Forschung & Innovation
Publikationen
Emerging DNA & RNA editing strategies for the treatment of epidermolysis bullosa
PMU Autor*innen
Ulrich Koller, Johann W Bauer
Alle Autor*innen
Ulrich Koller, Johann W Bauer
Fachzeitschrift
JOURNAL OF DERMATOLOGICAL TREATMENT
Kurzfassung
Background: Epidermolysis bullosa (EB) is a clinically-heterogeneous genodermatosis with severe manifestations in the skin and other organs. The significant burden this condition places on patients justifies the development of gene therapeutic strategies targeting the genetic cause of the disease. Methods: Emerging RNA and DNA editing tools have shown remarkable advances in efficiency and safety. Applicable both in ex vivo- and in vivo settings, these gene therapeutics based on gene replacement or editing are either at the pre-clinical or clinical stage. Results: The recent landmark FDA approvals for gene editing based on CRISPR/Cas9, along with the first FDA-approved redosable in vivo gene replacement therapy for EB, will invigorate ongoing research efforts, increasing the likelihood of achieving local cure via CRISPR-based technologies in the near future. Conclusions: This review discusses the status quo of current gene therapeutics that act at the level of RNA or DNA, all with the common aim of improving the quality of life for EB patients.
Keywords
Humans, Epidermolysis Bullosa/therapy, Gene editing, Quality of life, Genetic Therapy, CRISPR-Cas Systems, RNA Editing