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Helene Vincon
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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

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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

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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

A combination of cyclophosphamide and interleukin-2 allows CD4 + T cells converted to Tregs to control scurfy syndrome

Free
Marianne Delville, Florence Bellier, Juliette Leon, Roman Klifa, Sabrina Lizot, Hélène Vinçon, Steicy Sobrino, Romane Thouenon, Armance Marchal, Alexandrine Garrigue, Juliette Olivré, Soëli Charbonnier, Chantal Lagresle-Peyrou, Mario Amendola, Axel Schambach, David Gross, Baptiste Lamarthée, Christophe Benoist, Julien Zuber, Isabelle André, Marina Cavazzana, Emmanuelle Six
Journal: Blood
Blood (2021) 137 (17): 2326–2336.
https://doi.org/10.1182/blood.2020009187
Published: 2021
Includes: Supplemental data