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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

T-allo10 Immunotherapy Results in Enhanced Early Immune Reconstitution and Reduced GvHD with Excellent Clinical Outcomes Post Αβ-Depleted HSCT in Pediatric and Young Adult Hematologic Malignancy Patients

Alice Bertaina, Rosa Bacchetta, Alma-Martina Cepika, David C Shyr, Giulia Barbarito, Linda Oppizzi, Pauline Chen, Gopin Saini, Jennifer Lee, Karen Kristovich, Rajni Agarwal, Orly Klein, Kathryn Melsop, Keri Tate, Matthew Porteus, Maria Grazia Roncarolo

Blood (2024) 144 (Supplement 1): 3525.

https://doi.org/10.1182/blood-2024-194276

Published: 2024

Journal Articles

One Year Follow-up on the First Patient Treated with Nula-Cel: An Autologous CRISPR/Cas9 Gene Corrected CD34+ Cell Product to Treat Sickle Cell Disease

Blood (2023) 142 (Supplement 1): 5000.

https://doi.org/10.1182/blood-2023-188963

Published: 2023

Journal Articles

Radiation and Busulfan-Free Hematopoietic Stem Cell Transplantation Using Briquilimab (JSP191) Anti-CD117 Antibody-Conditioning, Transient Immunosuppression and TCRαβ + T-Cell/CD19 + B-Cell Depleted Haploidentical Grafts in Patients with Fanconi Anemia

Blood (2023) 142 (Supplement 1): 229.

https://doi.org/10.1182/blood-2023-178400

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

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.

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

Published: 2022

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.

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

Published: 2021

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.

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.

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

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.

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

Published: 2021

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

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.

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.

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.

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.

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.

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.

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

Published: 2015

NARROW

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

T-allo10 Immunotherapy Results in Enhanced Early Immune Reconstitution and Reduced GvHD with Excellent Clinical Outcomes Post Αβ-Depleted HSCT in Pediatric and Young Adult Hematologic Malignancy Patients

One Year Follow-up on the First Patient Treated with Nula-Cel: An Autologous CRISPR/Cas9 Gene Corrected CD34+ Cell Product to Treat Sickle Cell Disease

Radiation and Busulfan-Free Hematopoietic Stem Cell Transplantation Using Briquilimab (JSP191) Anti-CD117 Antibody-Conditioning, Transient Immunosuppression and TCRαβ + T-Cell/CD19 + B-Cell Depleted Haploidentical Grafts in Patients with Fanconi Anemia

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

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

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

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

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

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

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

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

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American Society of Hematology

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American Society of Hematology

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