CRISPR gene editing moves into living animals, opening new frontiers in disease research

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Scientists are taking gene-editing research to the next level – out of the petri dish and into living animals. A new review by researchers from Randall Platt’s Biological Engineering lab which highlights how scientists are using CRISPR, the revolutionary genetic “scissors,” to study how genes work inside living organisms. The approach could dramatically speed up discoveries in medicine, from understanding cancer to developing new treatments for rare diseases.

For years, CRISPR has allowed scientists to switch genes on or off in cells grown in labs. But these lab-grown cells can’t fully replicate the complexity of the human body. That’s why researchers are now turning to in vivo CRISPR screening – a method that allows gene-editing experiments to take place inside living animals, usually mice.

“This is about moving from simplified models to the real-world biology happening inside the body,” the authors of the review published by Nature Reviews Genetics write.

These advanced screens allow scientists to test the function of dozens – or even thousands – of genes at once, directly inside specific organs like the brain, lungs, or liver. By doing so, researchers can see how genes behave in the context of entire systems, where cells interact with one another and respond to real-life conditions like disease, immune responses, or drug treatments.

The review compiles available knowledge on methods and applications of in vivo CRISPR screening, explaining how these in vivo experiments are designed, how CRISPR is delivered to the right tissues, and how scientists interpret the vast amount of data that comes from these studies. It also explores how powerful tools like artificial intelligence and multi-omics, which looks at DNA, RNA, proteins, and more, are helping make sense of it all.

The authors believe that in vivo CRISPR screening could become a key driver of both basic science and medical breakthroughs, mapping out which genes do what, how they interact, and which ones might be targets for future therapies.

As the review concludes, this emerging field is still in its early days, but the potential is enormous: a faster, more precise way to understand how our genes influence health – and how we might fix them when things go wrong.

Find original article published in Nature Reviews Genetics:

Santinha, A.J., Strano, A. & Platt, R.J. (2025) external page Methods and applications of in vivo CRISPR screening. Nat Rev Genet. https://doi.org/10.1038/s41576-025-00873-8

\ Learn about research in the Biological Engineering laboratory led by Randall Platt.