CHOP Presenters at ASH 2022
SATURDAY, 12/10
Stefano Rivella, PhD, presenting author and senior author
- Saturday, December 10, 2022: 9:30 AM
- 121 Lnp-Targeting Hematopoietic Stem Cells and Lentiviral Gene Transfer to Generate and Rescue a Novel Mouse Model of Lethal Alpha-Thalassemia
- https://ash.confex.com/ash/2022/webprogram/Paper166621.html
Fatemeh Alikarami, presenting author
Kathrin Bernt, MD, senior author
- Saturday, December 10, 2022: 9:45 AM
- 44 The Role of GATA2 in Drug Resistance in AML
- https://ash.confex.com/ash/2022/webprogram/Paper166666.html
Laura Breda, PhD, presenting author
Stefano Rivella, PhD, co-senior author
- Saturday, December 10, 2022: 10:00 AM
- 123 In Vivo Modification of Hematopoietic Stem Cells By Targeted Lipid Nanoparticles Encapsulating mRNA
- https://ash.confex.com/ash/2022/webprogram/Paper165089.html
Stephan Grupp, MD, PhD, senior author
Haydar Frangoul, MD, presenting author from Sarah Cannon Center Research Institute
- Saturday, December 10, 2022: 10:45 AM
- 12 Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Severe Sickle Cell Disease
- https://ash.confex.com/ash/2022/webprogram/Paper162353.html
Yang Ding, MD, presenting author
Kai Tan, PhD, senior author
- Saturday, December 10, 2022: 12:00 PM
- 145 Transcriptional Rewiring and Therapeutic Resistance in Philadelphia Chromosome-like Acute Lymphoblastic Leukemia
- https://ash.confex.com/ash/2022/webprogram/Paper169341.html
Tim Olson, MD, PhD, senior author
Joseph Oved, MD, presenting author from Memorial Sloan Kettering Cancer Center
- Saturday, December 10, 2022: 4:15 PM
- 290 Pediatric-Specific Patterns of Clonal Evolution Arising from Acquired Aplastic Anemia
- https://ash.confex.com/ash/2022/webprogram/Paper170538.html
Ben Samelson-Jones, MD, PhD, senior author
Lucas Van Gorder, BS, presenting author from Perelman School of Medicine
- Saturday, December 10, 2022, 5:30 PM-7:30 PM
- 2145 Vector Analysis of Multicentric Lymphoma in a Severe Hemophilia Α Dog after AAV Gene Therapy
- https://ash.confex.com/ash/2022/webprogram/Paper164440.html
Catherine Falkenstein, BS, presenting author
Sarah Tasian, MD, senior author
- Saturday, December 10, 2022, 5:30 PM-7:30 PM
- 1516 Preclinical In Vivo Activity of the Menin Inhibitor Ziftomenib (KO-539) in Pediatric KMT2A-Rearranged Acute Lymphoblastic Leukemia
- https://ash.confex.com/ash/2022/webprogram/Paper157225.html
Haley Newman, MD, presenting author
David Teachey, MD, senior author
- Saturday, December 10, 2022, 5:30 PM-7:30 PM
- 1501 The Influence of Genetic Ancestry on Disease Biology in Pediatric T-Cell Acute Lymphoblastic Leukemia
- https://ash.confex.com/ash/2022/webprogram/Paper159159.html
Kristina Wagner, MD, presenting author
Hilary Whitworth, MD, senior author
- Saturday, December 10, 2022, 5:30 PM-7:30 PM
- 1189 Clinical Characteristics and Acute Management of Pediatric Pulmonary Embolism
- https://ash.confex.com/ash/2022/webprogram/Paper164830.html
Lusha Cao, PhD, presenting author
Richard Aplenc, MD, PhD, MSCE, senior author
- Saturday, December 10, 2022, 5:30 PM-7:30 PM
- 1486 Proteotranscriptomic Strategy to Discover Acute Myeloid Leukemia Immunotherapy Target
