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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

Blood (2024) 144 (Supplement 1): 5702.

https://doi.org/10.1182/blood-2024-209506

Published: 2024

Journal Articles

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

Blood (2024) 144 (Supplement 1): 7463.

https://doi.org/10.1182/blood-2024-194638

Published: 2024

Journal Articles

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

Blood (2024) 144 (Supplement 1): 3583.1.

https://doi.org/10.1182/blood-2024-201734

Published: 2024

Journal Articles

Emapalumab, a Fully Human Anti-Interferon Gamma Monoclonal Antibody, in Pediatric Patients with Primary Hemophagocytic Lymphohistiocytosis: Long-Term Follow-up of a Phase 2/3 Study

Michael B. Jordan, Carl Allen, Anupama Rao, Carmelo Rizzari, Simone Cesaro, Maria Caterina Putti, Julian Sevilla, Cristina de Min, Maria Ballabio, Veronica Asnaghi, Anna Stoltenberg, Radmila Kanceva, Franco Locatelli

Blood (2023) 142 (Supplement 1): 1174.

https://doi.org/10.1182/blood-2023-179362

Published: 2023

Journal Articles

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

Blood (2022) 140 (Supplement 1): 10646–10647.

https://doi.org/10.1182/blood-2022-168342

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

Blood (2022) 140 (Supplement 1): 4902–4903.

https://doi.org/10.1182/blood-2022-170948

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)

Blood (2022) 140 (Supplement 1): 7774–7775.

https://doi.org/10.1182/blood-2022-158578

Published: 2022

Journal Articles

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Interim Results of a Global Phase 1 Study for Adult and Pediatric Patients

Blood (2021) 138 (Supplement 1): 563.

https://doi.org/10.1182/blood-2021-148161

Published: 2021

Journal Articles

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

Blood (2021) 138 (Supplement 1): 3968.

https://doi.org/10.1182/blood-2021-147071

Published: 2021

Journal Articles

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

Donald B. Kohn, Claire Booth, Julián Sevilla, Gayatri R Rao, Elena Almarza, Dayna Terrazas, Eileen Nicoletti, Augustine Fernandes, Caroline Kuo, Satiro de Oliveira, Theodore B. Moore, Kenneth M Law, Brian C Beard, Grace Choi, Miriam Zeini, Cristina Mesa-Núñez, Adrian J. Thrasher, Juan A Bueren, Jonathan D Schwartz

Blood (2021) 138 (Supplement 1): 2932.

https://doi.org/10.1182/blood-2021-151898

Published: 2021

Journal Articles

Sensitivity Analysis of Overall Response Rate (ORR) with Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis (HLH)

Franco Locatelli, MD PhD, Michael B. Jordan, MD, Carl Allen, Simone Cesaro, MD, Carmelo Rizzari, Anupama Rao, Barbara Degar, MD, Tim Garrington, MD, Julian Sevilla, MD PhD, Maria Caterina Putti, MD, Franca Fagioli, MD, Martina Ahlmann, MD, Jose-Luis Dapena Diaz, Michael Henry, MD, Fabrizio De Benedetti, MD, Alexei Grom, Anna Stoltenberg, Mårten Vågerö, Cristina de Min

Blood (2020) 136 (Supplement 1): 14–15.

https://doi.org/10.1182/blood-2020-140692

Published: 2020

Journal Articles

Safety of Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis: Results of the Primary Analysis of the Pivotal Phase 2/3 Study

Blood (2020) 136 (Supplement 1): 24–25.

https://doi.org/10.1182/blood-2020-140766

Published: 2020

Journal Articles

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients

Blood (2020) 136 (Supplement 1): 47.

https://doi.org/10.1182/blood-2020-137246

Published: 2020

Journal Articles

Gene Therapy for Fanconi Anemia, Complementation Group a: Updated Results from Ongoing Global Clinical Studies of RP-L102

Agnieszka Czechowicz, MDPhD, Rajni Agarwal, MD, Julián Sevilla, MD PhD, Paula Río, PhD, Susana Navarro, PhD, Brian C Beard, PhD, Kenneth M Law, PhD, Grace Choi, BS, Miriam Zeini, PhD, Eileen Nicoletti, MD, John E Wagner, MD, Claire Booth, MBBS, PhD MSc, Jonathan D Schwartz, MD, Juan A Bueren, PhD, Maria Grazia Roncarolo, MD

Blood (2020) 136 (Supplement 1): 14.

