Efficient In Vivo Targeting of Epidermal Stem Cells by Early Gestational Intraamniotic Injection of Lentiviral Vector Driven by the Keratin 5 Promoter

    October 2007 in “ Molecular Therapy ”
    Masayuki Endo, Philip W. Zoltick, William H. Peranteau, Antoneta Radu, Nidal Muvarak, Mayumi Ito, Zaixin Yang, George Cotsarelis, Alan W. Flake
    TLDR Injecting lentiviral vectors into early gestation mice effectively targets skin stem cells for potential gene therapy.
    Researchers investigated a method for gene transfer to skin stem cells by injecting lentiviral vectors into the amniotic space of mice during early gestation. The study involved 470 fetuses and found that using the keratin 5 (K5) promoter led to sustained expression of green fluorescent protein (GFP) in basal epidermal stem cells and hair follicle bulge cells, with improved efficiency compared to the cytomegalovirus promoter. The transduced stem cells participated in wound healing, indicating successful targeting. The study suggested potential applications for gene therapy in skin disorders, though it raised concerns about insertional mutagenesis and developmental effects due to the early timing of the intervention.
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