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Scientific Research Grant Recipients/Scholars

2017 Grant Recipients

Benjamin Samelson-Jones, MD, PhD

Instructor
Department of Pediatrics, Division of Hematology
Children’s Hospital of Philadelphia
Philadelphia, PA

Novel Therapeutics for Hemophilia B: A Rational Pursuit of Bioengineered Factor IX Variants with Enhanced Clotting Activity

A rational strategy, based on studies of Factor IX (FIX) R338L, is used to identify new hyperactive FIX variants. New variants will be biochemically characterized utilizing recombinant protein and the in vivo hemostatic efficacy and immunogenicity will be determined. This approach is anticipated to develop highly translatable FIX variants with enhanced activity and comparable immunogenicity for use in hemophilia B therapeutic

Juliana Xavier-Ferrucio, PhD

Postdoctoral Associate
Laboratory Medicine
Yale University
New Haven, CT

 

Megakaryocyte Erythroid Progenitor Fate Specification under Iron and Oxygen Deficiency

Little is known regarding how megakaryocyte-erythroid progenitor cells (MEP) make their fate decisions towards megakaryocyte (Mk) or erythroid (E) lineages. We propose to study iron deficiency anemia as a model of benign elevated platelet and reduced RBC, with a novel focus on the molecular mechanisms of the MEP biphenotypic fate decision. Potential clinical relevance includes improving strategies for in vitro maturation of RBC’s and platelets for transfusion purposes.