https://doi.org/10.1182/blood-2020-137106

Published: 2020

Journal Articles

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1

Donald B. Kohn, MD, Gayatri R Rao, MD JD, Elena Almarza, PhD, Dayna Terrazas, RN, Eileen Nicoletti, MD, Augustine Fernandes, PhD, Caroline Kuo, MD, Satiro N. De Oliveira, MD, Theodore B. Moore, MD, Kenneth M Law, PhD, Brian C Beard, PhD, Julian Sevilla, MD PhD, Cristina Mesa-Núñez, PhD, Juan A Bueren, PhD, Jonathan D Schwartz, MD

Blood (2020) 136 (Supplement 1): 15.

https://doi.org/10.1182/blood-2020-142484

Published: 2020

Journal Articles

NHEJ-Mediated Gene Editing, a Versatile Approach to Correct a Variety of Fanconi Anemia Genes in HSCs

Francisco J Roman-Rodriguez, PhD, Laura Ugalde, Lara A. Alvarez, Begoña Díez-Cabezas, PhD, Jordi Surrallés, PhD, Maria Jose Ramirez, PhD, Massimo Bogliolo, PhD, Helmut Hanenberg, MD PhD, Julian Sevilla, MD PhD, Sandra Rodriguez-Perales, PhD, Raul Torres-Ruiz, PhD, Juan A. Bueren, PhD, Paula Rio, PhD

Blood (2019) 134 (Supplement_1): 4639.

https://doi.org/10.1182/blood-2019-129946

Published: 2019

Journal Articles

Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

Blood (2019) 134 (Supplement_1): 4627.

https://doi.org/10.1182/blood-2019-129686

Published: 2019

Journal Articles

Different Clinical Implications of Kinase Domain BCR-ABL1 Variants Detected in Chronic Myeloid Leukemia and Acute Lymphoblastic Leukemia Patients

Blood (2019) 134 (Supplement_1): 5368.

https://doi.org/10.1182/blood-2019-127069

Published: 2019

Journal Articles

Safety and Efficacy of Emapalumab in Pediatric Patients with Primary Hemophagocytic Lymphohistiocytosis

Franco Locatelli, Michael B. Jordan, MD, Carl E. Allen, MDPhD, Simone Cesaro, MD, Carmelo Rizzari, MD, Anupama Rao, MD, Barbara Degar, MD, Timothy Garrington, Julian Sevilla, MD PhD, Maria Caterina Putti, MD, Franca Fagioli, MD, Martina Ahlmann, MD, Jose-Luis Dapena, MD, Alexei A. Grom, MD, Fabrizio De Benedetti, MD, Walter Giovanni Ferlin, PhD, Maria Ballabio, MD, Cristina De Min, MD

Blood (2018) 132 (Supplement 1): LBA-6.

https://doi.org/10.1182/blood-2018-120810

Published: 2018

Journal Articles

Advances in the Gene Therapy of Patients with Fanconi Anemia

Blood (2018) 132 (Supplement 1): 1022.

https://doi.org/10.1182/blood-2018-99-111160

Published: 2018

NARROW

Decoding Transcriptomic Landscape and Molecular Pathways in DNAJC21 Deficiency

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

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

Emapalumab, a Fully Human Anti-Interferon Gamma Monoclonal Antibody, in Pediatric Patients with Primary Hemophagocytic Lymphohistiocytosis: Long-Term Follow-up of a Phase 2/3 Study

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

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

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)

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Interim Results of a Global Phase 1 Study for Adult and Pediatric Patients

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

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

Sensitivity Analysis of Overall Response Rate (ORR) with Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis (HLH)

Safety of Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis: Results of the Primary Analysis of the Pivotal Phase 2/3 Study

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients

Gene Therapy for Fanconi Anemia, Complementation Group a: Updated Results from Ongoing Global Clinical Studies of RP-L102

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1

NHEJ-Mediated Gene Editing, a Versatile Approach to Correct a Variety of Fanconi Anemia Genes in HSCs

Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

Different Clinical Implications of Kinase Domain BCR-ABL1 Variants Detected in Chronic Myeloid Leukemia and Acute Lymphoblastic Leukemia Patients

Safety and Efficacy of Emapalumab in Pediatric Patients with Primary Hemophagocytic Lymphohistiocytosis

Advances in the Gene Therapy of Patients with Fanconi Anemia

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American Society of Hematology

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ASH Publications

American Society of Hematology

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