Researchers from the United States have created a miniature multifunctional genome editing system. Casmini is half less than other CRISPR options, which greatly facilitates its delivery to the cage. Developed by the article by scientists was published in Molecular Cell magazine.
Creating CRISPR / CAS systems opened the way for new ways to treat many diseases. However, their development is hampered by the size of the CRISPr system: they are often too high in order to penetrate the cage. The basis for the mini-system that would solve the problem, scientists from Stanford University chose CAS12F protein (also known as CAS14) – compared to CAS9 or CAS12A it is almost half less: it is from 400 to 700 amino acids. However, he developed at Archey – and was not adapted to work in mammalian cells.
Researchers allocated 40 mutations that would solve this problem, and using protein engineering created a class of CAS12F variants, which could activate in mammalian fluorescent cells and force them to “glow”. In addition, the researchers modified RNA, a protein guide to the desired DNA.
Scientists have successfully tested the ability of Casmini to change and regulate the effect 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 therapeutic purposes, emphasize researchers. At the same time, they recognize, to experience it in vivo still have to be.