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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

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

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

A New Step in Understanding of Fanconi Patients Peripheral Stem Cell Harvesting, a Bridge to Gene Therapy

Jean-Sebastien Diana, MD, Sandra Manceau, PhD, Thierry Leblanc, MD, Chloé Couzin, MD, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Matthieux Bendavid, MD, Laure Joseph, MD, Marianne Delville, MD, Stephane Blanche, MD, Jean Soulier, MD PhD, Marina Cavazzana, MD PhD, Francois Lefrere, MD

Blood (2019) 134 (Supplement_1): 1970.

https://doi.org/10.1182/blood-2019-128051

Published: 2019

Journal Articles

CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies

Blood (2019) 134 (Supplement_1): 2066.

https://doi.org/10.1182/blood-2019-131318

Published: 2019

Journal Articles

TEM-MM-101: A Phase I/IIa Dose Escalation Study Evaluating the Safety and Activity of Autologous CD34+ Enriched Hematopoietic Progenitor Cells Genetically Modified for Human Interferon-α2 in Multiple Myeloma Patients with Early Relapse after Intensive Front Line Therapy

Bernhard Gentner, MD, Francesca Farina, MD, Matteo Giovanni Carrabba, MD, Magda Marcatti, MD, Stefania Girlanda, MD, Gabriele Antonarelli, MD, Capotondo Alessia, PhD, Stefania Mazzoleni, PhD, Carlo Russo, MD, Luigi Naldini, MD PhD, Fabio Ciceri, MD

Blood (2019) 134 (Supplement_1): 2064.

https://doi.org/10.1182/blood-2019-123749

Published: 2019

Journal Articles

Targeting CXCR4, VLA4, and CXCR2 for Hematopoietic Stem Cell Mobilization

Daniel Cancilla, BS, Haresh Thakellapalli, Marvin J Meyers, PhD, Michael P. Rettig, PhD, Ezhilarasi Chendamarai, PhD, Stephanie Christ, MS, Darja Karpova, PhD, Peter G Ruminski, BS, Julie K. Ritchey, Dwight M. Morrow, PhD, Michael Prinsen, John F. DiPersio, MD PhD

Blood (2019) 134 (Supplement_1): 1916.

https://doi.org/10.1182/blood-2019-124373

Published: 2019

Journal Articles

Are We Choosing Mobilization Regimens for Autologous Stem Cell Transplantation in Multiple Myeloma Wisely?: A Single Centre Comparison of GCSF+/-Plerixafor Vs Cyclophosphamide/GCSF+/- Plerixafor

Chloe Yang, MD, Mina Dehghani, MD, Wilma Hopman, MSc, Sita D. Bhella, MD

Blood (2019) 134 (Supplement_1): 2136.

https://doi.org/10.1182/blood-2019-128819

Published: 2019

Journal Articles

R4 Rgs Subfamily Proteins Suppress Engraftment and Regulate SDF-1/CXCR4 Signaling in Human CD34+ Hematopoietic Stem/Progenitor Cells

Kathy Chan, PhD, Tsin Sik Wong, Mphil, Karen Li, PhD, Xiao-Bing Zhang, PhD, Ronald Wang, PhD, King Yiu Lee, PhD, Frankie Wai Tsoi Cheng, MD, Chi Kong Li, MBBS,MD, Kam Tong Leung, PhD

Blood (2019) 134 (Supplement_1): 1927.

https://doi.org/10.1182/blood-2019-129498

Published: 2019

Journal Articles

A Randomized Evaluation of Vinorelbine Versus Gemcitabine Chemotherapy Mobilization of Stem Cells in Myeloma Patients

Barbara Jeker, Sarah Farag, Urban Novak, Ulrike Bacher, Behrouz Mansouri Taleghani, Qiyu Li, Thomas Pabst, MD

Blood (2019) 134 (Supplement_1): 1963.

https://doi.org/10.1182/blood-2019-127848

Published: 2019

Journal Articles

Safe and Efficient Peripheral Blood Stem Cell Collection in Patients with Sickle Cell Disease Using Plerixafor

Alexis Leonard, MD, Naoya Uchida, MD, David F. Stroncek, MD, Sandhya R. Panch, MDMPH, Kamille West, Eoghan George Molloy, MD, Tom Hughes, Pharm.D., Sara Hauffe, RN, Tiffani Taylor, Courtney D. Fitzhugh, MD, Jane S. Hankins, MD MS, Megan Wilson, MSW, CCRP, Akshay Sharma, MBBS, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD, Matthew Hsieh, MD, John F. Tisdale, MD

Blood (2019) 134 (Supplement_1): 1964.

