A novel genome-editing tool described in Science offers a breakthrough method for inserting entire genes into human DNA efficiently and precisely, without DNA cutting. This innovation could revolutionize gene-correction therapies, making them applicable to various mutations in diseases. It utilizes a CRISPR-associated transposase (CAST) to facilitate the targeted introduction of genes, addressing limitations found in previous methods, such as random insertion by viruses and the complexity of CRISPR's DNA cutting. Major implications include improvements in cancer cell therapies and research model creation.
CRISPR offers more control than viral carriers do, but typically requires the cutting of DNA - raising the chances of unwanted mutations and incomplete repair.
The new system circumvents both problems by introducing full-length genes at targeted sites in a single step, without cutting DNA or needing bespoke designs.
It could really be a big part of the future, says study co-author David Liu, a chemical biologist at the Broad Institute.
This innovative genome-editing tool could pave the way for gene-correction therapies that would be effective regardless of the specific mutation causing an individual's disease.
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