Department of Chemical and Systems Biology, Stanford University School of Medicine, Stanford, CA 94305, USA.
ACS Chem Biol. 2010 Jan 15;5(1):15-34. doi: 10.1021/cb900249y.
Controlling cell fate is essential for embryonic development, tissue regeneration, and the prevention of human disease. With each cell in the human body sharing a common genome, achieving the appropriate spectrum of stem cells and their differentiated lineages requires the selective activation of developmental signaling pathways, the expression of specific target genes, and the maintenance of these cellular states through epigenetic mechanisms. Small molecules that target these regulatory processes are therefore valuable tools for probing and manipulating the molecular mechanisms by which stem cells self-renew, differentiate, and arise from somatic cell reprogramming. Pharmacological modulators of cell fate could also help remediate human diseases caused by dysregulated cell proliferation or differentiation, heralding a new era in molecular therapeutics.
控制细胞命运对于胚胎发育、组织再生和预防人类疾病至关重要。由于人体中的每个细胞都共享一个共同的基因组,因此要实现适当的干细胞谱系及其分化谱系,需要选择性地激活发育信号通路、表达特定的靶基因,并通过表观遗传机制维持这些细胞状态。因此,靶向这些调节过程的小分子是研究和操纵干细胞自我更新、分化以及体细胞重编程的分子机制的有价值的工具。细胞命运的药理学调节剂也可能有助于治疗由细胞增殖或分化失调引起的人类疾病,这标志着分子治疗的新时代的到来。