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Ami Shah
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Journal Articles

Correction of ineffective erythropoiesis and durable clinical benefit with exagamglogene autotemcel for transfusion-dependent β-thalassemia

Free
Franco Locatelli, Roland Meisel, Selim Corbacioglu, Josu de la Fuente, Mattia Algeri, Joachim Rupprecht, Kevin Kuo, Ami Shah, Peter Lang, Hayley Merkeley, Ben Carpenter, Markus Mapara, Robert Liem, Stephan Grupp, Yogi Chopra, Amanda Li, Janet Kwiatkowski, Melanie Kirby-Allen, Maria Domenica Cappellini, Antonis Kattamis, Sujit Sheth, Kevin Boerner, Sakellarios Zairis, Tina Liu, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2025) 146 (Supplement 1): 6089.
https://doi.org/10.1182/blood-2025-6089
Published: 2025
Journal Articles

Pre-transplant CAR T-cell therapy is associated with inferior survival and increased non-relapse mortality in pediatric B-ALL: A single-center retrospective analysis

Free
Rozalyn Chok, Minxuan Huang, Catherine Aftandilian, Liora Schultz, David Shyr, Norman Lacayo, Ami Shah, Raya Saab, Tanja Gruber, Crystal Mackall, Alice Bertaina, Kara Davis, Orly Klein
Journal: Blood
Blood (2025) 146 (Supplement 1): 6077.
https://doi.org/10.1182/blood-2025-6077
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

Free
Stephan Grupp, Franco Locatelli, Akshay Sharma, Monica Bhatia, Markus Mapara, Robert Liem, Laurence Dedeken, Lyndsay Molinari, Michael Eckrich, Kevin Kuo, Paul Telfer, Ami Shah, Marina Cavazzana, Selim Corbacioglu, Mariane de Montalembert, Stephan Lobitz, Damiano Rondelli, Martin Steinberg, Mark Walters, Kevin Boerner, Tina Liu, Suzan Imren, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2025) 146 (Supplement 1): 2537.
https://doi.org/10.1182/blood-2025-2537
Published: 2025
Journal Articles

Anti-Fungal Prophylaxis Using Intermediate Dose Ambisome Is Associated with Delayed Methotrexate Clearance in Pediatric Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Free
Liora M. Schultz, MD, Karan Kumar, MD, Suzette Stone, Elizabeth Callard, Jessica Witkowski, MD, Leigh Shinn, Lisa Pinner, Shizuka Franklin, Julianna Kula, Namrata Patel, Prianka Kumar, Kenneth I. Weinberg, MD, Matthew Porteus, MD PhD, Rajni Agarwal, MD, Ami Shah, MD, Sandhya Kharbanda, MD
Journal: Blood
Blood (2016) 128 (22): 5762.
https://doi.org/10.1182/blood.V128.22.5762.5762
Published: 2016
Journal Articles

Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans

Free
Clinical Trials & Observations
Fabio Candotti, Kit L. Shaw, Linda Muul, Denise Carbonaro, Robert Sokolic, Christopher Choi, Shepherd H. Schurman, Elizabeth Garabedian, Chimene Kesserwan, G. Jayashree Jagadeesh, Pei-Yu Fu, Eric Gschweng, Aaron Cooper, John F. Tisdale, Kenneth I. Weinberg, Gay M. Crooks, Neena Kapoor, Ami Shah, Hisham Abdel-Azim, Xiao-Jin Yu, Monika Smogorzewska, Alan S. Wayne, Howard M. Rosenblatt, Carla M. Davis, Celine Hanson, Radha G. Rishi, Xiaoyan Wang, David Gjertson, Otto O. Yang, Arumugam Balamurugan, Gerhard Bauer, Joanna A. Ireland, Barbara C. Engel, Gregory M. Podsakoff, Michael S. Hershfield, R. Michael Blaese, Robertson Parkman, Donald B. Kohn
Journal: Blood
Blood (2012) 120 (18): 3635–3646.
https://doi.org/10.1182/blood-2012-02-400937
Published: 2012
Includes: Supplemental data
Journal Articles

Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report

Free
Brief Report
Barbara C. Engel, Greg M. Podsakoff, Joanna L. Ireland, E. Monika Smogorzewska, Denise A. Carbonaro, Kathy Wilson, Ami Shah, Neena Kapoor, Mirna Sweeney, Mark Borchert, Gay M. Crooks, Kenneth I. Weinberg, Robertson Parkman, Howard M. Rosenblatt, Shi-Qi Wu, Michael S. Hershfield, Fabio Candotti, Donald B. Kohn
Journal: Blood
Blood (2006) 109 (2): 503–506.
https://doi.org/10.1182/blood-2006-06-031476
Published: 2006