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Musical chairs: in utero HCT via mobilization.
A trial of unrelated donor marrow transplantation for children with severe sickle cell disease.
Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency.
Pediatric MDS: GATA screen the germline.
A booster shot to cure hemoglobinopathies.
MAPK signaling cascades mediate distinct glucocorticoid resistance mechanisms in pediatric leukemia.
The LSD1 inhibitor RN-1 induces fetal hemoglobin synthesis and reduces disease pathology in sickle cell mice.
Efficacy of JAK/STAT pathway inhibition in murine xenograft models of early T-cell precursor (ETP) acute lymphoblastic leukemia.
Subclonal mutations in SETBP1 confer a poor prognosis in juvenile myelomonocytic leukemia.
Preclinical efficacy of MEK inhibition in Nras-mutant AML.