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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

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

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

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

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

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.

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.

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

Published: 2013

Journal Articles

Interleukin-10 Anergized Donor T Cell Infusion Improves Immune Reconstitution without Severe Graft-Versus-Host-Disease After Haploidentical Hematopoietic Stem Cell Transplantation.

Rosa Bacchetta, MD, Claudia Sartirana, Barbarella Lucarelli, Patrick Miqueu, Maria Teresa Lupo Stanghellini, MD, Raffaella Greco, M.D., Andrea Velardi, Franco Aversa, Massimo F Martelli, Silvia Gregori, Elisabetta Zappone, Katharina Fleischhauer, Massimo Bernardi, MD, Jacopo Peccatori, MD, Fabio Ciceri, MD, Maria Grazia Roncarolo, MD, Prof.

Blood (2009) 114 (22): 45.

https://doi.org/10.1182/blood.V114.22.45.45

Published: 2009

Journal Articles

Rapamycin-Based GvHD Prophylaxis Is Effective in T-Cell Replete Unmanipulated Haploidentical Peripheral Stem Cell Transplantation for Advanced Haematological Malignancies: Results in 46 Patients.

Blood (2009) 114 (22): 666.

https://doi.org/10.1182/blood.V114.22.666.666

Published: 2009

Journal Articles

Optimal Thalassemia Free Survival and Minimal Regimen Related Toxicity in 50 Consecutive Transplants of High Risk Beta Thalassemia Pediatric Patients Using Myelablative Therapy with Intravenous Busulphan.

Blood (2008) 112 (11): 3266.

https://doi.org/10.1182/blood.V112.11.3266.3266

Published: 2008

Journal Articles

Functional Correction of Lymphocytes and Dendritic Cells of Patients with WAS or XLT, Using Lentiviral Vectors Targeting the Expression of WAS Protein to Hematopoietic Cells.

Sabine Charrier, PhD, Loic Dupre, PhD, Mike Blundell, PhD, Samantha Scaramuzza, Laurence Jeanson, PhD, William Vainchenker, MD, Olivier Danos, PhD, Maria-Grazia Roncarolo, MD, Adrian Thrasher, MD, Anne Galy, PhD

Blood (2005) 106 (11): 3042.

https://doi.org/10.1182/blood.V106.11.3042.3042

Published: 2005

NARROW

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

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

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

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

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

Interleukin-10 Anergized Donor T Cell Infusion Improves Immune Reconstitution without Severe Graft-Versus-Host-Disease After Haploidentical Hematopoietic Stem Cell Transplantation.

Rapamycin-Based GvHD Prophylaxis Is Effective in T-Cell Replete Unmanipulated Haploidentical Peripheral Stem Cell Transplantation for Advanced Haematological Malignancies: Results in 46 Patients.

Optimal Thalassemia Free Survival and Minimal Regimen Related Toxicity in 50 Consecutive Transplants of High Risk Beta Thalassemia Pediatric Patients Using Myelablative Therapy with Intravenous Busulphan.

Functional Correction of Lymphocytes and Dendritic Cells of Patients with WAS or XLT, Using Lentiviral Vectors Targeting the Expression of WAS Protein to Hematopoietic Cells.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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