title: Application of CRISPR/Cas9 for biomedical discoveries.
abstract: The Clustered Regions of Interspersed Palindromic Repeats-Cas9 (CRISPR/Cas9), a viral defense system found in bacteria and archaea, has emerged as a tour de force genome editing tool. The CRISPR/Cas9 system is much easier to customize and optimize because the site selection for DNA cleavage is guided by a short sequence of RNA rather than an engineered protein as in the systems of zinc finger nucleases (ZFN), transcription activator-like effector nucleases (TALEN), and meganucleases. Although it still suffers from some off-target effects, the CRISPR/Cas9 system has been broadly and successfully applied for biomedical discoveries in a number of areas. In this review, we present a brief history and development of the CRISPR system and focus on the application of this genome editing technology for biomedical discoveries. We then present concise concluding remarks and future directions for this fast moving field.
authors.affiliation: Department of Pediatrics, The Children's Mercy Hospital, Kansas City, MO USA.
authors.forename: Sean M
authors.initials: SM
authors.lastname: Riordan
authors.affiliation: Department of Pediatrics, The Children's Mercy Hospital, Kansas City, MO USA.
authors.forename: Daniel P
authors.initials: DP
authors.lastname: Heruth
authors.affiliation: Department of Pediatrics, The Children's Mercy Hospital, Kansas City, MO USA.
authors.forename: Li Q
authors.initials: LQ
authors.lastname: Zhang
authors.affiliation: Department of Pediatrics, The Children's Mercy Hospital, Kansas City, MO USA ; Department of Biomedical and Health Informatics, University of Missouri Kansas City School of Medicine, Kansas City, MO USA.
authors.forename: Shui Qing
authors.initials: SQ
authors.lastname: Ye
keywords: Animal model; CRISPR; Cas9; Genome editing