Abstract
The discovery of the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system has revolutionized gene editing research. Through the repurposing of programmable RNA-guided CRISPR-associated (Cas) nucleases, CRISPR-based genome editing systems allow for the precise modification of specific sites in the human genome and inspire novel approaches for the study and treatment of inherited and acquired human diseases. Here, we review how CRISPR technologies have stimulated key advances in dermatologic research. We discuss the role of CRISPR in genome editing for cutaneous disease and highlight studies on the use of CRISPR-Cas technologies for genodermatoses, cutaneous viruses and bacteria, and melanoma. Additionally, we examine key limitations of current CRISPR technologies, including the challenges these limitations pose for the widespread therapeutic application of CRISPR-based therapeutics.
Highlights
Gene editing technologies have been transformative in biological research and show immense potential for the study and treatment of inherited and acquired human diseases[1]
The skin is an accessible organ that allows for extraction and in vitro culture of target cells as well as direct localized administration of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cas therapeutics through topical, grafting or injection methods[3]
For a variety of reasons, dermatology is likely to continue to be at the center of the development and clinical application of CRISPR-Cas therapeutics
Summary
Gene editing technologies have been transformative in biological research and show immense potential for the study and treatment of inherited and acquired human diseases[1]. Researchers have leveraged CRISPR-Cas constructs to develop diverse treatment strategies for genodermatoses including the targeted addition of genes to specific genomic sites, the correction of diseasecausing point mutations, and the removal of disease-causing genes or genomic sequences. Such strategies expand the scope of gene therapy beyond additive approaches, allowing for corrective gene editing and the targeted ‘knockout’ of mutant alleles in dominant negative disorders. The studies reported here largely demonstrate the ability of CRISPR-Cas systems to treat human disease both in cell culture models and through ex vivo modification of primary patient cell lines While such methods currently represent the safest approach to gene editing in humans, such a technique is technologically challenging and of limited use for routine clinical practice. CRISPR therapeutics employing nucleases from bacteria to which humans are not exposed may not be subject to pre-existing immunity, allowing for a more robust genome editing effect
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