CRISPR-Cas9 has rapidly transformed our ability to perform targeted gene editing. While the technique has received much interest for its potential in the field of gene therapy, advances in its high-throughput use mean it can now open up a wealth of opportunities in drug discovery too.
CRISPR-Cas9, short for clustered regularlyinterspaced short palindromic repeats and CRISPR-associated protein-9, has quickly established itself as an important tool for precision gene editing. This powerful technology has transformed our ability to precisely target genomic sites, proving to be faster, cheaper and more accurate than other existing genome editing methods.
While other gene editing technologies such as zinc finger nucleases (ZFNs) and transcription activator- like effector nucleases (TALENs) have played a key role in expanding our gene editing capabilities, these approaches have their limitations. Unlike ZFN and TALEN approaches, CRISPRCas9 uses short synthetic guide RNAs (gRNAs) that direct the Cas9 endonuclease to act on specific genomic sites. As this specificity results from the gRNA, changing the target requires a relatively simple tweak to the RNA sequence.
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