According to a study recently published in the journal Molecular Cell, a compact and efficient CRISPR-Cas system called CasMINI can be widely used in cell engineering and gene therapy because it is easier to enter cells
Qi Lei, assistant professor of bioengineering at Stanford University in the United States, said that this is a key step forward for the application of CRISPR genome engineering
The CRISPR-Cas system provides opportunities for the development of gene therapies for various genetic diseases, but their size is too large and their delivery to cells, tissues or organisms will be limited, which hinders clinical applications.
One possible solution is Cas12f
In this new research, Qi Lei and his team applied RNA and protein engineering to the Cas12f system to generate an efficient mini-Cas system for mammalian genome engineering
The engineered Cas12f protein variant is combined with the engineered single guide RNA to show effective gene regulation and gene editing activities
The size of the engineered CasMINI molecule is only 529 amino acids
"The accessibility of mini-CasMINI enables new applications, from in vitro applications (such as designing better tumor killer lymphocytes or reprogramming stem cells) to in vivo gene therapy (treating genetic diseases of the eye, muscle or liver)
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