Gene Delivery to the Skin – How Far Have We Come?. Issue 5 (May 2021)

• Record Type: Journal Article
• Authors: Ain, Qurrat Ul; Campos, Estefania V.R.; Huynh, Ariel; Witzigmann, Dominik; Hedtrich, Sarah
• Is Part Of: Trends in biotechnology. Volume 39:Issue 5(2021)
• Page Start: 474
• Page End: 487
• Publication Date: 2021-05
• Subjects: gene therapy -- skin -- gene editing -- topical gene delivery -- genodermatoses -- CRISPR -- RNA therapy; Biotechnology -- Periodicals; Biochemical engineering -- Periodicals; Genetic engineering -- Periodicals; Industrial microbiology -- Periodicals; 660.605
• DOI: 10.1016/j.tibtech.2020.07.012
• Languages: English
• ISSNs: 0167-7799

Abstract

Abstract : Gene therapies are powerful tools to prevent, treat, and cure human diseases. The application of gene therapies for skin diseases received little attention so far, despite the easy accessibility of skin and the urgent medical need. A major obstacle is the unique barrier properties of human skin, which significantly limits the absorption of biomacromolecules, and thus hampers the efficient delivery of nucleic acid payloads. In this review, we discuss current approaches, successes, and failures of cutaneous gene therapy and provide guidance toward the development of next-generation concepts. We specifically allude to the delivery strategies as the major obstacle that prevents the full potential of gene therapies – not only for skin disorders but also for almost any other human disease. Highlights: Gene therapies are powerful tools for the treatment of inflammatory, genetic, and cancer-related skin diseases. The skin barrier function and the low number of cells that get transfected are the main hurdles for cutaneous gene therapy and contribute to the fact that gene therapies for skin diseases are an underexplored area. Gene editing provides an approach to cure rare and severe genodermatoses-like epidermolysis bullosa. First studies demonstrate the potential and invaluable impact these treatments may have even if only a small percentage of the gene function can be restored. Recent advancements demonstrate the power of non-viral delivery systems for the delivery of gene therapeutics to the skin. They may prove superior to viral vectors, the current gold standard, because their use is not limited by packaging size, serious safety concerns, or manufacturing issues.