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Journal Articles

The feasibility of collecting social determinants of health in the national multi-center GRASP sickle cell gene therapy trial

Colleen Kelly, Morgan Paul, Wendy London, Rahela Aziz-Bose, Puja Umaretiya, Erica Esrick, Victoria Coleman-Cowger, Gary Schiller, Mehrdad Abedi, Joseph Antin, Mary Horowitz, Neena Kapoor, Edmund Waller, Sonali Chaudhury, Mary Eapen, Mark Walters, David Williams, Kira Bona

Blood (2025) 146 (Supplement 1): 6224.

https://doi.org/10.1182/blood-2025-6224

Published: 2025

Journal Articles

SEPTIN6 mutations in pediatric myelodysplastic syndrome disrupt nuclear actin organization

Xin Jiang, Safa Mohamad, Yu Zhou, Chad Harris, Jose Cancelas, David Williams

Blood (2025) 146 (Supplement 1): 4999.

https://doi.org/10.1182/blood-2025-4999

Published: 2025

Journal Articles

Long-term follow-up of the first in human post-transcriptional genetic silencing of BCL11A in sickle cell disease in a phase 1 pilot and feasibility trial

Blood (2025) 146 (Supplement 1): 2533.

https://doi.org/10.1182/blood-2025-2533

Published: 2025

Journal Articles

Structure guided targeting of the GATAD2A-CHD4 interaction in the MBD2-NuRD complex results in high levels of HbF

Shengzhe Shang, Glory Dan-Dukor, Sangam Goswami, Torry Li, He Wang, Christopher Travis, David Williams, Gordon Ginder

Blood (2025) 146 (Supplement 1): 731.

https://doi.org/10.1182/blood-2025-731

Published: 2025

Journal Articles

Sickle cell disease is associated with early-onset clonal hematopoiesis involving DNA damage response pathway mutations

Blood (2025) 146 (Supplement 1): 8.

https://doi.org/10.1182/blood-2025-8

Published: 2025

Journal Articles

Apheresis blood processing with heparin improves stem cell collection outcomes for sickle cell gene therapy

David Justus, Carly Howard, Ellen Proeung, Aimee Sarnie, Jen Fortin, Erica Esrick, Matthew Heeney, Daniel Bauer, Sarah Nikiforow, Jose Cancelas, Leslie Lehmann, David Williams, John Manis

Blood (2025) 146 (Supplement 1): 6096.

https://doi.org/10.1182/blood-2025-6096

Published: 2025

Journal Articles

Highly efficient collection and manufacture of autologous HSC gene therapy cell product for patients with sickle cell disease using a lentiviral vector containing a shmir targeting BCL11a

Blood (2025) 146 (Supplement 1): 6094.

https://doi.org/10.1182/blood-2025-6094

Published: 2025

Journal Articles

Refined design of a CD117 antibody-drug conjugate safely and effectively conditions non-human primates for autologous transplant-based gene therapy

Blood (2025) 146 (Supplement 1): 5871.

https://doi.org/10.1182/blood-2025-5871

Published: 2025

Journal Articles

Germline SEPT6 mutations in congenital neutropenia and myelodysplastic syndrome induce de novo protein function disrupting hematopoietic stem cell maintenance

Safa Mohamad, Meaghan McGuinness, Yu Zhou, Gabriele Casirati, Adele Mucci, Sara Canovas Nunes, Akiko Shimamura, Pietro Genovese, Eric Allenspach, David Williams

Blood (2025) 146 (Supplement 1): 6754.

https://doi.org/10.1182/blood-2025-6754

Published: 2025

Journal Articles

Expansion of Content in the Phenx Toolkit for Sickle Cell Disease Pain

Stephanie H Guarino, Mark D Nelms, David Williams, Amanda M. Brandow, C. Patrick Carroll, Nitya Bakshi, Claudia M Campbell, Deepika S. Darbari, Martha Kenney, Wally R Smith, Jennifer Stinson, William Zempsky, Wayne Huggins, Deborah Maiese, Tabitha Hendershot, Carol Hamilton

Blood (2022) 140 (Supplement 1): 8302–8304.

https://doi.org/10.1182/blood-2022-169277

Published: 2022

Journal Articles

Novel Class of Direct Thrombin Inhibitors Prevent Thrombosis By Inhibiting Fibrinogen Cleavage While Preserving Platelet Function

Mohan Sivaraja, PhD, Sivan Sizikov, PhD, Nichole Sandoval, Kenneth Lin, PhD, Lev Igoudin, Subhadra Dash, MSc, Chengpei Xu, MD PhD, Daniel Clemens, PhD, Angels Estiarte, PhD, Timothy Shiau, PhD, Kevin Short, PhD, David Williams, PhD, Anirban Datta, PhD, David Kita, PhD

Blood (2016) 128 (22): 3834.

https://doi.org/10.1182/blood.V128.22.3834.3834

Published: 2016

Journal Articles

A novel and essential role for FcγRIIa in cancer cell–induced platelet activation

Annachiara Mitrugno, David Williams, Steven W. Kerrigan, Niamh Moran

Blood (2014) 123 (2): 249–260.

https://doi.org/10.1182/blood-2013-03-492447

Published: 2014

Includes: Supplemental data

Journal Articles

High-Risk Multiple Myeloma Is Characterized by Uniform Over-Expression of Mirnas and Increased Copy Number and Expression of Argonaute 2 , A Master Regulator of Mirna Maturation and B-Cell Development.

