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How do scientists use CRISPR to create animal models of human disease?
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gene editing
The CRISPR-Cas9 gene-editing complex from the bacterium Streptococcus pyogenes.
Scientists use CRISPR-Cas9 technology to create animal models of human disease by leveraging its high precision to modify the genomes of laboratory organisms, such as mice, rats, and nonhuman primates.1
The process generally involves the following steps:
- Targeting: Researchers design specific "guide RNA" sequences that correspond to the target DNA in the animal's genome. Because these guide RNAs are based on simple base-pairing rules, they can be easily customized to bind to unique locations.12
- Cleavage: The guide RNA directs a nuclease, such as Cas9 or Cpf-1, to the desired site, where it introduces a double-stranded break in the DNA.12
- Modification: This break allows scientists to remove, insert, or replace specific DNA sequences, effectively introducing the genetic mutations associated with a particular human disease into the animal model.12
By modifying the genomes of these models, researchers can gain a deeper understanding of the mechanisms behind genetic diseases and test potential therapies. In some cases, this technology has even been used to correct genetic errors within these models, leading to the clinical rescue of diseased animals.12