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David A. Williams
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Journal Articles

UM171 Enhances Fitness and Engraftment of Gene Modified Hematopoietic Stem Cells from Sickle Cells Disease Patients

Boya Liu, Denise Klatt, Yu Zhou, John Manis, Guy Sauvageau, Christian Brendel, David A. Williams
Journal: Blood
Blood (2023) 142 (Supplement 1): 18.
https://doi.org/10.1182/blood-2023-182099
Published: 2023
Journal Articles

Septin11 and Septin 8 Play Critical and Overlapping Roles Murine Stem Cell Function

Safa F Mohamad, Meaghan McGuinness, Chad Everett Harris, David A. Williams
Journal: Blood
Blood (2023) 142 (Supplement 1): 5583.
https://doi.org/10.1182/blood-2023-181991
Published: 2023
Journal Articles

Patient and Parent-Reported Outcomes Post-Treatment with Shmir-Based Lentiviral Gene Therapy for Sickle Cell Disease

Lauren Jimenez-Kurlander, Pei-Chi Kao, Mira Magner, Emily Morris, Amy Federico, Theodore Moore, Satiro DeOliveira, Augustine Fernandes, Donald B. Kohn, Wendy B. London, David A. Williams, Erica B. Esrick
Journal: Blood
Blood (2023) 142 (Supplement 1): 3750.
https://doi.org/10.1182/blood-2023-186915
Published: 2023
Journal Articles

The Association of SEPTIN6 Mutations with Pediatric Myelodysplasia

Safa F Mohamad, Meaghan McGuinness, Gabriele Casirati, Chad Everett Harris, Raffaele Renella, Pietro Genovese, Eric Allenspach, David A. Williams
Journal: Blood
Blood (2023) 142 (Supplement 1): 1846.
https://doi.org/10.1182/blood-2023-182069
Published: 2023
Journal Articles

Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome

Clinical Trials & Observations
Roxane Labrosse, Julia I. Chu, Myriam A. Armant, John K. Everett, Danilo Pellin, Niharika Kareddy, Andrew L. Frelinger, Lauren A. Henderson, Amy E. O’Connell, Amlan Biswas, Jet Coenen-van der Spek, Alexandra Miggelbrink, Claudia Fiorini, Hriju Adhikari, Charles C. Berry, Vito Adrian Cantu, Johnson Fong, Jason Jaroslavsky, Derin F. Karadeniz, Quan-Zhen Li, Shantan Reddy, Aoife M. Roche, Chengsong Zhu, Jennifer S. Whangbo, Colleen Dansereau, Brenda Mackinnon, Emily Morris, Stephanie M. Koo, Wendy B. London, Safa Baris, Ahmet Ozen, Elif Karakoc-Aydiner, Jenny M. Despotovic, Lisa R. Forbes Satter, Akihiko Saitoh, Yuta Aizawa, Alejandra King, Mai Anh Thi Nguyen, Vy Do Uyen Vu, Scott B. Snapper, Anne Galy, Luigi D. Notarangelo, Frederic D. Bushman, David A. Williams, Sung-Yun Pai
Journal: Blood
Blood (2023) 142 (15): 1281–1296.
https://doi.org/10.1182/blood.2022019117
Published: 2023
Includes: Supplemental data
Journal Articles

Regulated Expression of GATA1 As a Gene Therapy Cure for Diamond-Blackfan Anemia

Richard A Voit, Xiaotian Liao, Blake Cohen, Myriam Armant, Elena Kamal, Mei-Mei Huang, William Clarke, David A. Williams, Akiko Shimamura, Vijay G. Sankaran
Journal: Blood
Blood (2022) 140 (Supplement 1): 986–987.
https://doi.org/10.1182/blood-2022-165848
Published: 2022
Journal Articles

Altered Cellular Sedimentation during Apheresis Impacts Outcomes of Peripheral Blood Stem Cell Collection Efficiency in Patients with Sickle Cell Disease

David G. Justus, Erica B. Esrick, Matthew M. Heeney, Ellen Proeung, Carly Howard, Carlo Brugnara, David A. Williams, John P Manis
Journal: Blood
Blood (2022) 140 (Supplement 1): 5715–5716.
https://doi.org/10.1182/blood-2022-170450
Published: 2022
Journal Articles

