarajan, Jaime M. Preussler, Jesse D. Troy, Luke P. Akard, Neel S. Bhatt, Tony H. Truong, William A. Wood, Jr., Douglas Rizzo, Wael Saber

Blood (2022) 140 (Supplement 1): 10845–10846.

https://doi.org/10.1182/blood-2022-163451

Published: 2022

Journal Articles

Treatment of Individuals with Severe Sickle Cell Disease with OTQ923, an Autologous, Ex Vivo , CRISPR/Cas9-Edited, CD34+ Hematopoietic Stem and Progenitor Cell Product, Leads to Durable Engraftment and Fetal Hemoglobin Induction

Akshay Sharma, Jaap Jan Boelens, Maria I. Cancio, Jane Hankins, Prafulla A Bhad, Andrew Lewandowski, Xiaojun Zhao, Shripad Chitnis, Radhika Peddinti, Yan Zheng, Neena Kapoor, Fabio Ciceri, Jianping Yuan, Vionnie W. Yu, Susan C. Stevenson, Serena De Vita, James LaBelle

Blood (2022) 140 (Supplement 1): 1906–1908.

https://doi.org/10.1182/blood-2022-166254

Published: 2022

Journal Articles

Specific Cytogenetic Abnormalities at Diagnosis Predict Survival after Hematopoietic Cell Transplant in Poor-Risk Pediatric Acute Myeloid Leukemia: A PDWP/EBMT Study

Blood (2022) 140 (Supplement 1): 1882–1884.

https://doi.org/10.1182/blood-2022-162596

Published: 2022

Journal Articles

Machine Learning Approaches Incorporating High-Dimensional Longitudinal Data Improve the Prediction of Survival after Allogeneic Hematopoietic Cell Transplantation

Yiwang Zhou, Jesse Smith, Dinesh Keerthi, Suraj Sarvode Mothi, David C. Shyr, Barbara Spitzer, Avijit Chatterjee, Subrata Chatterjee, Swati Naik, Li Tang, Akshay Sharma

Blood (2022) 140 (Supplement 1): 322–323.

https://doi.org/10.1182/blood-2022-162890

Published: 2022

Journal Articles

Increased Potency and Uniformity of Fetal Hemoglobin Induction from Base Editing Compared to Cas9 Nuclease

Blood (2022) 140 (Supplement 1): 7784–7785.

https://doi.org/10.1182/blood-2022-162639

Published: 2022

Journal Articles

Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Severe Sickle Cell Disease

Haydar Frangoul, Franco Locatelli, Monica Bhatia, Markus Y. Mapara, Lyndsay Molinari, Akshay Sharma, Stephan Lobitz, Mariane de Montalembert, Damiano Rondelli, Martin Steinberg, Mark C. Walters, Suzan Imren, Lanju Zhang, Anjali Sharma, Yang Song, Christopher Simard, William Hobbs, Stephen Grupp

Blood (2022) 140 (Supplement 1): 29–31.

https://doi.org/10.1182/blood-2022-162353

Published: 2022

Journal Articles

Major ABO Incompatibility Significantly Influences the Survival and Outcomes after Allogeneic Hematopoietic Cell Transplantation in Leukemia - CIBMTR Analysis

Guru Subramanian Guru Murthy, Brent R. Logan, Stephanie Bo-Subait, Amer Beitingjaneh, Steven Devine, Nosha Farhadfar, Lohith Gowda, Shahrukh Hashmi, Hillard Lazarus, Sunita Nathan, Akshay Sharma, Jean A. Yared, Heather E. Stefanski, Michael A. Pulsipher, Jack W. Hsu, Galen E. Switzer, Sandhya R. Panch, Bronwen E. Shaw

Blood (2021) 138 (Supplement 1): 907.

https://doi.org/10.1182/blood-2021-150635

Published: 2021

Journal Articles

Patient and Caregiver Attitudes Towards Gene Therapy for Sickle Cell Disease: A Need for Partnership and Education

Akshay Sharma, Amanda Young, Yvonne Carroll, Belinda Mandrell, Mary Caples, Jami Gattuso, Lauren Jerkins, Lakiea Bailey, Benjami Wilfond, Yoram Unguru, Yian Guo, Yimei Li, Liza-Marie Johnson

Blood (2021) 138 (Supplement 1): 918.

https://doi.org/10.1182/blood-2021-148795

Published: 2021

NARROW

How I Treat Sickle Cell Disease with Gene Therapy

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

Myocardial Fibrosis Improves in Young Patients with Sickle Cell Disease after Hematopoietic Cell Transplantation

Cerebral Oxygen Metabolism Improves Following Hematopoietic Cell Transplantation in Children 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

Impact of Publicly Reported Center Specific Analysis on Patient Selection Practices for Hematopoietic Stem Cell Transplantation

Risk-Adapted Donor Lymphocyte Infusion after Hematopoietic Cell Transplant in Pediatric and Young Adult Patients Is Associated with Low Risk of Graft Versus Host Disease

Allogeneic Hematopoietic Cell Transplantation for Acute Megakaryocytic Leukemia: A Single Center Study

Diffuse myocardial fibrosis occurs in young patients with sickle cell anemia despite early disease-modifying therapy

A Phase 2, Open-Label Study to Evaluate the Efficacy and Safety of Mgta-145 in Combination with Plerixafor for the Mobilization of Hematopoietic Stem Cells in Patients with Sickle Cell Anemia

Impact of Public Reporting of Center-Specific Analysis Scores on Hematopoietic Cell Transplant Center Volumes

Treatment of Individuals with Severe Sickle Cell Disease with OTQ923, an Autologous, Ex Vivo , CRISPR/Cas9-Edited, CD34+ Hematopoietic Stem and Progenitor Cell Product, Leads to Durable Engraftment and Fetal Hemoglobin Induction

Specific Cytogenetic Abnormalities at Diagnosis Predict Survival after Hematopoietic Cell Transplant in Poor-Risk Pediatric Acute Myeloid Leukemia: A PDWP/EBMT Study

Machine Learning Approaches Incorporating High-Dimensional Longitudinal Data Improve the Prediction of Survival after Allogeneic Hematopoietic Cell Transplantation

Increased Potency and Uniformity of Fetal Hemoglobin Induction from Base Editing Compared to Cas9 Nuclease

Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Severe Sickle Cell Disease

Major ABO Incompatibility Significantly Influences the Survival and Outcomes after Allogeneic Hematopoietic Cell Transplantation in Leukemia - CIBMTR Analysis

Patient and Caregiver Attitudes Towards Gene Therapy for Sickle Cell Disease: A Need for Partnership and Education

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American Society of Hematology

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American Society of Hematology

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