https://doi.org/10.1182/blood-2019-126091

Published: 2019

Journal Articles

Rapid and Robust Mobilization of CD34+ HSCs without G-CSF Following Administration of Mgta-145 Alone or in Combination with Plerixafor

John F. DiPersio, MD PhD, Jonathan Hoggatt, PhD, Steven Devine, MD, Lukasz Biernat, MD, Haley Howell, BS, BA, Veit Schmelmer, PhD, Jason R. Neale, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD, Kevin A. Goncalves, PhD, Glen D. Raffel, MD PhD, Patrick C. Falahee, PhD, Dwight M. Morrow, PhD, John C. Davis, Jr., MD MPH, MS

Blood (2019) 134 (Supplement_1): 1961.

https://doi.org/10.1182/blood-2019-127523

Published: 2019

Journal Articles

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

Alexis A. Thompson, MD MPH, Mark C. Walters, MD, Janet L. Kwiatkowski, MD MSCE, Suradej Hongeng, MD, John B. Porter, MA MDFRCP FRCPath, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Heidi Elliot, Ge Tao, PhD, Richard A. Colvin, MD PhD, Franco Locatelli, MD PhD

Blood (2019) 134 (Supplement_1): 3543.

https://doi.org/10.1182/blood-2019-126046

Published: 2019

Journal Articles

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Angela R. Smith, MD MS, Gary J. Schiller, MD, Gregory M Vercellotti, MD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Erica B. Esrick, MD, David A. Williams, MD, Weston P. Miller, MD, Adrian Woolfson, MD PhD, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 3544.

https://doi.org/10.1182/blood-2019-125743

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

Presence of Chip-Mutated Autologous Hematopoietic Cells in Mobilized Peripheral Blood Products Is Associated with Shorter Progression-Free Survival after Autologous Transplants for Multiple Myeloma

Ehsan Malek, MD, Petra Martin, Paolo F Caimi, MD, Benjamin K. Tomlinson, MD, Michael Caldwell, Gayathri Ravi, MD, Sowjanya Vuyyala, MD, Hannah Schmikla, RN, Robert Fox, Marcos de Lima, MD, Navid Sadri, MD, James Driscoll, MD PhD

Blood (2019) 134 (Supplement_1): 515.

https://doi.org/10.1182/blood-2019-127335

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

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

Blood (2019) 134 (Supplement_1): 608.

https://doi.org/10.1182/blood-2019-127439

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

Therapeutic Base Editing of Human Hematopoietic Stem Cells

Jing Zeng, MD, Yuxuan Wu, PhD, Chunyan Ren, PhD, Jasmine Bonanno, Anne H. Shen, Devlin Shea, Jason M Gehrke, PhD, John P Manis, MD, J. Keith Gehrke, Daniel E. Bauer, MD PhD

Blood (2019) 134 (Supplement_1): 612.

https://doi.org/10.1182/blood-2019-129173

Published: 2019

NARROW

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

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

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

A New Step in Understanding of Fanconi Patients Peripheral Stem Cell Harvesting, a Bridge to Gene Therapy

CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies

TEM-MM-101: A Phase I/IIa Dose Escalation Study Evaluating the Safety and Activity of Autologous CD34+ Enriched Hematopoietic Progenitor Cells Genetically Modified for Human Interferon-α2 in Multiple Myeloma Patients with Early Relapse after Intensive Front Line Therapy

Targeting CXCR4, VLA4, and CXCR2 for Hematopoietic Stem Cell Mobilization

Are We Choosing Mobilization Regimens for Autologous Stem Cell Transplantation in Multiple Myeloma Wisely?: A Single Centre Comparison of GCSF+/-Plerixafor Vs Cyclophosphamide/GCSF+/- Plerixafor

R4 Rgs Subfamily Proteins Suppress Engraftment and Regulate SDF-1/CXCR4 Signaling in Human CD34+ Hematopoietic Stem/Progenitor Cells

A Randomized Evaluation of Vinorelbine Versus Gemcitabine Chemotherapy Mobilization of Stem Cells in Myeloma Patients

Safe and Efficient Peripheral Blood Stem Cell Collection in Patients with Sickle Cell Disease Using Plerixafor

Rapid and Robust Mobilization of CD34+ HSCs without G-CSF Following Administration of Mgta-145 Alone or in Combination with Plerixafor

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

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

Presence of Chip-Mutated Autologous Hematopoietic Cells in Mobilized Peripheral Blood Products Is Associated with Shorter Progression-Free Survival after Autologous Transplants for Multiple Myeloma

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

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

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

Therapeutic Base Editing of Human Hematopoietic Stem Cells

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American Society of Hematology

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ASH Publications

American Society of Hematology

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