Eltrombopag in Pediatric Patients with Previously Untreated or Refractory/Relapsed Severe Aplastic Anemia: The Phase II Escalate Trial

Akiko Shimamura, Alexey Maschan, Carolyn Bennett, Jason E Farrar, Sujith Samarasinghe, Brigitte Strahm, Winfred C. Wang, Adrianna Vlachos, Charlotte M. Niemeyer, Timothy S. Olson, Denise D'Alessio, Elise Burmeister Getz, Tomasz Lawniczek, Yunnan Xu, David A. Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 705–707.
https://doi.org/10.1182/blood-2022-158214
Published: 2022
Journal Articles

Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial

Erica B. Esrick, Amy Federico, Daniela Abriss, Myriam Armant, Kari Boardman, Christian Brendel, Marioara-Felicia Ciuculescu, Heather Daley, Colleen Dansereau, Augustine Fernandes, Matthew M. Heeney, David G. Justus, Pei-Chi Kao, Annette S. Kim, Donald B. Kohn, Leslie E. Lehmann, Donghui Liu, Wendy B. London, John P Manis, Theodore B. Moore, Emily Morris, Danilo Pellin, Ellen Proeung, Shanna Richard, Gavin D. Roach, Kit L. Shaw, Dayna Terrazas, Shanna L White, David A. Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 10665–10667.
https://doi.org/10.1182/blood-2022-169089
Published: 2022
Journal Articles

Combining the Anti-Sickling βAS3-Globin Gene with BCL11A and ZNF410 Double Shmirs for the Treatment of Sickle Cell Disease

Boya Liu, Kevyn Hart, Christian Brendel, Donald B. Kohn, David A. Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 7786–7787.
https://doi.org/10.1182/blood-2022-163469
Published: 2022
Journal Articles

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

Sung-Yun Pai, Nisha Nagarsheth, Susan Prockop, Myriam Armant, Donald B. Kohn, Rebecca A. Marsh, Claire Booth, John K. Everett, Jack Bleesing, Deepak Chellapandian, Colleen Dansereau, Satiro de Oliveira, Wendy B. London, M. Angélica Marinovic, Theodore B. Moore, Matias Oleastro, Grainne O'Toole, Mark Vander Lugt, Luciano Urdinez, Kelly J. Walkovich, Jolan E. Walter, Axel Schambach, Adrian J. Thrasher, Frederic D. Bushman, David A Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 10643–10645.
https://doi.org/10.1182/blood-2022-159768
Published: 2022
Journal Articles

Septin-6 Regulates Murine and Human Hematopoiesis, and Its Dysregulation Is Associated with Pediatric Myelodysplasia

Safa F Mohamad, Meaghan McGuinness, Gabriele Casirati, Alejo E Rodriguez-Fraticelli, Chad E. Harris, Fernando D. Camargo, Pietro Genovese, Eric J Allenspach, David A. Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 8588–8589.
https://doi.org/10.1182/blood-2022-163606
Published: 2022
Journal Articles

A Phase 2, Open-Label Study to Evaluate the Efficacy and Safety of Mgta-145 in Combination with Plerixafor for the Mobilization of Hematopoietic Stem Cells in Patients with Sickle Cell Anemia

Akshay Sharma, John P Manis, David G. Justus, David A. Williams, Sergey Efuni, Ji Hyun Lee, Erin Conlin, Shawn Rose, Jeffrey Humphrey, Kirk Bertelsen, Nicole Henry, June Li, Donald Pardy, David Santos, Christian Zdybowicz, Alexis Leonard, John Tisdale
Journal: Blood
Blood (2022) 140 (Supplement 1): 8263–8264.
https://doi.org/10.1182/blood-2022-159728
Published: 2022
Journal Articles

Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion

Claire Booth, Donald B. Kohn, Myriam Armant, Susan Prockop, Karen Buckland, Sharat Chandra, Shanmuganathan Chandrakasan, Kritika Chetty, Colleen Dansereau, Satiro de Oliveira, John Everett, Diego León Rico, Wendy B. London, Jin Hua Xu-Bayford, Theodore B. Moore, Nisha Nagarsheth, Suhag Parikh, Dayna Terrazas, Shanna L White, Axel Schambach, Frederic D. Bushman, Adrian J. Thrasher, David A. Williams, Sung-Yun Pai
Journal: Blood
Blood (2022) 140 (Supplement 1): 7770–7771.
https://doi.org/10.1182/blood-2022-159979
Published: 2022
Journal Articles

