SMRTR Science & EngineeringMay 15, 2025Live Science

New CRISPR alternative can 'install' whole genes, paving the way to treatment for many genetic disorders

SMRTR summary

Scientists at Harvard have unveiled a groundbreaking gene-editing tool that could revolutionize treatment for genetic diseases. Unlike current methods that target specific DNA mutations, this new system can weave entire genes into human DNA.

"A single genetic disease can be caused by many different mutations in that gene," explains Isaac Witte, a doctoral student at Harvard and co-lead author of the study. Take cystic fibrosis, for instance, which can result from over 2,000 distinct mutations.

The researchers evolved a bacterial system called CAST to work efficiently in human cells, dubbing it "evoCAST". This ingenious approach sidesteps the need for DNA cutting, potentially reducing unwanted mutations.

While still in early stages, evoCAST shows promise for treating a range of genetic conditions and even enhancing cancer immunotherapies. However, funding cuts to the NIH pose challenges for further research in this exciting field.

SMRTR provides this summary for quick context. The original article belongs to Live Science.

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