- https://ash.confex.com/ash/2022/webprogram/Paper167378.html
Manuel Torres-Diz, PhD, presenting author
Andre Thomas-Tikonehko, PhD, co-senior author
- Saturday, December 10, 2022, 5:30 PM-7:30 PM
- 1416 Non-Canonical Exon Usage in Pediatric Leukemia: Implications for Chemoresistance
- https://ash.confex.com/ash/2022/webprogram/Paper167006.html
Omar Banda, PhD, presenting author
Peter Kurre, MD, senior author
- Saturday, December 10, 2022, 5:30 PM-7:30 PM
- 2147 Direct Delivery of mRNA-Loaded Lipid Nanoparticles to the Hematopoietic Stem Cell Niche
- https://ash.confex.com/ash/2022/webprogram/Paper159085.html
Scott Peslak, MD, PhD, presenting author
Gerd Blobel, MD, PhD, senior author
- Saturday, December 10, 2022, 5:30 PM-7:30 PM
- 1041 Regulation of Fetal Hemoglobin Production in Adult Erythroid Cells By Protein Phosphatase 6C (PPP6C)
- https://ash.confex.com/ash/2022/webprogram/Paper165286.html
Stephanie Hurwitz, MD, PhD, presenting author
Peter Kurre, MD, senior author
- Saturday, December 10, 2022, 5:30 PM-7:30 PM
- 1210 Blockade of Ceramide-Dependent Extracellular Vesicle Secretion Improves Hematopoietic Stem Cell Transplantation
- https://ash.confex.com/ash/2022/webprogram/Paper159793.html
SUNDAY, 12/11
Anna Sternberg, PhD, presentng author
Lindsey George, MD, senior author
- Sunday, December 11, 2022: 9:45 AM
- 398 Inhibiting Activated Protein C Cleavage of Factor VIII for Hemophilia a Gene Therapy
- https://ash.confex.com/ash/2022/webprogram/Paper170646.html
Kelly Getz, PhD, MPH, presenting author
Richard Aplenc, MD, PhD, MSCE, senior author
- Sunday, December 11, 2022: 9:45 AM
- 422 Distinct Latent Trajectory Phenotypes of Left Ventricular Function Associated with Differential Survival Among Children Treated for Acute Myeloid Leukemia: A Report from the Children’s Oncology Group
- https://ash.confex.com/ash/2022/webprogram/Paper170218.html
Changya Chen, PhD, presenter
Kai Tan, PhD, co-senior author
David Teachey, MD, co-senior author
- Sunday, December 11, 2022: 12:30 PM
- 531 Single-Cell Pan-Cancer Analysis Reveals Treatment Resistance Stem/Progenitor-like Subpopulation in the High-Risk Pediatric Leukemia
- https://ash.confex.com/ash/2022/webprogram/Paper167057.html
Ahn T. P. Ngo, PhD, presenting author
Kandace Gollomp, MD, senior author
- Sunday, December 11, 2022, 6:00 PM-8:00 PM
- 2434 Neutrophil Extracellular Traps (NETs) Thrombogenicity and Endothelial Cell Toxicity Are Ameliorated By Platelet Factor 4 (PF4): Implications to the Treatment of Sepsis
- https://ash.confex.com/ash/2022/webprogram/Paper169709.html
Alexis Thompson, MD, MPH, senior author
Mark Walters, MD, presenting author from UCSF
- Sunday, December 11, 2022, 6:00 PM-8:00 PM
- 2348 Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials
- https://ash.confex.com/ash/2022/webprogram/Paper162400.html
Amaliris Guerra, PhD, presenting author
Peter Kurre, MD, senior author
- Sunday, December 11, 2022, 6:00 PM-8:00 PM
- 2323 TNFα and Anti-IFNγ Rescues Chronic Anemia of Inflammation Phenotype in Tnfαko Mice Treated with Heat-Killed Brucella Abortus
