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Journal Articles

Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither

Richard J. Jones, Michael R. DeBaun

Blood (2021) 138 (11): 942–947.

https://doi.org/10.1182/blood.2021011488

Published: 2021

Journal Articles

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

Lauri M. Burroughs, Aleksandra Petrovic, Ruta Brazauskas, Xuerong Liu, Linda M. Griffith, Hans D. Ochs, Jack J. Bleesing, Stephanie Edwards, Christopher C. Dvorak, Sonali Chaudhury, Susan E. Prockop, Ralph Quinones, Frederick D. Goldman, Troy C. Quigg, Shanmuganathan Chandrakasan, Angela R. Smith, Suhag Parikh, Blachy J. Dávila Saldaña, Monica S. Thakar, Rachel Phelan, Shalini Shenoy, Lisa R. Forbes, Caridad Martinez, Deepak Chellapandian, Evan Shereck, Holly K. Miller, Neena Kapoor, Jessie L. Barnum, Hey Chong, David C. Shyr, Karin Chen, Rolla Abu-Arja, Ami J. Shah, Katja G. Weinacht, Theodore B. Moore, Avni Joshi, Kenneth B. DeSantes, Alfred P. Gillio, Geoffrey D. E. Cuvelier, Michael D. Keller, Jacob Rozmus, Troy Torgerson, Michael A. Pulsipher, Elie Haddad, Kathleen E. Sullivan, Brent R. Logan, Donald B. Kohn, Jennifer M. Puck, Luigi D. Notarangelo, Sung-Yun Pai, David J. Rawlings, Morton J. Cowan

Blood (2020) 135 (23): 2094–2105.

https://doi.org/10.1182/blood.2019002939

Published: 2020

Journal Articles

Is there an optimal conditioning for older patients with AML receiving allogeneic hematopoietic cell transplantation?

Stefan O. Ciurea, Piyanuch Kongtim, Ankur Varma, Gabriela Rondon, Julianne Chen, Samer Srour, Qaiser Bashir, Amin Alousi, Rohtesh Mehta, Betul Oran, Uday Popat, Chitra Hosing, Amanda Olson, Naval Daver, Marina Konopleva, Richard E. Champlin

Blood (2020) 135 (6): 449–452.

https://doi.org/10.1182/blood.2019003662

Published: 2020

Includes: Supplemental data

Journal Articles

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Erica B. Esrick, MD, Maureen Achebe, MD MPH, Myriam Armant, PhD, Pablo Bartolucci, MD PhD, Marioara Felicia Ciuculescu, MD, Heather Daley, Colleen Dansereau, MSN, RN, CPN, Giuseppe Di Caprio, PhD, Bronner Goncalves, MD PhD, Nicolas Hebert, Matthew M Heeney, MD, John M. Higgins, MD PhD, Leslie E. Lehmann, MD, John P Manis, MD, Olivier Negre, PhD, Sarah Nikiforow, MD PhD, Ethan Schonbrun, PhD, David K. Wood, PhD, David A. Williams, MD

Blood (2019) 134 (Supplement_2): LBA-5.

https://doi.org/10.1182/blood-2019-132745

Published: 2019

Journal Articles

Profound Lymphopenia at the Time of ATG Administration Is Not Predictive of Survivals after Allotransplant Using Purine Analogue/Busulfan-Based Conditioning Regimen

Blood (2019) 134 (Supplement_1): 1985.

https://doi.org/10.1182/blood-2019-122013

Published: 2019

Journal Articles

Combination of a Cyclophosphamide-Total Body Irradiation Conditioning Regimen and Tacrolimus Plus Mycophenolate Mofetil for Gvhd Prophylaxis Is Associated with Higher HHV-6 Encephalitis Incidence after Cord Blood Transplantation

Takuto Mori, MD, Nobuhiro Hiramoto, MD, Ryosuke Ueda, MD PhD, Yoshimitsu Shimomura, Satoshi Yoshioka, MD PhD, Takayuki Ishikawa, MD PhD

Blood (2019) 134 (Supplement_1): 1986.

https://doi.org/10.1182/blood-2019-125209

Published: 2019

Journal Articles

Alternative Donor and Disease-Specific Conditioning Regimen Hematopoietic Stem Cell Transplantation for Inherited Bone Marrow Failure Syndromes

Fang Xu, Min Xiong, Ruijuan Sun, Yanli Zhao, Jianping Zhang, Xingyu Cao, Deyan Liu, Zhijie Wei, Jiarui Zhou, Yue Lu, Peihua Lu, Daopei Lu

