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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

Synergistic Targeting of BTK and E-Selectin/CXCR4 in the Microenvironment of Mantle Cell Lymphomas

Mahesh Basyal, Weiguo Zhang, MD PhD, Nalini B Patel, Sujan Piya, PhD, Hongying Zhang, MD PhD, William E. Fogler, PhD, William G Rice, PhD, John L. Magnani, PhD, Gautam M. Borthakur, MD, Michael Andreeff, MD PhD

Blood (2019) 134 (Supplement_1): 3802.

https://doi.org/10.1182/blood-2019-126030

Published: 2019

Journal Articles

Trends in Autologous Transplantation for Myeloma in EBMT Centres between 1993 and 2017

Blood (2019) 134 (Supplement_1): 4575.

https://doi.org/10.1182/blood-2019-127166

Published: 2019

Journal Articles

Engraftment Syndrome in the Setting of Autologous Stem Cell Transplantation for Multiple Myeloma-a Single Institution Review of over 600 Patients

Sudarsan Kollimuttathuliam, MD, Phyllis McKiernan, David S. Siegel, Scott D. Rowley, MD, Noa Biran, MD, David H. Vesole, MD PhD, Themba Nyirenda, PhD, Andrew L. Pecora, MD, Stuart L. Goldberg, MD, Andre Goy, MD, Michele L. Donato, MD

Blood (2019) 134 (Supplement_1): 4576.

https://doi.org/10.1182/blood-2019-132052

Published: 2019

Journal Articles

The Impact of Different Mobilization Strategies in the Setting of Multiple Myeloma

Francesco Mazziotta, MD, Gabriele Buda, MD PhD, Nadia Cecconi, MD, Giulia Cervetti, MD, Lorenzo Iovino, MD, Enrico Orciuolo, MD PhD, Matteo Pelosini, MD PhD, Edoardo Benedetti, MD, Francesco Caracciolo, MD, Giovanni Carulli, MD, Sara Galimberti, MD PhD, Mario Petrini, MD PhD

Blood (2019) 134 (Supplement_1): 3254.

https://doi.org/10.1182/blood-2019-126118

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

Novel Evidence That the Pannexin 1 Channel Is Involved in Adenosine Triphosphate (ATP) Release from Cells for Optimal Mobilization of Hematopoietic Stem Progenitor Cells, and the Pannexin 1 SNP 5 (Rs3020015) T/C Polymorphism Characterizes Poor Mobilizer Status in Patients

Monika Cymer, MS, Mateusz Adamiak, PhD, Marta Skoda, PhD, Marta Antonina Libura, MD PhD, Elzbieta Urbanowska, PhD, Magdalena Kucia, PhD DSci, Mariusz Z Ratajczak, MD PhD DSc

Blood (2019) 134 (Supplement_1): 3248.

https://doi.org/10.1182/blood-2019-125883

Published: 2019

Journal Articles

Autologous Stem Cell Transfusions on Multiple Days in Patients with Multiple Myeloma - Does It Matter?

Thomas Pabst, MD, Sebastian Moser, Ulrike Bacher, Barbara Jeker, Behrouz Mansouri Taleghani, Prof., Urban Novak

Blood (2019) 134 (Supplement_1): 3252.

https://doi.org/10.1182/blood-2019-127940

Published: 2019

Journal Articles

Novel Evidence That the Nlrp3 Inflammasome Plays a Role in Bone Marrow As a "Cogwheel" Connecting Purinergic Signaling with Activation of the Complement Cascade to Induce "Sterile Inflammation", Which Is Required for Optimal Mobilization of Hematopoietic Stem/Progenitor Cells

Mateusz Adamiak, PhD, Anna Lenkiewicz, PhD, Kamila Bujko, MS, Arjun Thapa, PhD, Magdalena Kucia, PhD DSci, Ahmed Abdel-Latif, MD PhD, Janina Ratajczak, MD PhD DSci, Mariusz Z Ratajczak, MD PhD DSc

Blood (2019) 134 (Supplement_1): 4468.

https://doi.org/10.1182/blood-2019-125838

Published: 2019

Journal Articles

Novel Evidence That Extracellular Adenosine Triphosphate (ATP), As a Purinergic Signaling Mediator, Activates Mobilization By Engaging a P2X4 Ligand-Gated Cation Channel Receptor Expressed on the Surface of Hematopoietic and Innate Immunity Cells

Kamila Bujko, MS, Mateusz Adamiak, PhD, Arjun Thapa, PhD, Magdalena Kucia, PhD DSci, Janina Ratajczak, MD PhD DSci, Mariusz Z Ratajczak, MD PhD DSc

Blood (2019) 134 (Supplement_1): 4472.

https://doi.org/10.1182/blood-2019-125858

Published: 2019

Journal Articles

Oncostatin M Is a Novel Niche Factor That Restrains Hematopoietic Stem Cell Mobilization in Response to G-CSF and CXCR4 Antagonist Plerixafor

Kavita Bisht, PhD, Crystal McGirr, Kylie A Alexander, Whitney Fleming, Natalie Sims, Gerhard Müller- Newen, Ingrid G Winkler, PhD, Jean-Pierre Levesque, PhD

Blood (2019) 134 (Supplement_1): 4469.

https://doi.org/10.1182/blood-2019-127431

Published: 2019

Journal Articles

Delayed Neutrophil Engraftment in Patients Receiving Daratumumab As Part of Their First Induction Regimen for Multiple Myeloma