- https://ash.confex.com/ash/2022/webprogram/Paper166643.html
Apeksha Dave, MD, presenting author
William Peranteau, MD, senior author
- Sunday, December 11, 2022, 6:00 PM-8:00 PM
- 3272 Sustained Engraftment in Fanconi Anemia (FA) Mice Using in Utero Hematopoietic Cell Transplantation (IUHCT)
- https://ash.confex.com/ash/2022/webprogram/Paper170456.html
Bhavya Doshi, MD, presenting author
Ben Samelson-Jones, MD, PhD, senior author
- Sunday, December 11, 2022, 6:00 PM-8:00 PM
- 3463 Long-Term Real-World Safety and Efficacy of Liver-Directed AAV Gene Therapy for Severe Hemophilia with and without Inhibitors in Privately Owned Dogs
- https://ash.confex.com/ash/2022/webprogram/Paper164470.html
Ding-Wen Chen, PhD, presenting author
Peter Kurre, senior author
- Sunday, December 11, 2022, 6:00 PM-8:00 PM
- 2557 Developing a Humanized Murine Model for Co-Transplantation of Acute Myeloid Leukemia
- https://ash.confex.com/ash/2022/webprogram/Paper158615.html
Kunhua Qin, presenting author
Gerd Blobel, MD, PhD, senior author
- Sunday, December 11, 2022, 6:00 PM-8:00 PM
- 2361 Mechanisms of Polycomb Group Protein-Mediated Fetal Hemoglobin Silencing
- https://ash.confex.com/ash/2022/webprogram/Paper165560.html
Susan E. McClory, MD, PhD, presenting and senior author
- Sunday, December 11, 2022, 6:00 PM-8:00 PM
- 2416 Trib1 Inhibition Promotes the Klr Effector State and Augments Checkpoint Blockade to Enhance T Cell Function during T Cell Exhaustion
- https://ash.confex.com/ash/2022/webprogram/Paper165346.html
Ben Samelson-Jones, MD, PhD, presenting author and senior author
- Sunday, December 11, 2022, 6:00 PM-8:00 PM
- 2456 Factor VIII Mimetic Rescue of Hemophilia B Causing Factor IX Variants
- https://ash.confex.com/ash/2022/webprogram/Paper164689.html
Lisa Niswander, MD, PhD, presenting author
Sarah Tasian, MD, senior author
- Sunday, December 11, 2022, 6:00 PM-8:00 PM
- 2747 Clinical Experience with Azacitidine and Venetoclax +/- Gemtuzumab in Pediatric Patients with Relapsed or Refractory Acute Leukemias: A Report from the Children’s Hospital of Philadelphia
- https://ash.confex.com/ash/2022/webprogram/Paper166232.html
Narasaiah Kovuru, PhD, presenting author
Peter Kurre, MD, senior author
- Sunday, December 11, 2022, 6:00 PM-8:00 PM
- 2579 Excess Ribosome Biogenesis and Dysregulated Proteostasis Restrain Expansion of the Fetal HSC Pool in Fanconi Anemia
- https://ash.confex.com/ash/2022/webprogram/Paper159761.html
Sarah Tasian, MD, presenting author
Mignon L. Loh, MD, senior author
- Sunday, December 11, 2022, 6:00 PM-8:00 PM
- 2725 A Phase 2 Study of Ruxolitinib with Chemotherapy in Children with Philadelphia Chromosome-like Acute Lymphoblastic Leukemia (AALL1521/INCB18424-269): Biologic Characteristics and Minimal Residual Disease Response of Patients with Non-CRLF2-Rearranged JAK Pathway Alterations
- https://ash.confex.com/ash/2022/webprogram/Paper164699.html
Ying Ting Sit, presenting author
Stella Chou, MD, senior author
- Sunday, December 11, 2022, 6:00 PM-8:00 PM
- 2592 Intersection of GATA1 and DYRK1A in Trisomy 21 Leukemogenesis
- https://ash.confex.com/ash/2022/webprogram/Paper169399.html
MONDAY, 12/12
