Cutaneous Gene Therapy

    January 2007 in “ Annals of Medicine ”
    Thomas G. Jensen
    TLDR Cutaneous gene therapy could become a viable treatment for skin and hair disorders with improved vector development and gene expression control.
    Cutaneous gene therapy research, conducted over more than 20 years, explored both direct and indirect gene transfer methods using viral and nonviral vectors to treat inherited skin diseases and potentially systemic disorders. Despite reaching clinical phases, challenges such as gene expression loss and immune reactions persisted. The study highlighted the potential of epidermal and hair follicle stem cells in skin regeneration and gene therapy, with telomerase influencing hair growth and cell proliferation. Clinical trials, such as those for junctional epidermolysis bullosa, showed potential for long-term expression and phenotypic correction. The research also explored hair follicle gene therapy, noting that adenoviral-mediated expression of Sonic hedgehog (Shh) could enhance hair growth, suggesting potential benefits for alopecia treatment. The document concluded that advancements in vector development and gene expression control could make cutaneous gene therapy a viable option for various diseases.
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