Gene therapy for congenital marrow failure syndromes – no longer grasping at straws?

• Richard A. Voit,
• Seth J. Corey

Affiliations

Richard A. Voit

Division of Hematology/Oncology, Boston Children’s Hospital, Harvard Medical School, Boston, MA 02115, USA; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02115

Seth J. Corey

Departments of Pediatrics and Cancer Biology, Cleveland Clinic, Cleveland, OH 44195, USA; Case Comprehensive Cancer Center, Cleveland, OH 44106