Kai Tan, PhD, co-senior author
David Teachey, MD, co-senior author
Jason Xu, PhD, presenting author from Perelman School of Medicine
- Monday, December 12, 2022: 11:00 AM
- 717 Progenitor Sub-Populations in Treatment Resistant T-ALL
- https://ash.confex.com/ash/2022/webprogram/Paper166521.html
David Teachey, MD, senior author
Petri Polonen, PhD, presenting author from St. Jude’s
- Monday, December 12, 2022: 11:15 AM
- 718 Comprehensive Genome Characterization of Childhood T-ALL Links Oncogene Activation Mechanism and Subtypes to Prognosis
- https://ash.confex.com/ash/2022/webprogram/Paper167157.html
Denise Sabatino, PhD, presenting author and senior author
- Monday, December 12, 2022: 11:15 AM
- 784 Second Generation Adeno-Associated Viral Vector Using a Novel Factor VIII Variant with Improved Secretion Achieves Higher Factor VIII Expression in Non-Human Primates
- https://ash.confex.com/ash/2022/webprogram/Paper168340.html
Eugene Khandros, MD, PhD, presenting author
Gerd Blobel, MD, PhD, senior author
- Monday, December 12, 2022: 2:45 PM
- 799 Let-7 Inhibits HIC2 to Control BCL11A Transcription and Hemoglobin Switching\
- https://ash.confex.com/ash/2022/webprogram/Paper157978.html
Eugene Khandros, MD, PhD, presenting author and senior author
- Monday, December 12, 2022: 3:30 PM
- 802 Examining the Role of Individual Baf Chromatin Remodeling Complex Components in Globin Gene Regulation
- https://ash.confex.com/ash/2022/webprogram/Paper159347.html
Kemar Prussien, PhD, presenting author
Lisa Schwartz, PhD, senior author
- Monday, December 12, 2022: 3:30 PM
- 898 Barriers to Transition from Pediatric to Adult Care in Adolescents and Young Adults with Sickle Cell Disease: A Mixed-Method Study on the Impact of Systems of Inequity
- https://ash.confex.com/ash/2022/webprogram/Paper159007.html
Sergey Zaytsev, PhD, presenting author
Victoria Stephanova, PhD, senior author from Perelman School of Medicine
- Monday, December 12, 2022: 3:30 PM
- 814 Fusion of uPA to Anti-aIIbb3 Scfv Facilitates Its Uptake By in Vitro-Generated CD34+ Megakaryocytes (MKs) to Promote Targeted Fibrinolysis
- https://ash.confex.com/ash/2022/webprogram/Paper168072.html
Amaliris Guerra, PhD, presenting author
Stefano Rivella, PhD, senior author
- Monday, December 12, 2022: 3:45 PM
- 803 Combination of a TGF-β Ligand Trap (RAP-GRL) and TMPRSS6-ASO Is Superior for Correcting β-Thalassemia
- https://ash.confex.com/ash/2022/webprogram/Paper166801.html
Sean Quinn, PhD, presenting author
Rodney Camire, PhD, senior author from Perelman School of Medicine
- Monday, December 12, 2022, 4:30 PM-6:05 PM
- 397 An Antibody Targeting Human FV Promotes Thrombin Generation and Reduces Bleeding in a Hemophilic Mouse Model
- https://ash.confex.com/ash/2022/webprogram/Paper157997.html
Regina Myers, MD, presenting author
Stephan Grupp; MD, PhD, senior author
- Monday, December 12, 2022: 5:30 PM
- 983 CD22-Targeted CAR-Modified T-Cells Safely Induce Remissions in Children and Young Adults with Relapsed, CD19-Negative B-ALL after Treatment with CD19-Targeted CAR T-Cells
- https://ash.confex.com/ash/2022/webprogram/Paper168139.html
