Several bone marrow failure syndromes are caused by mutations in genes functioning in ribosome biogenesis. They include Dyskeratosis congenita (DC), Shwachman-Diamond syndrome (SDS), and Diamond-Blackfan Anemia (DBA). A DBA, DC, and SDS syndrome, besides defective hematopoiesis, shares the presence of congenital anomalies and predisposition to cancer. Moreover, deficiency of RPS14 was found in myelodysplastic patients with 5q- syndrome further connecting ribosomal deficiency and bone marrow failure. We propose to explore aspects of basic and clinical translational research on diseases based on defective ribosomal synthesis, including basic biology of ribosome synthesis, alternative models of disease, controversies in current treatment, and future directions.