Blood (2019) 134 (Supplement_1): 2047.

https://doi.org/10.1182/blood-2019-125695

Published: 2019

Journal Articles

Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) with Different Intensity Conditioning Regimens in Children with Very High Risk Acute Lymphoblastic Leukemia (VHR ALL): A Single-Center Experience

Elena Vladimirovna Semenova, MD PhD, Olesia V Paina, PhD, Zhemal Zarifovna Rakhmanova, Dr., Polina Valerievna Kozhokar, Dr., Anastasia S Frolova, Dr, Kirill Alexandrovich Ekushov, Dr., Tatiana A Bykova, MD PhD, Anna Osipova, Inna V Markova, Dr., Sergey N. Bondarenko, PhDMD, Ildar Barhatov, Aleksandr Alyanski, Elena Babenko, Ivan S Moiseev, MD PhD, Ludmila S Zubarovskaya, MD PhD professor, Axel Zander, Boris V Afanasyev, MD PhD professor

Blood (2019) 134 (Supplement_1): 2050.

https://doi.org/10.1182/blood-2019-128163

Published: 2019

Journal Articles

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Laura Breda, PhD, Valentina Ghiaccio, PhD, Silvia Pires Lourenco, MSc, Danuta Jadwiga Jarocha, PhD, Yasuhiro Ikawa, MD PhD, Naoto Tanaka, PhD, Virginia Guzikowski, Carla Casu, PhD, Kim Smith-Whitley, MD, Janet L. Kwiatkowski, MD MSCE, Stefano Rivella, PhD

Blood (2019) 134 (Supplement_1): 2242.

https://doi.org/10.1182/blood-2019-124043

Published: 2019

Journal Articles

Differential Alemtuzumab Dosage Effects in T-Cell Deplete Allogeneic Haematopoietic Stem Cell Transplants for Myeloid Malignancies- King's College Hospital London Experience

Varun Mehra, MBBS,MRCP,FRCPath, Daniele Avenoso, MD, Ximena Cabrera Castellano, Adrian Choy, Stefani Widya, Mili Shah, FRCPath, Shafqat Inam, MD, Shreyans Gandhi, MBBS,MDDNB,MRCP,FRCPath,MPhil, Austin Kulasekararaj, MD MRCP, FRCPath, Francesco Dazzi, PhD, Ghulam J. Mufti, MBBS, FRCP, FRCPath, DM, Antonio Pagliuca, MBBS, MA, FRCP, FRCPath, Hugues De Lavallade, Victoria Potter, MBBS, BSc, FRACP, FRCPA

Blood (2019) 134 (Supplement_1): 4622.

https://doi.org/10.1182/blood-2019-131239

Published: 2019

Journal Articles

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Blood (2019) 134 (Supplement_1): 4629.

https://doi.org/10.1182/blood-2019-126161

Published: 2019

Journal Articles

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Janet L. Kwiatkowski, MD MSCE, Alexis A. Thompson, MD MPH, John E.J. Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, MBBS DPhil FRACP FRCPA, Manfred Schmidt, PhD, Morris Kletzel, MD MBA, Elliott P. Vichinsky, MD, Briana Deary, Ying Chen, PhD, Alexandria Petrusich, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 4628.

https://doi.org/10.1182/blood-2019-125807

Published: 2019

Journal Articles

A Busulfan Following Cyclophosphamide and Constant Cell Dose Protocol Improved Results in Alternative Donor Hematologic Stem Cell Transplantation in Thalassemia Major: A Result from Long-Term Large-Size Multi-Center Study

Yuelin He, MD, Hua Jiang, MD PhD, Changgang Li, MD, Yiping Zhu, MD, Xuedong Wu, MD PhD, Liu Sixi, MD PhD, Qu Yuhua, MD, Jiangyu Liao, MD, Xiaoqin Feng, Huaying Liu, MD, Zhiyong Peng, MD, Lan He, MD, Chaoke Bu, MD, Gautam Mandeen, MD, Leung Wing, MD PhD, Chunfu Li, MD

Blood (2019) 134 (Supplement_1): 970.

https://doi.org/10.1182/blood-2019-130202

Published: 2019

Journal Articles

Post-Transplant Cyclophosphamide Improved Outcomes of HLA Mismatched Hematopoietic Stem Cell Transplantation in Children with β-Thalassemia Major

Jianyun Wen, Yuelin He, MD, Xiaoxiao Xu, Libai Chen, MD, Xuedong Wu, MD PhD, Xiaoqin Feng, Chunfu Li, Xuan Liu, Fuyu Pei

