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

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
Departments of Pediatrics and Cancer Biology, Cleveland Clinic, Cleveland, OH 44195, USA; Case Comprehensive Cancer Center, Cleveland, OH 44106
Haematologica Early view Jun 15, 2023 https://doi.org/10.3324/haematol.2023.283462