Yiming Zhou, Lijuan Chen, Bart Barlogie, MD, PhD, Owen Stephens, PhD, Xiaosong Wu, PhD, David Williams, PhD, Marie-astrid Cartron, PhD, Frits van Rhee, MD, PhD, Bijay Nair, MD, Sarah Waheed, MD, Mauricio Pineda-Roman, MD, PhD, Yazan Alsayed, MD, PhD, Elias Anaissie, MD, PhD, John D Shaughnessy, Jr.

Blood (2009) 114 (22): 1804.

https://doi.org/10.1182/blood.V114.22.1804.1804

Published: 2009

Journal Articles

Changes in the Expression of Proteasome Genes in Tumor Cells Following Short-Term Proteasome Inhibitor Therapy Predicts Survival in Multiple Myeloma Treated with Bortezomib-Containing Multi-Agent Chemotherapy

John D. Shaughnessy, Jr., PhD, Pingping Qu, MD, Ricky Edmondson, PhD, Damir Herman, PhD, Yiming Zhou, PhD, Erming Tian, Frits van Rhee, MD, PhD, Mauricio Pineda-Roman, MD, PhD, Yazan Alsayed, Elias Anaissie, Ya-wei Qiang, Joshua Epstein, PhD, Shmuel Yaccoby, PhD, David Williams, Fenghuang Zhan, PhD, Owen Stephens, Antje Hoering, PhD, John Crowley, PhD, Bart Barlogie, MD, PhD

Blood (2008) 112 (11): 733.

https://doi.org/10.1182/blood.V112.11.733.733

Published: 2008

Journal Articles

Integration of DNA Copy Number and Gene Expression Alterations Reveal Novel Insights into the Molecular Pathogenesis and Prognosis of Multiple Myeloma

Yiming Zhou, PhD, Bart Barlogie, MD, PhD, Damir Herman, PhD, Owen Stephens, PhD, Erming Tian, PhD, David Williams, PhD, Marie-astrid Cartron, PhD, Frits van Rhee, MD, PhD, Mauricio Pineda-Roman, MD, PhD, Yazan Alsayed, MD, PhD, Elias Anaissie, MD, PhD, Jeffrey Sawyer, Ph.D., John D. Shaughnessy, Jr., PhD

Blood (2008) 112 (11): 250.

https://doi.org/10.1182/blood.V112.11.250.250

Published: 2008

Journal Articles

Loss of the Rho GTPase Activating Protein p190-B Enhances Hematopoietic Stem Cell Engraftment Potential.

Haiming Xu, Harmut Geiger, PhD, Kathleen Szczur, BS, Deidra Deira, MS, Yi Zheng, PhD, Jeffrey Settleman, PhD, David Williams, MD, Marie-Dominique Filippi, PhD

Blood (2007) 110 (11): 508.

https://doi.org/10.1182/blood.V110.11.508.508

Published: 2007

Journal Articles

microRNA Control of Hematopoietic Stem Cell Fitness and Myelopoiesis.

Chinavenmeni S. Velu, PhD, Sarah Porteous, PhD, Haiming Xu, Avinash M. Baktula, Philip Roehrs, MD, Tristan Bourdeau, Ed Stavnezer, PhD, David Williams, MD, Clemencia Colmenares, PhD, H. Leighton Grimes, PhD

Blood (2007) 110 (11): 373.

https://doi.org/10.1182/blood.V110.11.373.373

Published: 2007

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The feasibility of collecting social determinants of health in the national multi-center GRASP sickle cell gene therapy trial

SEPTIN6 mutations in pediatric myelodysplastic syndrome disrupt nuclear actin organization

Long-term follow-up of the first in human post-transcriptional genetic silencing of BCL11A in sickle cell disease in a phase 1 pilot and feasibility trial

Structure guided targeting of the GATAD2A-CHD4 interaction in the MBD2-NuRD complex results in high levels of HbF

Sickle cell disease is associated with early-onset clonal hematopoiesis involving DNA damage response pathway mutations

Apheresis blood processing with heparin improves stem cell collection outcomes for sickle cell gene therapy

Highly efficient collection and manufacture of autologous HSC gene therapy cell product for patients with sickle cell disease using a lentiviral vector containing a shmir targeting BCL11a

Refined design of a CD117 antibody-drug conjugate safely and effectively conditions non-human primates for autologous transplant-based gene therapy

Germline SEPT6 mutations in congenital neutropenia and myelodysplastic syndrome induce de novo protein function disrupting hematopoietic stem cell maintenance

Expansion of Content in the Phenx Toolkit for Sickle Cell Disease Pain

Novel Class of Direct Thrombin Inhibitors Prevent Thrombosis By Inhibiting Fibrinogen Cleavage While Preserving Platelet Function

A novel and essential role for FcγRIIa in cancer cell–induced platelet activation

High-Risk Multiple Myeloma Is Characterized by Uniform Over-Expression of Mirnas and Increased Copy Number and Expression of Argonaute 2 , A Master Regulator of Mirna Maturation and B-Cell Development.

Changes in the Expression of Proteasome Genes in Tumor Cells Following Short-Term Proteasome Inhibitor Therapy Predicts Survival in Multiple Myeloma Treated with Bortezomib-Containing Multi-Agent Chemotherapy

Integration of DNA Copy Number and Gene Expression Alterations Reveal Novel Insights into the Molecular Pathogenesis and Prognosis of Multiple Myeloma

Loss of the Rho GTPase Activating Protein p190-B Enhances Hematopoietic Stem Cell Engraftment Potential.

microRNA Control of Hematopoietic Stem Cell Fitness and Myelopoiesis.

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