12:00 AM
May 16, 2013
 |  BC Innovations  |  Tools & Techniques

CRISPR model building

Boston researchers have used the CRISPR-Cas9 genome modification platform to simultaneously engineer mutations into multiple genes in mice.1 The results from the rapid, one-step process provide the best evidence to date for the potential of this method to revolutionize the creation of complex disease models.

Earlier this year, five separate teams reported on how the CRISPR (clustered, regularly interspaced short palindromic repeats)-Cas9 (CRISPR-associated protein 9) system could be adapted to engineer site-specific mutations in the genomes of mammals, bacteria and zebrafish.2-7 The method was derived from a recently identified acquired immunity-like system in bacteria, in which CRISPR-associated proteins, including Cas9, are guided by short CRISPR-encoded RNAs to cleave homologous foreign DNA contained within plasmids or bacteriophages.

Unlike other approaches that rely on large protein domains such as zinc fingers or transcription activator-like effectors (TALEs) to guide site-specific DNA editing, the specificity of Cas9 is dictated solely by DNA-RNA base pairing, greatly enhancing ease of use. Because of this, researchers and biotech executives told SciBX in January that the system was likely to be rapidly developed and widely used for functional genetic analysis.8

Now, less than four months after the proof of concept for using the method to modify a single gene at a time in cell lines was published, researchers at the Whitehead Institute for Biomedical Research and the Broad Institute of MIT and Harvard have...

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