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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

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

Investigation of Cell-Contact Dependent Alterations in Immune Synapse Formation between T Cells and BCP-ALL Cells

Britta Wenske, PhD, Lothar Marischen, PhD, Julia Welzenbach, André Heimbach, PhD, Per Hoffmann, PhD, Selim Corbacioglu, MD, David A Williams, MD, Anja Troeger, MD

Blood (2019) 134 (Supplement_1): 2754.

https://doi.org/10.1182/blood-2019-128835

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

Interleukin-10 Signaling in Hematopoietic Stem and Progenitor Cells Maintains Stem Cell Function and Regulates Inflammation-Induced Myeloid Cell Output

Amanda McCabe, PhD, Christian Brendel, PhD, Sandra Frei, PhD, Denise Klatt, MSc, Martin Bentler, Scott Snapper, MD PhD, David A. Williams, MD

Blood (2018) 132 (Supplement 1): 2407.

https://doi.org/10.1182/blood-2018-99-116168

Published: 2018

Journal Articles

Padi4 Regulates NET Formation and Inflammatory Cell Death Downstream of Mlkl

Akshay A D'Cruz, PhD, Mary Speir, PhD, Meghan Bliss-Moreau, PhD, Sylvia Dietrich, PhD, Alyce A Chen, PhD, Mathilde Gavillet, MD PhD, Kate E Lawlor, PhD, James E Vince, PhD, Michelle Kelliher, PhD, Razq Hakem, PhD, Manolis Pasparakis, PhD, David A Williams, MD, Maria Ericsson, PhD, Ben A Croker, PhD

Blood (2018) 132 (Supplement 1): 276.

https://doi.org/10.1182/blood-2018-99-110051

Published: 2018

Journal Articles

Highly Efficient Therapeutic Gene Editing of BCL11A enhancer in Human Hematopoietic Stem Cells from ß-Hemoglobinopathy Patients for Fetal Hemoglobin Induction

Blood (2018) 132 (Supplement 1): 3482.

https://doi.org/10.1182/blood-2018-99-119365

Published: 2018

Journal Articles

Congenital X-Linked Myelodysplasia with Tetraploidy Is Associated with De Novo Germline C-Terminal Mutation of SEPT6, a Septin Filament Protein

Raffaele Renella, MD PhD, Katelyn Gagne, BS, Ellen Beauchamp, BA, Thorsten Schlaeger, PhD, Inga Hofmann, MD, Akiko Shimamura, MD PhD, Jonathan Fogel, BSc, Suneet Agarwal, MDPhD, Benjamin L. Ebert, MDPhD, Kyriacos Markianos, Timothy A. Springer, PhD, Mark D. Fleming, MD DPhil, David A. Williams, MD

Blood (2018) 132 (Supplement 1): 644.

https://doi.org/10.1182/blood-2018-99-114682

Published: 2018

Journal Articles

Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients

Blood (2018) 132 (Supplement 1): 1023.

https://doi.org/10.1182/blood-2018-99-116733

Published: 2018

Journal Articles

Deletion of Murine Rhoh induces More Aggressive Diffuse Large B Cell Lymphoma (DLBCL) Via Interaction with Kaiso and Regulation of BCL-6 Expression

Hiroto Horiguchi, MDPhD, Marioara Felicia Ciuculescu, MD, Anja Troeger, MD, Haiming Xu, PhD, Christian Brendel, PhD, David A. Williams, MD

Blood (2018) 132 (Supplement 1): 1574.

https://doi.org/10.1182/blood-2018-99-115238

Published: 2018

Journal Articles

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown, β-Globin Induction and Simultaneous Repression of β-Globin for the Potential Treatment of Sickle Cell Disease

Olivier Negre, PhD, Christian Brendel, PhD, Geoffrey B. Parsons, Min Luo, PhD, Myriam A Armant, PhD, Swaroopa Guda, PhD, Lauryn Christiansen, Gretchen Lewis, BMBS, Gabor Veres, PhD, Melissa Bonner, PhD, David A. Williams, MD

Blood (2017) 130 (Supplement 1): 3557.

https://doi.org/10.1182/blood.V130.Suppl_1.3557.3557

Published: 2017

Journal Articles

Outcomes of Immunosuppressive Therapy for Pediatric Aplastic Anemia: A North American Pediatric Aplastic Anemia Consortium (NAPAAC) Study

Blood (2017) 130 (Supplement 1): 1168.

https://doi.org/10.1182/blood.V130.Suppl_1.1168.1168

Published: 2017

Journal Articles

Deletion of Rhoh Accelerates Development of Diffuse Large B Cell Lymphoma in Bcl-6 Transgenic Mouse Model

Hiroto Horiguchi, MDPhD, Marioara Felicia Ciuculescu, MD, Christian Brendel, PhD, David A. Williams, MD

Blood (2017) 130 (Supplement 1): 1470.

https://doi.org/10.1182/blood.V130.Suppl_1.1470.1470

Published: 2017

Journal Articles

Preclinical Development of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1)

Valentina Poletti, PhD, Sabine Charrier, PhD, Samia Martin, Bernard Gjata, Alban Vignaud, PhD, Fang Zhang, PhD, Karen Buckland, PhD, Michael Rothe, PhD, Axel Schambach, MD PhD, Sung-Yun Pai, MD, David A. Williams, MD, H. Bobby Gaspar, MD PhD, Adrian J Thrasher, MD PhD, Fulvio Mavilio, PhD

Blood (2016) 128 (22): 4705.

