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PC Project Awards Gene Editing Delivery Grant

August 15, 2024

Awarded a total amount of €55,500 in installments of €18,500 per year for three (3) years.

The funding will be awarded in collaboration with the EI Cure Project paying an equal amount of funds per year for the duration of the grant period of three (3) years.

“Delivery of keratinocyte-targeting nanocarriers loaded with gene editing tools to skin”

Summary: 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 nanoparticlecarrier. 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 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.

Main outcomes anticipated from research: 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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PACHYONYCHIA CONGENITA PROJECT

P.O. Box 17850
Holladay, UT 84117

info@pachyonychia.org

801-987-8758

EIN 68-0567493

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