Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Severe Sickle Cell Disease-Reference-Cited by-同舟云学术

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

Published:2022-11-15 Issue:Supplement 1 Volume:140 Page:29-31
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Frangoul Haydar¹,Locatelli Franco²,Bhatia Monica³,Mapara Markus Y.⁴,Molinari Lyndsay⁵,Sharma Akshay⁶,Lobitz Stephan⁷,de Montalembert Mariane⁸,Rondelli Damiano⁹,Steinberg Martin¹⁰,Walters Mark C.¹¹,Imren Suzan¹²,Zhang Lanju¹²,Sharma Anjali¹³,Song Yang¹²,Simard Christopher¹²,Hobbs William¹²,Grupp Stephen¹⁴

Affiliation:

1. 1Sarah Cannon Center Research Institute and The Children's Hospital at TriStar Centennial, Nashville, TN

2. 2Department of Pediatric Hematology and Oncology, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome, Italy

3. 3Department of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY

4. 4Herbert Irving Comprehensive Cancer Center, Columbia University, New York, NY

5. 5Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX

6. 6Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN

7. 7Gemeinschaftsklinikum Mittelrhein, Koblenz, Germany

8. 8Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), University of Paris, Paris, France

9. 9University of Illinois at Chicago, Chicago, IL

10. 10Boston University School of Medicine, Boston, MA

11. 11University of California San Francisco Benioff Children's Hospital, Oakland, CA

12. 12Vertex Pharmaceuticals Incorporated, Boston, MA

13. 13CRISPR Therapeutics, Cambridge, MA

14. 14Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Cited by 15 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. A budget impact analysis of gene therapy for sickle cell disease: an updated analysis;Blood Advances;2024-08-30

2. Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease;Molecular Therapy;2024-07

3. Viral and nonviral nanocarriers for in vivo CRISPR-based gene editing;Nano Research;2024-06-20

4. Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34⁺cell therapy to induce fetal hemoglobin for sickle cell disease;2024-05-02

5. Autologous gene therapy for hemoglobinopathies: From bench to patient’s bedside;Molecular Therapy;2024-05