Exploring methods to deliver keratinocyte-targeting nano-carriers loaded with gene-editing tools to skin
Doctor Daniela Ortner-Tobider and Professor Matthias Schmüth
Project Duration: 3 year PhD; Project Start date: 1st November 2024
Total Project Cost: EURO 231,000
EI Cure Project Award: EURO 55,000; Project Collaborators: Medizinische Universität Innsbruck and PC Project
Delivery of keratinocyte-targeting nanocarriers loaded with gene editing tools to skin
Summary of the Project
To explore methods of delivery with gene-editing for topical delivery to the skin. The primary objective is to explore methods to transfect base-editing tools through the skin barrier using a lipid nanoparticle carrier. Proposed methods include direct topical application, use of lasers, and use of micro-needling to permeate the skin barrier. The primary target for gene-editing is the DNA of skin cells including skin stem cells located in the base layer of the epidermis. The secondary objective is to establish the efficacy of gene-editing needed to result in curative treatment of keratin-mutation related skin disorders.
Types of Keratin disorder included in the Project
Epidermolytic Ichthyosis Pathogenic Variants on KRT 1, 2, 9, 10
Pachyonychia Congenita Pathogenic Variants on KRT 6, 16, 17
Main outcomes anticipated from the Project
The anticipated outcome will be a method to enhance delivery of gene editing medication to intact human skin, and human skin affected with Epidermolytic Ichthyosis or Pachyonychia Congenita. This strategy can be applied to a variety of keratins, and therefore it will have broad implications for several keratin-mutation-related skin diseases.