Blood (2019) 134 (Supplement_1): 801.

https://doi.org/10.1182/blood-2019-130038

Published: 2019

Journal Articles

Anti-CD19 CAR T Therapy Following Autologous HSCT May be Safe and Effective in Patients with Refractory Large B-Cell Lymphoma

Wei Liu, Wenyang Huang, Rui Lv, Shuhui Deng, MD, Shuhua Yi, MD, Lugui Qiu, MD PhD, Dehui Zou, MD

Blood (2019) 134 (Supplement_1): 784.

https://doi.org/10.1182/blood-2019-127659

Published: 2019

Journal Articles

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

Julie Kanter, MD, John F. Tisdale, MD, Markus Y. Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Wenmei Huang, PhD MS, Jean-Antoine Ribeil, MD PhD, Alexis A. Thompson, MD MPH, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 990.

https://doi.org/10.1182/blood-2019-128894

Published: 2019

Journal Articles

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, Andreas E. Kulozik, MD PhD, Evangelia Yannaki, MD, John B. Porter, MA MDFRCP FRCPath, Isabelle Thuret, MD, Martin G. Sauer, MD, Heidi Elliot, Ying Chen, PhD, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 815.

https://doi.org/10.1182/blood-2019-128482

Published: 2019

Journal Articles

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

Bernhard Gentner, MD, Maria Ester Bernardo, MD PhD, Francesca Tucci, MD, Erika Zonari, PhD, Francesca Fumagalli, MD, Silvia Pontesilli, MD, Serena Acquati, PhD, Paolo Silvani, MD, Fabio Ciceri, MD, Attilio Rovelli, MD, Giancarlo La Marca, PhD, Rossella Parini, MD, Silvia Gregori, PhD, Eugenio Montini, PhD, Luigi Naldini, MD PhD, Alessandro Aiuti, MD PhD

Blood (2019) 134 (Supplement_1): 607.

https://doi.org/10.1182/blood-2019-128805

Published: 2019

Journal Articles

A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates

Blood (2019) 134 (Supplement_1): 610.

https://doi.org/10.1182/blood-2019-125968

Published: 2019

Journal Articles

Fludarabine in Combination with Busulfan As Pretransplant Conditioning for Platelet Gene Therapy in Murine Hemophilia a with Inhibitors

Yingyu Chen, PhD, Jing Li, MD PhD, Jocelyn A. Schroeder, Weiqing Jing, MD PhD, Jianda Hu, PhD, Qizhen Shi, MD PhD

Blood (2019) 134 (Supplement_1): 1108.

https://doi.org/10.1182/blood-2019-127410

Published: 2019

NARROW

Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

Is there an optimal conditioning for older patients with AML receiving allogeneic hematopoietic cell transplantation?

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Profound Lymphopenia at the Time of ATG Administration Is Not Predictive of Survivals after Allotransplant Using Purine Analogue/Busulfan-Based Conditioning Regimen

Combination of a Cyclophosphamide-Total Body Irradiation Conditioning Regimen and Tacrolimus Plus Mycophenolate Mofetil for Gvhd Prophylaxis Is Associated with Higher HHV-6 Encephalitis Incidence after Cord Blood Transplantation

Alternative Donor and Disease-Specific Conditioning Regimen Hematopoietic Stem Cell Transplantation for Inherited Bone Marrow Failure Syndromes

Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) with Different Intensity Conditioning Regimens in Children with Very High Risk Acute Lymphoblastic Leukemia (VHR ALL): A Single-Center Experience

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Differential Alemtuzumab Dosage Effects in T-Cell Deplete Allogeneic Haematopoietic Stem Cell Transplants for Myeloid Malignancies- King's College Hospital London Experience

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

A Busulfan Following Cyclophosphamide and Constant Cell Dose Protocol Improved Results in Alternative Donor Hematologic Stem Cell Transplantation in Thalassemia Major: A Result from Long-Term Large-Size Multi-Center Study

Post-Transplant Cyclophosphamide Improved Outcomes of HLA Mismatched Hematopoietic Stem Cell Transplantation in Children with β-Thalassemia Major

Anti-CD19 CAR T Therapy Following Autologous HSCT May be Safe and Effective in Patients with Refractory Large B-Cell Lymphoma

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates

Fludarabine in Combination with Busulfan As Pretransplant Conditioning for Platelet Gene Therapy in Murine Hemophilia a with Inhibitors

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American Society of Hematology

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