The smallest

A mini genome editing system has been developed

<url> Researchers from the USA have created a miniature multifunctional genome editing system.

CasMINI is half the size of other CRISPR variants, which greatly facilitates its delivery to the cell. The scientists' article on the development was published in the journal Molecular Cell (Xu et al., Engineered miniature CRISPR-Cas system for mammalian genome regulation and editing).

The creation of CRISPR/Cas systems has opened the way for new ways to treat many diseases. However, their development is hindered by the size of the CRISPR system: they are often too large to penetrate the cell. The basis for a mini-system that would solve the problem, scientists from Stanford University chose the Cas12f protein (also known as Cas14) – compared to Cas9 or Cas12a, it is almost half as small: it contains from 400 to 700 amino acids. However, it developed in archaea – and was not adapted to work in mammalian cells.

The researchers identified 40 mutations that would solve this problem, and using protein engineering created a class of Cas12f variants that could activate a green fluorescent protein in mammalian cells and make them "glow". In addition, the researchers modified the RNA directing the protein to the desired DNA.

Scientists have successfully tested CasMINI's ability to alter and regulate the action of genes – including those associated with HIV infection and anemia - in cell cultures. The size of the resulting CasMINI molecule – 529 amino acids – allows it to be widely used for medicinal purposes, the researchers emphasize. At the same time, they admit, it has yet to be tested in vivo.

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