title: Research Techniques Made Simple: The Application of CRISPR-Cas9 and Genome Editing in Investigative Dermatology.
abstract: Designer nucleases have gained widespread attention for their ability to precisely modify genomic DNA in a programmable manner. These genome-editing nucleases make double-stranded breaks at specified loci, and desired changes can be made to modify, ablate, or excise target genes. This technology has been used widely to develop human disease models in laboratory animals and to study gene functions by silencing, activating, or modifying them. Furthermore, the recent discovery of a bacterially derived programmable nuclease termed clustered regularly interspaced palindromic repeats (CRISPR)-associated protein 9 (Cas9) has revolutionized the field because of its versatility and wide applicability. In this article, we discuss various modalities used to achieve genome editing with an emphasis on CRISPR-Cas9. We discuss genome-editing strategies to either repair or ablate target genes, with emphasis on their applications for investigating dermatological diseases. Additionally, we highlight preclinical studies showing the potential of genome editing as a therapy for congenital blistering diseases and as an antimicrobial agent, and we discuss limitations and future directions of this technology.
authors.affiliation: National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, Maryland, USA. Electronic address: joan.guitart@northwestern.edu.
authors.forename: Joan Ramon
authors.initials: JR
authors.lastname: Guitart
authors.affiliation: Northwestern University Feinberg School of Medicine, Departments of Pathology and Dermatology, Chicago, Illinois, USA.
authors.forename: Jodi L
authors.initials: JL
authors.lastname: Johnson
authors.affiliation: National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, Maryland, USA; Johns Hopkins School of Medicine, Department of Otolaryngology-Head & Neck Surgery, Baltimore, Maryland, USA.
authors.forename: Wade W
authors.initials: WW
authors.lastname: Chien
mesh_terms: D000818:Animals; D064113:CRISPR-Cas Systems / Q000235:genetics*; D003880:Dermatology / Q000379:methods*; D005544:Forecasting; D000072669:Gene Editing / Q000379:methods*; D005818:Genetic Engineering / Q000379:methods / Q000639:trends*; D006801:Humans; D012107:Research Design