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.

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Doctor Leonie Frommherz and Professor Cristina Has