Pluripotent stem cell (PSC) (PSC)

Pluripotent stem cells (PSCs) are a unique class of undifferentiated cells characterized by their capacity for indefinite self-renewal and their potential to differentiate into any cell type derived from the three primary germ layers: ectoderm, endoderm, and mesoderm [NIH Stem Cell Information, 2016]. This category primarily encompasses embryonic stem cells (ESCs), obtained from the inner cell mass of a blastocyst, and induced pluripotent stem cells (iPSCs), which are adult somatic cells genetically reprogrammed to a pluripotent state using specific transcription factors [Takahashi & Yamanaka, Cell, 2006]. While PSCs are not traditional molecular targets like receptors or enzymes, they represent a revolutionary platform in regenerative medicine and drug discovery for treating conditions such as Parkinson's disease and heart failure [Lee et al., Nature Reviews Drug Discovery, 2013]. Small molecules are frequently employed to manipulate the signaling networks—such as the Wnt and TGF-beta pathways—that govern their fate and maintain their pluripotency [Wu & Belmonte, Nature Reviews Genetics, 2015]. However, the clinical application of PSC-derived therapies is currently limited by significant safety hurdles, most notably the risk of tumorigenesis and the potential for teratoma formation if undifferentiated cells remain in the transplant [Bulic-Jakus et al., World Journal of Stem Cells, 2016].

Other names

Pluripotential cellsPluripotent cellsEmbryonic stem cellsInduced pluripotent stem cellsPSCsHuman pluripotent stem cells

Drugs and small molecules interact with pluripotent cells by modulating key intracellular signaling pathways, such as Wnt/beta-catenin, TGF-beta/Smad, and Rho-associated protein kinase (ROCK), or by acting as epigenetic modifiers to maintain the undifferentiated state or direct lineage-specific differentiation into specialized cell types.