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Maria Chitty-Lopez
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Journal Articles

RP-L301, a Lentiviral-Mediated Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase 2 Clinical Trial Design

Ami J Shah, José Luis López Lorenzo, Rachael F Grace, E J van Beers, M Bartels, Andreas Glenthoej, Susana Navarro, Lucia Llanos, Begoña Pérez de Camino Gaisse, Sol Sanchez, Josune Zubicaray, May Chien, Oscar Quintana Bustamante, M González-Vicent, June Iriondo, Miriam Zeini, Grace Choi, Eileen Nicoletti, Maria Chitty-Lopez, Gayatri R Rao, Juan Bueren, Jonathan D Schwartz, Jose Carlos Segovia, Julián Sevilla
Journal: Blood
Blood (2024) 144 (Supplement 1): 3583.1.
https://doi.org/10.1182/blood-2024-201734
Published: 2024
Journal Articles

International Clinical Consensus on Leukocyte Adhesion Deficiency-I: A Modified Delphi Analysis

Jennifer Heimall, Luis Ignacio González Granado, Claire Booth, Patrice Carter, Emily Matthews, Maria Chitty-Lopez, Miranda Bailey, Susan E Prockop, Andrew R Gennery, Jennifer W Leiding
Journal: Blood
Blood (2024) 144 (Supplement 1): 3639.
https://doi.org/10.1182/blood-2024-207939
Published: 2024
Journal Articles

Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) Outcomes in Leukocyte Adhesion Deficiency Type -I

Claire Booth, Patrick Gilbert, Luuk Gras, Hawazen Alsaedi, Amir Ali Hamidieh, Mor Seny Gueye, Mary Slatter, Ashrafsadat Mousavi, Amos Toren, Musa Karakukcu, Abdelghani Tbakhi, Polina Stepensky, Gulyuz Ozturk, Peter Bader, Henrik Sengeloev, Tayfun Güngör, R.F. Wynn, Franco Locatelli, Maria Chitty-Lopez, Joana E Matos, Stuart Turner, Gayatri R Rao, Jonathan D Schwartz, Michael H Albert, Bénédicte Neven
Journal: Blood
Blood (2024) 144 (Supplement 1): 5079.
https://doi.org/10.1182/blood-2024-203643
Published: 2024
Journal Articles

Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)

Claire Booth, Julián Sevilla, Gayatri R. Rao, Maria Chitty Lopez, Elena Almarza, Dayna Terrazas, Josune Zubicaray, Marta González Vicent, Kritika Chetty, Grainne O'Toole, Jinhua Xu-Bayford, Eileen Nicoletti, Augustine Fernandes, Caroline Kuo, Satiro de Oliveira, Theodore B. Moore, Grace Choi, Miriam Zeini, Cristina Mesa-Núñez, Adrian J. Thrasher, Juan A. Bueren, Jonathan D. Schwartz, Donald B. Kohn
Journal: Blood
Blood (2022) 140 (Supplement 1): 7774–7775.
https://doi.org/10.1182/blood-2022-158578
Published: 2022