Small Molecules and Small Molecule Drugs in Regenerative Medicine

    November 2013 in “ Drug Discovery Today ”
    Biao Lu, Anthony Atala
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    TLDR Small molecule drugs show promise for advancing regenerative medicine but still face development challenges.
    The document from 2013 and 2014 reviews the role of small molecules in regenerative medicine, focusing on their applications in cell therapy, tissue engineering, and enhancing stem cell function. It discusses the use of small molecules in differentiating and reprogramming human pluripotent stem cells (hPSCs), including human embryonic stem cells (hESCs) and induced pluripotent stem cells (hiPSCs), into specific cell types and improving the safety and efficiency of these processes. The potential of small molecules to activate adult stem cells for tissue regeneration and the risks of tumorigenesis are also addressed. The document highlights the strategies for in vivo trans-differentiation and the use of high-throughput screening to identify useful small molecules. It concludes that while small molecule drugs are still in development, with some in clinical trials for conditions like androgenic alopecia, their role in regenerative medicine is expected to grow despite existing challenges.
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