TY - JOUR
T1 - Research Techniques Made Simple
T2 - The Application of CRISPR-Cas9 and Genome Editing in Investigative Dermatology
AU - Guitart, Joan Ramon
AU - Johnson, Jodi L.
AU - Chien, Wade W.
N1 - Funding Information:
Commercial Support Acknowledgment: This CME activity is supported by an educational grant from Lilly USA, LLC.
Publisher Copyright:
© 2016 The Authors
PY - 2016/9/1
Y1 - 2016/9/1
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=84992497254&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84992497254&partnerID=8YFLogxK
U2 - 10.1016/j.jid.2016.06.007
DO - 10.1016/j.jid.2016.06.007
M3 - Short survey
C2 - 27542298
AN - SCOPUS:84992497254
SN - 0022-202X
VL - 136
SP - e87-e93
JO - Journal of Investigative Dermatology
JF - Journal of Investigative Dermatology
IS - 9
ER -