https://doi.org/10.1182/blood.V128.22.4705.4705

Published: 2016

Journal Articles

Vav1, a Rho GTPase Guanine Exchange Factor (GEF) Is Required to Integrate gp130 Cytokine Signaling for Successful Engraftment of Murine Hematopoietic Stem Cells Following Irradiation

Serena De Vita, MD PhD, Yanhua Li, MD PhD, Chad Everett Harris, MS, Meaghan McGuinness, BS, David A. Williams, MD

Blood (2016) 128 (22): 2149.

https://doi.org/10.1182/blood.V128.22.2149.2149

Published: 2016

Journal Articles

Gene Therapy Using a Self-Inactivating Lentiviral Vector Improves Clinical and Laboratory Manifestations of Wiskott-Aldrich Syndrome

Blood (2015) 126 (23): 260.

https://doi.org/10.1182/blood.V126.23.260.260

Published: 2015

Journal Articles

Optimization of Bcl11a Knockdown By miRNA Scaffold Embedded Shrnas Leading to Enhanced Induction of Fetal Hemoglobin in Erythroid Cells for the Treatment of Beta-Hemoglobinopathies

Christian Brendel, PhD, Swaroopa Guda, PhD, Raffaele Renella, MD PhD, Peng Du, PhD, Daniel E. Bauer, MD, PhD, Matthew C. Canver, BS, Sophia C. Kamran, MD, James Thornton, PhD, Helen de Boer, BAS, Michael D. Milsom, PhD, Stuart H. Orkin, MD, Richard Gregory, PhD, David A. Williams, MD

Blood (2014) 124 (21): 2150.

https://doi.org/10.1182/blood.V124.21.2150.2150

Published: 2014

Journal Articles

The Role of Rac and Pak in Neutrophil Histone Hypercitrullination and Neutrophil Extracellular Traps Formation

Mathilde Gavillet, MD PhD, Kimberly Martinod, BS, BA, Denisa D. Wagner, PhD, David A. Williams, MD

Blood (2014) 124 (21): 462.

https://doi.org/10.1182/blood.V124.21.462.462

Published: 2014

Journal Articles

Full Donor Myeloid Engraftment with Minimal Toxicity in Dyskeratosis Congenita Patients Undergoing Allogeneic Bone Marrow Transplantation without Radiation or Alkylating Agents

Leslie E. Lehmann, MD, David A. Williams, MD, Wendy B. London, PhD, Suneet Agarwal, MD PhD

Blood (2014) 124 (21): 2941.

https://doi.org/10.1182/blood.V124.21.2941.2941

Published: 2014

Journal Articles

Pediatric Aplastic Anemia and Refractory Cytopenia of Childhood: A Retrospective Single Institutional Analysis to Determine Outcomes and Histomorphological Predictors

Craig Forester, Sarah E Sartain, MD, Dongjing Guo, MPH, Marian Harris, Olga Weinberg, Mark D. Fleming, MD DPhil, Wendy B. London, PhD, David A. Williams, MD, Inga Hofmann, MD

Blood (2014) 124 (21): 4382.

https://doi.org/10.1182/blood.V124.21.4382.4382

Published: 2014

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

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

Investigation of Cell-Contact Dependent Alterations in Immune Synapse Formation between T Cells and BCP-ALL Cells

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

Interleukin-10 Signaling in Hematopoietic Stem and Progenitor Cells Maintains Stem Cell Function and Regulates Inflammation-Induced Myeloid Cell Output

Padi4 Regulates NET Formation and Inflammatory Cell Death Downstream of Mlkl

Highly Efficient Therapeutic Gene Editing of BCL11A enhancer in Human Hematopoietic Stem Cells from ß-Hemoglobinopathy Patients for Fetal Hemoglobin Induction

Congenital X-Linked Myelodysplasia with Tetraploidy Is Associated with De Novo Germline C-Terminal Mutation of SEPT6, a Septin Filament Protein

Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients

Deletion of Murine Rhoh induces More Aggressive Diffuse Large B Cell Lymphoma (DLBCL) Via Interaction with Kaiso and Regulation of BCL-6 Expression

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown, β-Globin Induction and Simultaneous Repression of β-Globin for the Potential Treatment of Sickle Cell Disease

Outcomes of Immunosuppressive Therapy for Pediatric Aplastic Anemia: A North American Pediatric Aplastic Anemia Consortium (NAPAAC) Study

Deletion of Rhoh Accelerates Development of Diffuse Large B Cell Lymphoma in Bcl-6 Transgenic Mouse Model

Preclinical Development of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1)

Vav1, a Rho GTPase Guanine Exchange Factor (GEF) Is Required to Integrate gp130 Cytokine Signaling for Successful Engraftment of Murine Hematopoietic Stem Cells Following Irradiation

Gene Therapy Using a Self-Inactivating Lentiviral Vector Improves Clinical and Laboratory Manifestations of Wiskott-Aldrich Syndrome

Optimization of Bcl11a Knockdown By miRNA Scaffold Embedded Shrnas Leading to Enhanced Induction of Fetal Hemoglobin in Erythroid Cells for the Treatment of Beta-Hemoglobinopathies

The Role of Rac and Pak in Neutrophil Histone Hypercitrullination and Neutrophil Extracellular Traps Formation

Full Donor Myeloid Engraftment with Minimal Toxicity in Dyskeratosis Congenita Patients Undergoing Allogeneic Bone Marrow Transplantation without Radiation or Alkylating Agents

Pediatric Aplastic Anemia and Refractory Cytopenia of Childhood: A Retrospective Single Institutional Analysis to Determine Outcomes and Histomorphological Predictors

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American Society of Hematology

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American Society of Hematology

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