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How has CRISPR-Cas9 been used to create models of human disease in laboratory animals?
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genetic engineering
The generation of vaccines relies heavily on techniques in genetic engineering.
CRISPR-Cas9 has revolutionized the creation of animal models for human disease by allowing researchers to make precise, efficient, and cost-effective modifications to the genomes of various laboratory organisms, including mice, rats, and nonhuman primates.12
Scientists utilize this gene editing technology in several ways to establish these models:
- Embryonic Modification: CRISPR-Cas9 can be applied to early embryos to introduce specific genetic changes that mimic human disease conditions.1
- Systemic Delivery: The technology can be injected into the bloodstream of laboratory animals, enabling substantial gene editing within specific subsets of tissues.1
- Functional Validation: By modifying genes, researchers can investigate the effects of genetic modification and gain new insights into disease mechanisms.2
In a notable application, CRISPR-Cas9 was used to successfully correct a genetic error in a mouse model of human disease, which resulted in the clinical rescue of the affected animals. These advancements are essential for understanding disease pathology and developing potential therapies, though they also necessitate rigorous ethical oversight regarding the use of such powerful genetic tools.12