Researchers say a compact CRISPR system and an engineered variant could make gene editing easier to deliver inside the body.
The National Institutes of Health says researchers have developed a smaller CRISPR system that could make in-body gene editing easier to deliver.
The work centers on a naturally occurring Cas12f enzyme called Al3Cas12f, which is small enough to fit into adeno-associated virus, or AAV, delivery systems. Researchers also engineered a variant called RKK that improved editing efficiency in human cells.
The NIH said the advance could help broaden future therapeutic applications, including for diseases such as cancer and ALS. The supporting paper was published the same day in Nature Structural & Molecular Biology.
The key value of the work is size: smaller genome-editing tools are easier to package and may be more practical for precision delivery inside the body.
Follow-up studies will need to show how the system performs in vivo and whether it can translate into safe, effective treatments.
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