2017 Sickle Cell Disease/Advancing Cures Awardees
Approximately $6 Million
In 2017, seven projects were awarded a combined total of $6 million in research funding to address the underlying cause of sickle cell disease, attacking it at its core by restoring hemoglobin function through various approaches.
2017 SCD/AC Awardees
Allistair A. Abraham, M.D., and Robert S. Nickel, M.D.
Children’s National Health System and the George Washington University School of Medicine and Health Science
Project name: Minimizing Toxicity in HLA-identical Sibling Transplantation for Children with Sickle Cell Disease
Eric E. Bouhassira, Ph.D.
Albert Einstein College of Medicine
Project name: Characterization of the Stem and Progenitor Cell Compartment in Sickle Cell Disease and Optimization of Gene Transfer at AAVS1 Site in CD49f+ LT-HSCs
Donald B. Kohn, M.D.
University of California, Los Angeles
Project name: Optimizing Gene Editing for Sickle Cell Disease
Patrick T. McGann, M.D., M.S.
Cincinnati Children’s Hospital Medical Center
Project name: Maximizing Fetal Hemoglobin Responses to Hydroxyurea Using Precision Medicine
Stuart H. Orkin, M.D., and Daniel E. Bauer, M.D., Ph.D.
Boston Children’s Hospital
Project name: Small Molecule Targeted Reactivation of HbF Expression for Sickle Cell Disease
Mitchell J. Weiss, M.D., Ph.D., and Shengdar Q. Tsai, Ph.D.
St. Jude Children’s Research Hospital
Project name: Genome Editing of Bone Marrow and Plerixafor-mobilized CD34+ Cells to Raise Fetal Hemoglobin Levels in Sickle Cell Disease
Patrick M. Woster, Ph.D.
Medical University of South Carolina
Project name: Epigenetic Modulators for the Treatment of Sickle Cell Disease