Progress and Application of CRISPR/Cas Technology in Biological and Biomedical Investigation

Abstract

Based on the tremendous progress of the understanding on the CRISPR-Cas systems, the application CRISPR/Cas technology has been extended into increasing scenarios in biological and biomedical investigation. The potency of gene editing has been greatly improved by the rapid development of engineered Cas9 variants and modified CRISPR platforms. As advanced sequencing technology identified vast causative genetic basis for human diseases, CRISPR toolkits are now able to mediate precise genetic disruption or correction in vitro and in vivo. In this review, we have discussed the recent development of the CRISPR/Cas gene-editing technology and the extensive applications of the CRISPR platforms in biological and biomedical investigation, including disease modeling in animal and human cell line, development of gene therapy, as well as high-throughput genetic screening. J. Cell. Biochem. 118: 3061-3071, 2017. © 2017 Wiley Periodicals, Inc.

The Progress of CRISPR/Cas9-Mediated Gene Editing in Generating Mouse/Zebrafish Models of Human Skeletal Diseases

“…Since then, various disease models have been generated using the CRISPR/Cas9 system. Some adaptations, including using organism-specific codons in Cas9 RNA, were made to the CRISPR/Cas9 system to increase its activity in different organisms [[41], [42], [43], [44], [45]]. The RNA-guided targeting activity of the CRISPR/Cas9 system also potentiates it for other sequence-specific activities besides generating DSBs, such as transcription regulation, epigenomic modification, and base editing [[46], [47], [48], [49]].…”

Sleep Disorders and Genes

“…Briefly, CRISPR uses a guide RNA to send biological "scissors" -usually the CRISPR-associated protein, Cas9-to a precise spot to cut and make a substitution in the genome. As one can imagine, CRISPR-Cas9 provides the technology to manipulate genomic sequences, focusing in human diseases, such as HIV and cancer (95)(96)(97)(98)(99).…”

The End of Snoring? Application of CRISPR/Cas9 Genome Editing for Sleep Disorders

“…The advent of site-specific nucleases, particularly CRISPR/Cas9, provides the ability to manipulate genomic sequences, with special interest for develop treatments for human diseases [73,74]. The biomedical applications of CRISPR are just starting to emerge with a promising active role in the ability to remove genetic mutations associated with diseases, such as cystic fibrosis, HIV, and even certain types of cancers [75][76][77][78][79][80].…”