In this manuscript, the authors show deregulated protein synthesis as a novel, noncanonical defect in Fanconi Anemia. The observed deficits reflect the impact of proteostasis during fetal hematopoietic stem cell expansion and define the origins of hematopoietic failure in this…
Researchers found exposing cells to a small molecule drug improves cell fitness during hematopoietic stem cell transplants, which could improve ex vivo gene therapy.
CHOP researchers find exposing cells to a small molecule drug improves cell fitness during hematopoietic stem cell transplants, which could improve ex vivo gene therapy.