Heller P, DeSimone J
Am J Hematol. 1984;17(4):439-47. doi: 10.1002/ajh.2830170416.
The evidence that 5-azacytidine stimulates the production of Hb F and F cells in baboon and man is reviewed. The mechanism of this effect is not entirely clear, but 5-azacytidine produces hypomethylation of the gamma gene at certain sites, and gene expression and DNA hypomethylation are related phenomena in many other systems. Other mechanisms have been postulated by other investigators. The therapeutic significance of increased Hb F levels in homozygous beta thalassemia and sickle cell anemia is exemplified. The potential risk of carcinogenicity has delayed more extensive clinical trials.
本文综述了5-氮杂胞苷刺激狒狒和人类产生胎儿血红蛋白（Hb F）和F细胞的证据。这种作用的机制尚不完全清楚，但5-氮杂胞苷会使γ基因在某些位点发生低甲基化，并且在许多其他系统中，基因表达和DNA低甲基化是相关现象。其他研究者也提出了其他机制。文中举例说明了纯合β地中海贫血和镰状细胞贫血中胎儿血红蛋白水平升高的治疗意义。致癌性的潜在风险延缓了更广泛的临床试验。
