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Amy Federico
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Journal Articles

Long-term follow-up of the first in human post-transcriptional genetic silencing of BCL11A in sickle cell disease in a phase 1 pilot and feasibility trial

Free
Erica Esrick, Leslie Lehmann, Amy Federico, Heather Daley, Colleen Dansereau, Satiro DeOliveira, John Everett, Pei-Chi Kao, Boya Liu, Theodore Moore, Emily Morris, Helene Negre, Kit Shaw, Gavin Roach, Jerome Ritz, Aoife Doto, Olivia Silva, Helene Vincon, Frederic Bushman, Donald Kohn, Akshat Jain, Wendy London, David Justus, Myriam Armant, John Manis, David Williams
Journal: Blood
Blood (2025) 146 (Supplement 1): 2533.
https://doi.org/10.1182/blood-2025-2533
Published: 2025
Journal Articles

Highly efficient collection and manufacture of autologous HSC gene therapy cell product for patients with sickle cell disease using a lentiviral vector containing a shmir targeting BCL11a

Free
John Manis, David Justus, Erica Esrick, Edmund Waller, Mary Horowitz, Leslie Lehmann, Amy Federico, Colleen Dansereau, Emily Morris, Leah Cheng, Mehrdad Abedi, Michael Pulsipher, Neena Kapoor, Gary Schiller, Angela Treml, Sonali Chaudhury, Victoria Coleman-Cowger, Kit Shaw, Jerome Ritz, Heather Daley, Donald Kohn, Akshat Jain, Jeanne Hendrickson, Carly Howard, Aimee Sarnie, Ellen Proeung, Helene Vincon, Myriam Armant, Joseph Antin, Mark Walters, Mary Eapen, David Williams
Journal: Blood
Blood (2025) 146 (Supplement 1): 6094.
https://doi.org/10.1182/blood-2025-6094
Published: 2025
Journal Articles

Gene Therapy for Sickle Cell Disease Is Associated with a Decreased Transplant Length of Stay and Similar Rates of Short-Term Complications Compared to Allogeneic Matched Sibling Stem Cell Transplant: A Single Center Experience

Free
Lev Gorfinkel, Erica B. Esrick, Alexander Lake, Miki Nishitani, Malika Kapadia, Amy Federico, Colleen Dansereau, David A. Williams, Leslie E. Lehmann
Journal: Blood
Blood (2024) 144 (Supplement 1): 3574.
https://doi.org/10.1182/blood-2024-198677
Published: 2024
Journal Articles

Patient and Parent-Reported Outcomes Post-Treatment with Shmir-Based Lentiviral Gene Therapy for Sickle Cell Disease

Free
Lauren Jimenez-Kurlander, Pei-Chi Kao, Mira Magner, Emily Morris, Amy Federico, Theodore Moore, Satiro DeOliveira, Augustine Fernandes, Donald B. Kohn, Wendy B. London, David A. Williams, Erica B. Esrick
Journal: Blood
Blood (2023) 142 (Supplement 1): 3750.
https://doi.org/10.1182/blood-2023-186915
Published: 2023
Journal Articles

Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial

Free
Erica B. Esrick, Amy Federico, Daniela Abriss, Myriam Armant, Kari Boardman, Christian Brendel, Marioara-Felicia Ciuculescu, Heather Daley, Colleen Dansereau, Augustine Fernandes, Matthew M. Heeney, David G. Justus, Pei-Chi Kao, Annette S. Kim, Donald B. Kohn, Leslie E. Lehmann, Donghui Liu, Wendy B. London, John P Manis, Theodore B. Moore, Emily Morris, Danilo Pellin, Ellen Proeung, Shanna Richard, Gavin D. Roach, Kit L. Shaw, Dayna Terrazas, Shanna L White, David A. Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 10665–10667.
https://doi.org/10.1182/blood-2022-169089
Published: 2022