Updated 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

Mark C. Walters, Angela R. Smith, Gary J. Schiller, Erica B. Esrick, David A. Williams, Tatiana Gogoleva, Didier Rouy, Bettina M. Cockroft, Gregory M. Vercellotti
Journal: Blood
Blood (2021) 138 (Supplement 1): 3974.
https://doi.org/10.1182/blood-2021-147907
Published: 2021
Journal Articles

Combined +58 and +55 BCL11A enhancer Editing Yields Exceptional Efficiency, Specificity and HbF Induction in Human and NHP Preclinical Models

Jing Zeng, Selami Demirci, My Anh Nguyen, Linda Yingqi Lin, Stacy A. Maitland, Esther Mintzer, Yuxuan Wu, Danilo Pellin, Amornrat Tangprasittipap, Chokdee Vong, Shaina N. Porter, Kevin Luk, Pengpeng Liu, Varun Katta, Marioara-Felicia Ciuculescu, Daniela Abriss, Jonathan Hsu, Naoya Uchida, Khaled Essawi, Robert Donahue, Karl Petri, Vikram Pattanayak, Luca Pinello, Christian Brendel, David A Williams, John P Manis, Shengdar Q. Tsai, Shondra M. Pruett-Miller, J. Keith Joung, Duantida Songdej, Suradej Hongeng, Myriam Armant, Scot A. Wolfe, John F. Tisdale, Daniel E. Bauer
Journal: Blood
Blood (2021) 138 (Supplement 1): 1852.
https://doi.org/10.1182/blood-2021-152634
Published: 2021
Journal Articles

Effects of BCL11A Shmir-Induced Post-Transcriptional Silencing on Hemoglobin Polymer Inhibition in Single Red Blood Cells at Physiologic Oxygen Tension

Daniel C. De Souza, Erica B. Esrick, Nicolas Hebert, Giuseppe Di Caprio, Marioara Ciuculescu, Emily Morris, Myriam Armant, Bronner P. Gonçalves, Ethan Schonbrun, Christian Brendel, David K. Wood, Pablo Bartolucci, David A. Williams, John M. Higgins
Journal: Blood
Blood (2021) 138 (Supplement 1): 964.
https://doi.org/10.1182/blood-2021-147314
Published: 2021
Journal Articles

Effects of BCL11A Shmir-Induced Post-Transcriptional Silencing on Distributions of HbF in Single-RBCs and Reticulocytes

Nicolas Hebert, Erica B. Esrick, Myriam Armant, Christian Brendel, Marioara Felicia Ciuculescu, Etienne Audureau, John M. Higgins, David A. Williams, Pablo Bartolucci
Journal: Blood
Blood (2021) 138 (Supplement 1): 967.
https://doi.org/10.1182/blood-2021-150186
Published: 2021
Journal Articles

Clonal Tracking By Whole Genome Sequencing Permits Comprehensive Mapping of the Genomic Landscape in Pre- and Post-Gene Therapy Sickle Cell Patients

Alyssa H. Cull, Michael Spencer Chapman, Marioara Ciuculescu, Emily Mitchell, Myriam Armant, Grace Boyd, Erica B. Esrick, David A. Williams, Peter J. Campbell, David G. Kent
Journal: Blood
Blood (2021) 138 (Supplement 1): 559.
https://doi.org/10.1182/blood-2021-152262
Published: 2021
Journal Articles

Small Molecule Inhibition of PDE6D-RAS Interaction Suppresses the Growth of Acute Lymphoblastic Leukemia Cell Lines

Sara Canovas Nunes, Haiming Xu, Serena De Vita, Andrew Anighoro, Francois Autelitano, Edward Beaumont, Pamela Klingbeil, Meaghan McGuinness, Beatrice Duvert, Chad E. Harris, Lu Yang, Sheela Pangeni Pokharel, Chun-Wei David Chen, Monika Ermann, David A. Williams
Journal: Blood
Blood (2021) 138 (Supplement 1): 1199.
https://doi.org/10.1182/blood-2021-147009
Published: 2021