title: CRISPR/Cas9 system as an innovative genetic engineering tool: Enhancements in sequence specificity and delivery methods.
abstract: While human gene therapy has gained significant attention for its therapeutic promise, CRISPR/Cas9 technology has made a breakthrough as an efficient genome editing tool by emulating prokaryotic immune defense mechanisms. Although many studies have found that CRISPR/Cas9 technology is more efficient, specific and manipulable than previous generations of gene editing tools, it can be further improved by elevating its overall efficiency in a higher frequency of genome modifications and reducing its off-target effects. Here, we review the development of CRISPR/Cas9 technology, focusing on enhancement of its sequence specificity, reduction of off-target effects and delivery systems. Moreover, we describe recent successful applications of CRISPR/Cas9 technology in laboratory and clinical studies.
authors.affiliation: Brandeis University, Waltham, MA, United States.
authors.forename: Young-Il
authors.initials: YI
authors.lastname: Jo
authors.affiliation: Department of Pharmacology, Brain Korea 21 PLUS Project for Medical Science, Yonsei University College of Medicine, Seoul, South Korea; Department of Physiology, Graduate Program of Nano Science and Technology, Yonsei University, Seoul, South Korea.
authors.forename: Bharathi
authors.initials: B
authors.lastname: Suresh
authors.affiliation: Department of Pharmacology, Brain Korea 21 PLUS Project for Medical Science, Yonsei University College of Medicine, Seoul, South Korea; Department of Physiology, Graduate Program of Nano Science and Technology, Yonsei University, Seoul, South Korea.
authors.forename: Hyongbum
authors.initials: H
authors.lastname: Kim
authors.affiliation: Graduate School of Biomedical Science and Engineering, Hanyang University, Seoul, South Korea; College of Medicine, Hanyang University, Seoul, South Korea. Electronic address: suri28@hanyang.ac.kr.
authors.forename: Suresh
authors.initials: S
authors.lastname: Ramakrishna
mesh_terms: D001483:Base Sequence; D064130:CRISPR-Associated Proteins / Q000235:genetics*; D064113:CRISPR-Cas Systems / Q000235:genetics*; D064112:Clustered Regularly Interspaced Short Palindromic Repeats / Q000235:genetics*; D004247:DNA / Q000235:genetics*; D005818:Genetic Engineering / Q000379:methods*; D008969:Molecular Sequence Data; D015203:Reproducibility of Results; D012680:Sensitivity and Specificity; D014162:Transfection / Q000379:methods*
chemical_list: D064130:CRISPR-Associated Proteins; D004247:DNA
keywords: CRISPR/Cas9 applications; Delivery methods; Genome editing; Reduced off-target effects; Sequence specificity; Therapeutics