Sickle Cell Intervention

摘要

The damaging effects of sickle cell disease, caused by a point mutation in the gene that encodes a form of hemoglobin expressed in adults, can be lessened by reintroduction of the form of hemoglobin expressed during fetal development. Unfortunately, the fetal globin gene is usually turned off as development progresses. Using mice carrying both endogenous murine globin genes and transgenic human globin genes, Xu et al. (p. 993, published online 13 October) studied the re-pressor BCLlla, which silences fetal globin gene expression. Without BCLlla, expression of fetal qlobin persisted beyond its normal phase of development.