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Grainne O'Toole
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Journal Articles

Ex Vivo Lentiviral Hematopoietic Stem Cell (HSC) Gene Therapy May Represent a Curative Therapy for the Life-Threatening Inborn Error of Immunity Severe Combined Immunodeficiency Due to Adenosine Deaminase (ADA) Deficiency (ADA-SCID)

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Katelyn Masiuk, Jinhua Xu-Bayford, Augustine Fernandes, Konstantinos Vazouras, Danilo Pellin, Havinder Hara, Sohini Roy, Beatriz Campo Fernandez, Thao Thai Dang, Kimberly Gilmour, Elena Alvarez Mediavilla, Beatrice Curio-Penny, Gráinne O'Toole, Rima Ahmed, Jordyn Arnold, Elizabeth Garabedian, Harry L Malech, Kenneth Cornetta, Claire Booth, Donald B. Kohn
Journal: Blood
Blood (2024) 144 (Supplement 1): 1051.
https://doi.org/10.1182/blood-2024-202543
Published: 2024
Journal Articles

Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group a]: Updated Results from Global RP-L102 Clinical Trials

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Agnieszka D. Czechowicz, Julián Sevilla, Claire Booth, Josune Zubicaray, Paula Rio, Susana Navarro, Kritika Cherry, Gráinne O'Toole, Jinhua Xu-Bayford, Philip Ancliff, Elena Sebastián, Michael Rothe, Antonella L Bastone, Philipp John-Neek, Axel Schambach, Wei Wang, Grace Choi, Miriam Zeini, Eileen Nicoletti, Cindy Eide, John E Wagner, Gayatri R Rao, Adrian J. Thrasher, Maria-Grazia Roncarolo, Jonathan D Schwartz, Juan A Bueren, Rajni Agarwal
Journal: Blood
Blood (2024) 144 (Supplement 1): 7463.
https://doi.org/10.1182/blood-2024-194638
Published: 2024
Journal Articles

Lentiviral-mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials

Free
Agnieszka Czechowicz, Julián Sevilla, Claire Booth, Rajni Agarwal, Josune Zubicaray, Paula Río, Susana Navarro, Kritika Chetty, Grainne O'Toole, Jinhua Xu-Bayford, Philip Ancliff, Elena Sebastián, Grace Choi, Miriam Zeini, Eileen Nicoletti, John E Wagner, Gayatri R. Rao, Adrian J. Thrasher, Jonathan D. Schwartz, Maria Grazia Roncarolo, Juan A. Bueren
Journal: Blood
Blood (2022) 140 (Supplement 1): 10646–10647.
https://doi.org/10.1182/blood-2022-168342
Published: 2022
Journal Articles

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

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Sung-Yun Pai, Nisha Nagarsheth, Susan Prockop, Myriam Armant, Donald B. Kohn, Rebecca A. Marsh, Claire Booth, John K. Everett, Jack Bleesing, Deepak Chellapandian, Colleen Dansereau, Satiro de Oliveira, Wendy B. London, M. Angélica Marinovic, Theodore B. Moore, Matias Oleastro, Grainne O'Toole, Mark Vander Lugt, Luciano Urdinez, Kelly J. Walkovich, Jolan E. Walter, Axel Schambach, Adrian J. Thrasher, Frederic D. Bushman, David A Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 10643–10645.
https://doi.org/10.1182/blood-2022-159768
Published: 2022
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)

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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