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Current supported projects

 

Project title:
“Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer to Utilize Platelet-Derived Factor VIII for Hemophilia A”
Principal Investigator:
David A. Wilcox, PhD, Medical College of Wisconsin
 
Project title:  “Lentiviral Vector for the Treatment of Fanconi Anemia”
Principal Investigator: W. Scott Goebel, MD, PhD, Indiana University School of Medicine
Co-Investigator:  Grzegorz Nalepa, MD, PhD, Indiana University School of Medicine
 
Project title:  “Pulmonary Transplantation of Autologous CD34+ Cell Derived Macrophages for Hereditary Pul-monary Alveolar Proteinosis”
Principal Investigator: Bruce Trapnell, MD, Cincinnati Children’s Hospital Medical Center
Co-Investigator:  Thomas Moritz, MD, Hanover Medical School
 
Project title:  Therapeutic BCL11A enhancer editing for the β-hemoglobin disorders
Principal Investigator: Daniel E. Bauer, MD PhD, Boston Children’s Hospital, Harvard Medical School
 
Project title:  Clinical development of CAR T cell therapy targeting CD70 in glioblastoma
Principal Investigator: Jianping Huang, MD, PhD, University of Florida
Co-Investigator:  Duane A. Mitchell, MD, PhD, University of Florida
 
Project title:  Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Sickle Cell Disease
Principal Investigator: Lakshmanan Krishnamurti, MD, Emory University