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
The discovery of the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system has revolutionized gene editing research
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
Two epidermolysis bullosa (EB) subtypes—EB Simplex (EBS) and dominant dystrophic EB (DDEB)—are caused by dominant negative mutations that cannot be corrected with traditional additive gene therapies
Summary
Two EB subtypes—EB Simplex (EBS) and dominant dystrophic EB (DDEB)—are caused by dominant negative mutations that cannot be corrected with traditional additive gene therapies These disorders are well-suited for treatment with CRISPR-Cas genome editing, which allows for the direct modification of the dominant, disease-causing allele. 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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