Gene Therapy: Recent Advances and Applications in Dermatology

In 1996, gene therapy was an emerging field with over 500 individuals treated and more than 200 clinical trials in progress, focusing on cancer immunotherapy, vaccines, and potential dermatological applications. Although no diseases had been cured, progress was evident in manipulating epidermal stem cells, creating skin-specific gene expression sequences, and developing systemic gene delivery systems. The document highlighted the use of gene therapy in cancer treatment through prodrug-converting enzymes and the bystander effect, as well as AIDS therapy strategies involving immune response elicitation and HIV-1 replication inhibition. It also discussed gene-based vaccines, the reintroduction of the p53 gene in cancer therapy, gene replacement therapy for ADA-SCID and CF, and the challenges of gene delivery systems. Over 90% of trials used viral vectors, with nonviral vectors being developed to overcome safety and efficiency issues. The document also explored gene therapy for genetic skin disorders like XP and ichthyosis, and the potential for addressing hair loss through hair follicle transfection. Despite technical challenges, optimism remained for gene therapy's application in dermatology.