Abdullah S. Al Saleh, MBBS, M Hasib Sidiqi, MBBS, Morie A. Gertz, MD, Eli Muchtar, MD, Martha Q. Lacy, MD, Rahma M Warsame, MD, Wilson I Gonsalves, MD, Taxiarchis Kourelis, MD, William J Hogan, MB, BCh, Suzanne R. Hayman, MD, Prashant Kapoor, MD, Francis K. Buadi, MB, CHB, Angela Dispenzieri, MD, David Dingli, MD PhD, Shaji K. Kumar, MD

Blood (2019) 134 (Supplement_1): 4505.

https://doi.org/10.1182/blood-2019-131380

Published: 2019

Journal Articles

Incorporating Reduced-Dose Plerixafor to a Preemptive Mobilization Algorithm Increases Access to Autologous Transplantation in a Limited-Resource Setting

Andrés Gómez-De León, MD, Dalila Marisol Alvarado Navarro, MD, Cesar O Pezina Cantú, MD, Perla R. Colunga Pedraza, MD, David Gomez-Almaguer, MD, Rosario Salazar Riojas, Chem., Olga Graciela Cantu Rodriguez, MD, Luz del Carmen Tarín Arzaga, MD, Jose Carlos Jaime, MD PhD, Cesar Homero Gutierrez-Aguirre, PhD

Blood (2019) 134 (Supplement_1): 4476.

https://doi.org/10.1182/blood-2019-129516

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

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

Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing

Blood (2019) 134 (Supplement_1): 4632.

https://doi.org/10.1182/blood-2019-129394

Published: 2019

Journal Articles

Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing, Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells Derived from Sickle Cell Disease Patients

Samuel Lessard, PhD, Pauline Rimmele, PhD, Hui Ling, PhD, Kevin Moran, MSc, Benjamin Vieira, BS, Yi-Dong Lin, PhD, Vu Hong, PhD, Andreas Reik, PhD, Denny Dang, Naoya Uchida, MD, John F. Tisdale, MD, Pablo Rendo, MD, Ahmed Daak, PhD, Alexandra Hicks, PhD

Blood (2019) 134 (Supplement_1): 974.

https://doi.org/10.1182/blood-2019-126048

Published: 2019

Journal Articles

Phase 2 Trial of Daratumumab, Ixazomib, Lenalidomide and Modified Dose Dexamethasone in Patients with Newly Diagnosed Multiple Myeloma

Blood (2019) 134 (Supplement_1): 864.

https://doi.org/10.1182/blood-2019-131476

Published: 2019

Journal Articles

Daratumumab (DARA) Maintenance Therapy Improves Depth of Response and Results in Durable Progression-Free Survival (PFS) Following Dara Plus Cyclophosphamide, Bortezomib, and Dexamethasone (CyBorD) Induction Therapy in Multiple Myeloma (MM): Update of the Lyra Study

Robert M. Rifkin, MD, Jason M. Melear, MD, Edward Faber, DO, MS, William I. Bensinger, John M Burke, MD, Mohit Narang, MD, Don Stevens, MD, Sriya Gunawardena, Yana Lutska, PharmD, Keqin Qi, PhD, Jon Ukropec, PhD, Ming Qi, MD PhD, Thomas S. Lin, MD PhD, Habte A Yimer, MD

Blood (2019) 134 (Supplement_1): 863.

https://doi.org/10.1182/blood-2019-122497

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

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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

Synergistic Targeting of BTK and E-Selectin/CXCR4 in the Microenvironment of Mantle Cell Lymphomas

Trends in Autologous Transplantation for Myeloma in EBMT Centres between 1993 and 2017

Engraftment Syndrome in the Setting of Autologous Stem Cell Transplantation for Multiple Myeloma-a Single Institution Review of over 600 Patients

The Impact of Different Mobilization Strategies in the Setting of Multiple Myeloma

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

Novel Evidence That the Pannexin 1 Channel Is Involved in Adenosine Triphosphate (ATP) Release from Cells for Optimal Mobilization of Hematopoietic Stem Progenitor Cells, and the Pannexin 1 SNP 5 (Rs3020015) T/C Polymorphism Characterizes Poor Mobilizer Status in Patients

Autologous Stem Cell Transfusions on Multiple Days in Patients with Multiple Myeloma - Does It Matter?

Novel Evidence That the Nlrp3 Inflammasome Plays a Role in Bone Marrow As a "Cogwheel" Connecting Purinergic Signaling with Activation of the Complement Cascade to Induce "Sterile Inflammation", Which Is Required for Optimal Mobilization of Hematopoietic Stem/Progenitor Cells

Novel Evidence That Extracellular Adenosine Triphosphate (ATP), As a Purinergic Signaling Mediator, Activates Mobilization By Engaging a P2X4 Ligand-Gated Cation Channel Receptor Expressed on the Surface of Hematopoietic and Innate Immunity Cells

Oncostatin M Is a Novel Niche Factor That Restrains Hematopoietic Stem Cell Mobilization in Response to G-CSF and CXCR4 Antagonist Plerixafor

Delayed Neutrophil Engraftment in Patients Receiving Daratumumab As Part of Their First Induction Regimen for Multiple Myeloma

Incorporating Reduced-Dose Plerixafor to a Preemptive Mobilization Algorithm Increases Access to Autologous Transplantation in a Limited-Resource Setting

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

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

Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing

Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing, Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells Derived from Sickle Cell Disease Patients

Phase 2 Trial of Daratumumab, Ixazomib, Lenalidomide and Modified Dose Dexamethasone in Patients with Newly Diagnosed Multiple Myeloma

Daratumumab (DARA) Maintenance Therapy Improves Depth of Response and Results in Durable Progression-Free Survival (PFS) Following Dara Plus Cyclophosphamide, Bortezomib, and Dexamethasone (CyBorD) Induction Therapy in Multiple Myeloma (MM): Update of the Lyra Study

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

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