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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.
Blood (2019) 133 (12): 1378–1381.
Includes: Supplemental data
Reduced Toxicity Conditioning Is Better Tolerated but Has Higher Graft Failure Compared to Myeloablative Conditioning in Children with Inherited Metabolic Disorders
Michael Downey, BA, Ashish O. Gupta, MBBS, MPH, Ryan Shanley, M.S, Weston P. Miller, MD, Paul Orchard, MD, Angela R. Smith, MD MS
Blood (2018) 132 (Supplement 1): 3352.
Repair of the Blood Brain Barrier and Neutrophil Recovery Following HSCT in Cerebral Adrenoleukodystrophy
Troy C Lund, MDMS, PhD, Weston P. Miller, MD, Dave Nascene, MD, Paul Orchard, MD, Ashish O. Gupta, MBBS, MPH
Blood (2018) 132 (Supplement 1): 4628.
Immune Mediated Cytopenias Following Allogeneic Hematopoietic Cell Transplantation for Non-Malignant Disease: Risk Factors and Treatment Response in the Pediatric Population
Robert T. Galvin, MD, Qing Cao, MS, Weston P. Miller, MD, Angela R. Smith, MD MS, Christen L. Ebens, MD MPH
Blood (2018) 132 (Supplement 1): 3377.
Su Han Lum, Weston P Miller, Simon Jones, Jean Mercer, Troy Lund, Denise Bonney, Paul J Orchard, Robert Wynn
Blood (2016) 128 (22): 4700.
Neurocognitive Trajectory of Patients with Childhood Cerebral Adrenoleukodystrophy Who Received Allogeneic Hematopoietic Cell Transplantation at an Early Stage of Cerebral Disease
Elizabeth Pierpont, PhD, Julie Eisengart, PhD, Richard Ziegler, PhD, Gerald Raymond, Paul J Orchard, Troy Lund, Weston P Miller
Blood (2016) 128 (22): 4682.
High-Exposure, Targeted Daily Busulfan and Fludarabine-Based Conditioning for Children Undergoing Hematopoietic Stem Cell Transplantation for Inherited Metabolic Disorders: Outcomes at a Single Center
Weston P Miller, Melisa Kay Stricherz, PharmD, Cathryn Jennissen, PharmD, Nicole Sando, Jennifer Danielson, Troy Lund, Paul J Orchard
Blood (2016) 128 (22): 2191.
The Impact of Graft/Recipient ABO Compatibility on Outcomes after Umbilical Cord Blood Transplant for Non-Malignant Disease
Matthew R Kudek, MD, Ryan Shanley, MS, Nicole D. Zantek, MD, PhD, Angela R. Smith, MD MS, Weston P Miller, IV, MD
Blood (2015) 126 (23): 4369.
Patient and Parental Perspectives on Long-Term Adaptive, Behavioral and Functional Outcomes Following Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders
Rachel Phelan, MD MPH, Paul J Orchard, MD, Margaret Semrud-Clikeman, PhD, Nicholas Smiley, Weston P Miller, IV, MD
Blood (2015) 126 (23): 4351.
Outcomes after Donor Lymphocyte Infusion for Insufficient Donor Chimerism Following Hematopoietic Cell Transplantation for Non-Malignant Disorders
Blood (2015) 126 (23): 1969.
Outcomes Following Allogeneic Hematopoietic Stem Cell Transplantation For Metachromatic Leukodystrophy
Alexander A Boucher, MD, Gerald V Raymond, MD, Ryan Shanley, Paul J Orchard, MD, Weston P Miller, IV, MD
Blood (2013) 122 (21): 714.
Cardiac QTc Interval Characteristics and Behavior in 995 Consecutive Hematopoietic Cell Transplantation Patients At a Single Institution
Blood (2011) 118 (21): 1967.
Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report
Weston P. Miller, Steven M. Rothman, David Nascene, Teresa Kivisto, Todd E. DeFor, Richard S. Ziegler, Julie Eisengart, Kara Leiser, Gerald Raymond, Troy C. Lund, Jakub Tolar, Paul J. Orchard
Blood (2011) 118 (7): 1971–1978.
GVHD after haploidentical transplantation: a novel, MHC-defined rhesus macaque model identifies CD28 − CD8 + T cells as a reservoir of breakthrough T-cell proliferation during costimulation blockade and sirolimus-based immunosuppression
Weston P. Miller, Swetha Srinivasan, Angela Panoskaltsis-Mortari, Karnail Singh, Sharon Sen, Kelly Hamby, Taylor Deane, Linda Stempora, Jonathan Beus, Alexa Turner, Caleb Wheeler, Daniel C. Anderson, Prachi Sharma, Anapatricia Garcia, Elizabeth Strobert, Eric Elder, Ian Crocker, Timothy Crenshaw, M. Cecilia T. Penedo, Thea Ward, Mingqing Song, John Horan, Christian P. Larsen, Bruce R. Blazar, Leslie S. Kean
Blood (2010) 116 (24): 5403–5418.
Includes: Supplemental data
GvHD After Haploidentical Transplant: A Novel, MHC-Defined Rhesus Macaque Model Identifies CD28-Negative CD8+ T Cells as a Reservoir of Breakthrough T Cell Proliferation During Costimulation Blockade and Sirolimus-Based Immunosuppression
Swetha Srinivasan, Weston P. Miller IV, MD, Angela Panoskalltsis-Mortari, Sharon Sen, Kelly Hamby, Taylor Deane, Linda Stempora, Karnail Singh, Jonathan Beus, Bruce R. Blazar, MD, Leslie Kean, MD, PhD
Blood (2010) 116 (21): 2550.
Outcomes Following Allogeneic Hematopoietic Stem Cell Transplantation for Childhood Cerebral Variant Adrenoleukodystrophy: A Modern, Single-Institution Experience
Weston P Miller, IV, MD, Steven Rothman, MD, David Nascene, MD, Teresa Kivisto, RN, Todd Defor, Troy Lund, MD, PhD, Jakub Tolar, MD, PhD, Paul Orchard, MD
Blood (2010) 116 (21): 3543.