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Journal Articles

Hematopoietic and Immunological Assessment in Fanconi Anemia after Ex Vivo Lentiviral FANCA Gene Therapy with RP-L102

Rofida Nofal, Yan Yi Chan, Sushmita Sen, Ulises Juarez Figueroa, Hana Willner, Matthias Felber, Mark Krampf, Supawat Thongthip, Grace Choi, Eileen Nicoletti, Jonathan D. Schwartz, Kenneth I. Weinberg, Alfredo Rodriguez, Rajni Agarwal, Maria Grazia Roncarolo, Agnieszka Czechowicz

Blood (2022) 140 (Supplement 1): 7772–7773.

DOI: https://doi.org/10.1182/blood-2022-157840

Published: 2022

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.

DOI: 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.

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

Published: 2022

Journal Articles

JSP191 As a Single-Agent Conditioning Regimen Results in Successful Engraftment, Donor Myeloid Chimerism, and Production of Donor Derived Naïve Lymphocytes in Patients with Severe Combined Immunodeficiency (SCID)

Blood (2021) 138 (Supplement 1): 554.

DOI: https://doi.org/10.1182/blood-2021-153074

Published: 2021

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.

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

Published: 2021

Journal Articles

Engineering Human Invariant Natural Killer T (iNKT) Cells to Overexpress Immunomodulatory Cytokines

Melissa Mavers, Derek Hollingsworth, Chaiyaporn Boonchalermvichian, Jeffrey Liu, Jeanette Baker, Teresa Lopes Ramos, Juliane K. Lohmeyer, Po-Yu Lin, Maria Grazia Roncarolo, Robert S. Negrin

Blood (2021) 138 (Supplement 1): 3888.

DOI: https://doi.org/10.1182/blood-2021-153975

Published: 2021

Journal Articles

Functional Immune Tolerance Induced By Sequential Hematopoietic Stem Cell-Solid Organ Transplantation

Alice Bertaina, Giulia Barbarito, Vasavi V. Ramachandran, Karen Kristovich, Elizabeth Amanda Lippner, Sahar Fathallah-Shaykh, Amira Al-Uzri, Ami J Shah, Geraldine Aubert, Priscila Ferreira Slepicka, Linda Oppizzi, Rajni Agarwal, Maria Grazia Roncarolo, Amy Gallo, Waldo Concepcion, Kenneth I. Weinberg, Robertson Parkman, David B Lewis, Paul C Grimm

Blood (2021) 138 (Supplement 1): 1818.

DOI: https://doi.org/10.1182/blood-2021-149920

Published: 2021

Journal Articles

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

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

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

Published: 2021

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.

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

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.

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

Published: 2020

Journal Articles

First Report of Non-Genotoxic Conditioning with JSP191 (anti-CD117) and Hematopoietic Stem Cell Transplantation in a Newly Diagnosed Patient with Severe Combined Immune Deficiency

Blood (2020) 136 (Supplement 1): 10.

DOI: https://doi.org/10.1182/blood-2020-137762

Published: 2020

Journal Articles

Non-Genotoxic Anti-CD117 Antibody Conditioning Results in Successful Hematopoietic Stem Cell Engraftment in Patients with Severe Combined Immunodeficiency

Rajni Agarwal, MD, Christopher C. Dvorak, MD, Hye-Sook Kwon, PhD, Janel R Long-Boyle, PharmD, PhD, Susan S Prohaska, PhD MS, Janice W Brown, MD, Anne Le, Alyssa Guttman-Klein, Irving L Weissman, MD, Morton J Cowan, MD, Aaron C. Logan, MD PhD, Kenneth I. Weinberg, MD, Robertson Parkman, MD, Maria-Grazia Roncarolo, MD, Judith A Shizuru, MD PhD

Blood (2019) 134 (Supplement_1): 800.

DOI: https://doi.org/10.1182/blood-2019-126239

Published: 2019

Journal Articles

Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells

Agnieszka Czechowicz, MD PhD, Maria Grazia Roncarolo, MD, Brian C Beard, PhD, Ken Law, PhD, Eileen Nicoletti, MD, Paula Río, PhD, Juan A. Bueren, PhD, Jonathan D Schwartz, MD, Sandeep Soni, MBBS

Blood (2019) 134 (Supplement_1): 3350.

