
Smaller and More Efficient CRISPR-Based Tool
October 4, 2023 |
Researchers from Japan developed a novel CRISPR-based tool, AsCas12f, with comparable effectiveness but smaller size. It can be transported into living cells via carrier viruses, which makes it more efficient.
AsCas12a and SpCas9 are widely used for gene editing in human cells. However, their large size makes it difficult to deliver them using adeno-associated virus (AAV) vectors. To address this problem, researchers combined deep mutational scanning with structure-informed design to produce two variants of enAsCas12f.
The enAsCas12f variants demonstrated gene editing capabilities in human cells comparable with those of AsCas12a and SpCas9. The enAsCas12f combined with partner genes in an AAV vector showed efficient knock-in and knock-out activities, as well as transcriptional activation in mice. When taken together, the enAsCas12f variants have the potential to be used for in vivo gene therapy of patients.
Read the journal article in Cell for more information.
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