Journal
TRANSLATIONAL RESEARCH
Volume 168, Issue -, Pages 15-21Publisher
ELSEVIER SCIENCE INC
DOI: 10.1016/j.trsl.2015.09.008
Keywords
-
Categories
Funding
- Swiss National Science Foundation [31003A_160230]
- Human Frontiers in Science Program [LT000422/2012]
- Swiss National Science Foundation (SNF) [31003A_160230] Funding Source: Swiss National Science Foundation (SNF)
Ask authors/readers for more resources
Targeted nucleases are widely used as tools for genome editing. Two years ago the clustered regularly interspaced short palindromic repeat (CRISPR)-associated Cas9 nuclease was used for the first time, and since then has largely revolutionized the field. The tremendous success of the CRISPR/Cas9 genome editing tool is powered by the ease design principle of the guide RNA that targets Cas9 to the desired DNA locus, and by the high specificity and efficiency of CRISPR/Cas9-generated DNA breaks. Several studies recently used CRISPR/Cas9 to successfully modulate disease-causing alleles in vivo in animal models and ex vivo in somatic and induced pluripotent stem cells, raising hope for therapeutic genome editing in the clinics. In this review, we will summarize and discuss such preclinical CRISPR/Cas9 gene therapy reports.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available