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2016 ; 136
(9
): e87-e93
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Research Techniques Made Simple: The Application of CRISPR-Cas9 and Genome
Editing in Investigative Dermatology
#MMPMID27542298
Guitart JR Jr
; Johnson JL
; Chien WW
J Invest Dermatol
2016[Sep]; 136
(9
): e87-e93
PMID27542298
show ga
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.