DOI: https://doi.org/10.1182/blood-2019-127352

Published: 2019

Journal Articles

Engineering Regenerative Thymic Tissues to Restore Long-Term T Cell Lymphopoiesis

Hui Gai, MD, Rafa Gras~Pena, PhD, Yogendra Verma, MS, Vincent Fateh, BS, Kazuya Ikeda, MD, Beruh Dejene, MS, Dullei Min, Jing Wang, MD, Tomek Swigut, PhD, Ken I. Weinberg, MD, Georg A. Hollander, MD, Sarah Heilshorn, PhD, Maria-Grazia Roncarolo, MD, Vittorio Sebastiano, PhD, Katja G. Weinacht, MD PhD

Blood (2018) 132 (Supplement 1): 5092.

DOI: https://doi.org/10.1182/blood-2018-99-117499

Published: 2018

Journal Articles

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

Blood (2016) 128 (1): 45–54.

DOI: https://doi.org/10.1182/blood-2016-01-688226

Published: 2016

Includes: Supplemental data

Journal Articles

Safety and Clinical Benefit of Lentiviral Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Blood (2015) 126 (23): 259.

DOI: https://doi.org/10.1182/blood.V126.23.259.259

Published: 2015

Journal Articles

Fatal autoimmunity in mice reconstituted with human hematopoietic stem cells encoding defective FOXP3

Blood (2015) 125 (25): 3886–3895.

DOI: https://doi.org/10.1182/blood-2014-12-618363

Published: 2015

Includes: Supplemental data

Journal Articles

In Vivo Tracking of T Cells in Humans Unveils Decade-Long Survival and Activity of Genetically Modified T Memory Stem Cells

Serena Scala, Luca Biasco, PhD, Luca Basso Ricci, Francesca Dionisio, Cristina Baricordi, Andrea Calabria, Stefania Giannelli, Nicoletta Cieri, MD PhD, Federica Barzaghi, Roberta Pajno, Hamoud Al-Mousa, Alessia Scarselli, Caterina Cancrini, Claudio Bordignon, MD, Maria Grazia Roncarolo, MD Prof., Eugenio Montini, Chiara Bonini, MD, Alessandro Aiuti, MD

Blood (2014) 124 (21): 547.

DOI: https://doi.org/10.1182/blood.V124.21.547.547

Published: 2014

Journal Articles

T-Cell-Mediated Suppression: From Bench to Bedside

Maria Grazia Roncarolo, MD, Manuela Battaglia, PhD, Rosa Bacchetta, MD, Megan Levings, PhD, Silvia Gregori, PhD

Blood (2013) 122 (21): SCI-37.

DOI: https://doi.org/10.1182/blood.V122.21.SCI-37.SCI-37

Published: 2013

Journal Articles

A novel function for FOXP3 in humans: intrinsic regulation of conventional T cells

Alicia N. McMurchy, Jana Gillies, Maria Concetta Gizzi, Michela Riba, Jose Manuel Garcia-Manteiga, Davide Cittaro, Dejan Lazarevic, Sara Di Nunzio, Ignazio S. Piras, Alessandro Bulfone, Maria Grazia Roncarolo, Elia Stupka, Rosa Bacchetta, Megan K. Levings

Blood (2013) 121 (8): 1265–1275.

DOI: https://doi.org/10.1182/blood-2012-05-431023

Published: 2013

Includes: Supplemental data

NARROW

Hematopoietic and Immunological Assessment in Fanconi Anemia after Ex Vivo Lentiviral FANCA Gene Therapy with RP-L102

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

JSP191 As a Single-Agent Conditioning Regimen Results in Successful Engraftment, Donor Myeloid Chimerism, and Production of Donor Derived Naïve Lymphocytes in Patients with Severe Combined Immunodeficiency (SCID)

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

Engineering Human Invariant Natural Killer T (iNKT) Cells to Overexpress Immunomodulatory Cytokines

Functional Immune Tolerance Induced By Sequential Hematopoietic Stem Cell-Solid Organ Transplantation

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

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

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

First Report of Non-Genotoxic Conditioning with JSP191 (anti-CD117) and Hematopoietic Stem Cell Transplantation in a Newly Diagnosed Patient with Severe Combined Immune Deficiency

Non-Genotoxic Anti-CD117 Antibody Conditioning Results in Successful Hematopoietic Stem Cell Engraftment in Patients with Severe Combined Immunodeficiency

Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells

Engineering Regenerative Thymic Tissues to Restore Long-Term T Cell Lymphopoiesis

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

Safety and Clinical Benefit of Lentiviral Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Fatal autoimmunity in mice reconstituted with human hematopoietic stem cells encoding defective FOXP3

In Vivo Tracking of T Cells in Humans Unveils Decade-Long Survival and Activity of Genetically Modified T Memory Stem Cells

T-Cell-Mediated Suppression: From Bench to Bedside

A novel function for FOXP3 in humans: intrinsic regulation of conventional T cells

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American Society of Hematology

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American Society of Hematology

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