Gene-Targeted Therapies in Inherited Bone Marrow Failure Syndromes
Inherited bone marrow failure syndromes (IBMFS) are genetically heterogeneous multiorgan disorders sharing defective hematopoiesis and cancer predisposition. Here, we examine emerging strategies to suppress nonsense mutations, with a focus on translational readthrough-inducing drugs (TRIDs), nonsense-mediated decay mod...