CRISPR-Cas9-based genome editing enables the rapid genetic manipulation of any genomic locus without the need for gene targeting by homologous recombination. It can be used in the search for new treatment modalities, e.g. studies focusing on the identification of physiological targets of drugs and bioactive small molecules (Zhu et al. 2015; Kasap et al 2014). One of the benefits of this approach is that it can help uncover the mechanisms of drug action for existing drugs, thus enabling the trial of other candidate drugs with similar function. More recently, a conditional transgenic approach using CRISPR-Cas9-based genome editing was shown to allow temporal control of CRISPR-Cas9 activity for inducible genome editing in adult mice (via doxycycline-regulated Cas9 induction). This inducible CRISPR (iCRISPR) system can also be used effectively to create biallelic mutation in multiple target loci and, thus, provides a flexible and fast platform to study loss-of-function phenotypes in vivo.
The CRISPR/Cas9 system inactivates latent HIV-1 proviral DNA. Zhu et al. Retrovirology. 2015 Feb 27;12(1):22. PMID:25808449
DrugTargetSeqR: a genomics- and CRISPR-Cas9-based method to analyze drug targets. Kasap C, Elemento O, Kapoor TM. Nat Chem Biol. 2014 Aug;10(8):626-8. PMID: 24929528
Inducible in vivo genome editing with CRISPR-Cas9. Dow et al. Nat Biotechnol. 2015 Feb 18. PMID: 25690852
posted by Yannis Trakadis, MD