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Context-Dependent Strategies for Enhanced Genome Editing of Genodermatoses

EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, 5020 Salzburg, Austria
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These authors contributed equally to the work.
Cells 2020, 9(1), 112; https://doi.org/10.3390/cells9010112
Received: 3 December 2019 / Revised: 27 December 2019 / Accepted: 31 December 2019 / Published: 2 January 2020
(This article belongs to the Special Issue Genome Editing Systems, Methods, Techniques and Their Application)
The skin provides direct protection to the human body from assault by the harsh external environment. The crucial function of this organ is significantly disrupted in genodermatoses patients. Genodermatoses comprise a heterogeneous group of largely monogenetic skin disorders, typically involving mutations in genes encoding structural proteins. Therapeutic options for this debilitating group of diseases, including epidermolysis bullosa, primarily consist of wound management. Genome editing approaches co-opt double-strand break repair pathways to introduce desired sequence alterations at specific loci. Rapid advances in genome editing technologies have the potential to propel novel genetic therapies into the clinic. However, the associated phenotypes of many mutations may be treated via several genome editing strategies. Therefore, for potential clinical applications, implementation of efficient approaches based upon mutation, gene and disease context is necessary. Here, we describe current genome editing approaches for the treatment of genodermatoses, along with a discussion of the optimal strategy for each genetic context, in order to achieve enhanced genome editing approaches. View Full-Text
Keywords: genome editing; genodermatoses; designer nucleases; DNA repair pathways; epidermolysis bullosa; keratinopathies genome editing; genodermatoses; designer nucleases; DNA repair pathways; epidermolysis bullosa; keratinopathies
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March, O.P.; Kocher, T.; Koller, U. Context-Dependent Strategies for Enhanced Genome Editing of Genodermatoses. Cells 2020, 9, 112.

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