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Josune Zubicaray
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Journal Articles

Decoding Transcriptomic Landscape and Molecular Pathways in DNAJC21 Deficiency

Francesco Saettini, Shreya Kanade, Fabiola Guerra, Benedetta Elena Di Majo, Francesca Vendemini, Federica Mottadelli, Grazia Fazio, Deborah D'Aliberti, Silvia Spinelli, Vincenzo L'Imperio, Albert Catala, Elena Laura Gonzalez-Forster, Maria Solsona, Josune Zubicaray, Julián Sevilla, Eva Galvez Villa, Andrea Biondi, Yigal Dror, Rocco Piazza
Journal: Blood
Blood (2024) 144 (Supplement 1): 5702.
https://doi.org/10.1182/blood-2024-209506
Published: 2024
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

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

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

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

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
Journal Articles

Lentiviral-mediated Gene Therapy for Adults and Children with Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study

Ami J. Shah, José Luis López Lorenzo, Julián Sevilla, Susana Navarro, Lucía Llanos, Begoña Pérez de Camino Gaisse, Sol Sanchez, Josune Zubicaray, Bert Glader, May Chien, Oscar Quintana Bustamante, Miriam Zeini, Grace Choi, Eileen Nicoletti, Gayatri R. Rao, Maria Grazia Roncarolo, Juan A. Bueren, Jonathan D. Schwartz, José C. Segovia
Journal: Blood
Blood (2022) 140 (Supplement 1): 4902–4903.
https://doi.org/10.1182/blood-2022-170948
Published: 2022
Journal Articles

Gene Therapy for Fanconi Anemia [Group A]: Interim Results of RP-L102 Clinical Trials

Agnieszka Czechowicz, Julián Sevilla, Rajni Agarwal, Claire Booth, Josune Zubicaray, Paula Río, Susana Navarro, Philip Ancliff, Elena Sebastian, Brian C Beard, Kenneth M Law, Grace Choi, Miriam Zeini, Camilla Duran-Persson, Eileen Nicoletti, John E Wagner, Gayatri R Rao, Adrian J. Thrasher, Jonathan D Schwartz, Juan A Bueren, Maria Grazia Roncarolo
Journal: Blood
Blood (2021) 138 (Supplement 1): 3968.
https://doi.org/10.1182/blood-2021-147071
Published: 2021