Ben Samelson-Jones, MD, PhD, presenting author
Gallia Levy, MD, PhD, senior author from Spark Therapeutics
- Monday, December 12, 2022, 6:00 PM-8:00 PM
- 4783 Rapid Clearance of Vector Following AAV-Mediated FVIII Gene Transfer in the Phase I/II Trial of SPK-8011 in People with Hemophilia A
- https://ash.confex.com/ash/2022/webprogram/Paper159027.html
Alexis Thompson, MD, MPH, senior author
Franco Locatelli, MD, presenting author from IRCCS Ospedale Pediatrico Bambino Gesu
- Monday, December 12, 2022, 6:00 PM-8:00 PM
- 3665 Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia
- https://ash.confex.com/ash/2022/webprogram/Paper163178.html
Vinodh Pillai, MD, senior author
Joshua Anil, BSc, presenting author from Perelman School of Medicine
- Monday, December 12, 2022, 6:00 PM-8:00 PM
- 4144 Bone Marrow Fibrosis Is Associated with Non-Response to CD19 CAR-T Therapy
- https://ash.confex.com/ash/2022/webprogram/Paper156721.html
Amaliris Guerra, PhD, presenting author
Stefano Rivella, PhD, senior author
- Monday, December 12, 2022, 6:00 PM-8:00 PM
- 3655 Differential Effects of Monoferric Forms of Transferrin on Mouse Models of Anemia of Inflammation and Congenital Anemia
- https://ash.confex.com/ash/2022/webprogram/Paper166391.html
Caroline Diorio, MD, FRCPC, FAAP, presenting author
David Teachey, MD, senior author
- Monday, December 12, 2022, 6:00 PM-8:00 PM
- 4621 Targeting CD38 in T-ALL Upregulates the Targetable Polyamine Metabolism Pathway
- https://ash.confex.com/ash/2022/webprogram/Paper167893.html
Haley Newman, MD, presenting author
Kelly Getz, PhD, MPH senior author from Perelman School of Medicine
- Monday, December 12, 2022, 6:00 PM-8:00 PM
- 4942 Influence of Household Income on Health-Related Quality of Life in Children with Acute Myeloid Leukemia
- https://ash.confex.com/ash/2022/webprogram/Paper160336.html
Jenny Ruiz, MD, presenting author
Alix Seif, MD, MPH, senior author
- Monday, December 12, 2022, 6:00 PM-8:00 PM
- 4955 Septic Shock and Septic Shock Mortality in Relation to Social Disorganization Index Among Children Being Treated for De Novo Acute Myeloid Leukemia on COG Clinical Trials
- https://ash.confex.com/ash/2022/webprogram/Paper163573.html
John Riley, MD, presenting author
William Pereanteau, MD, senior author
- Monday, December 12, 2022, 6:00 PM-8:00 PM
- 4589 Late-Gestation Hematopoietic Cell Transplantation with Transient T Cell Depletion Significantly Improves Disease Phenotype in the Townes Mouse Model of Sickle Cell Disease
- https://ash.confex.com/ash/2022/webprogram/Paper166599.html
Paul Gallo, MD, PhD, presenting author
Michele Lambert, MD, senior author
- Monday, December 12, 2022, 6:00 PM-8:00 PM
- 3733 Analysis of Clinical Cytokine Panel Profiles As Diagnostic and Prognostic Biomarkers in Pediatric Hyperinflammatory Conditions
- https://ash.confex.com/ash/2022/webprogram/Paper170411.html
Suying Liu, presenting author
Peter Kurre, MD, senior author
- Monday, December 12, 2022, 6:00 PM-8:00 PM
- 3896 Clonal Diversity As an Alternative Measure for Hematopoietic Reserve in Bone Marrow Failure
- https://ash.confex.com/ash/2022/webprogram/Paper159730.html