System Maintenance Notice

Please note that maintenance work is scheduled between 4 AM and 5 AM - CET. During this time, you may experience reduced performance. Thanks for your patience.

Publications

Journal Article Review Ppublish

PubMed: 40077969 DOI: 10.1016/j.ymthe.2025.03.017

Progress in skin gene therapy: From the inside and out.

Mol Ther 33(5):2065-2081

Authors: Mark J Osborn, Sidharth Panda, Theresa M Reineke, Jakub Tolar, Alexander Nyström

Affiliations

  • Medical School, Department of Pediatrics, Division of Blood and Marrow Transplant and Cellular and Gene Therapy, University of Minnesota, Minneapolis, MN 55455, USA.
  • Department of Chemistry, University of Minnesota, Minneapolis, MN 55455, USA.
  • Medical School, Department of Pediatrics, Division of Blood and Marrow Transplant and Cellular and Gene Therapy, University of Minnesota, Minneapolis, MN 55455, USA.
  • Department of Dermatology, Medical Faculty, Medical Center, University of Freiburg, 79106 Freiburg, Germany.

Abstract

The skin is the largest organ of the body and forms and serves as the barrier for preventing external material from accessing and damaging internal organs. As the outward interface to the environment, it is accessible for the application of therapeutic agents and cellular and gene therapy represent attractive and promising options to treat severe genetic conditions for which palliation has long been the main stay. However, because of its barrier function, transit across and to the subdermal compartment can be challenging. This commentary examines the current approaches of cell and gene therapies for genetic skin disorders. We write this from a local and systemic "outside and inside." perspective. Delivery from the outside encompasses topical, intradermal, and transdermal strategies for cell and vector delivery and ex vivo cell expansion and grafting. The inside approach details systemic delivery via infusion of cells or agents toward providing benefit to the skin. We use recessive dystrophic epidermolysis bullosa (RDEB) as a representative and paradigmatic disease to showcase these approaches as a means to highlight potential broader applicability to other conditions.

MeSH

Skin, Cells, Genes, Epidermolysis Bullosa Dystrophica, Gene Editing, Therapeutics, Stromal Cells, Genetic Therapy

Cited by