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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.
NRASG12V oncogene facilitates self-renewal in a murine model of acute myelogenous leukemia.
Bedside to bench in juvenile myelomonocytic leukemia: insights into leukemogenesis from a rare pediatric leukemia.