Release date: 2017-10-30

British "Nature" magazine published a heavy paper online on the 25th: The Bode Institute of the United States announced a new base editor - programmable protein machine, which is effective against bacteria and human DNA, and is more efficient than any other The genome editing method will promote the research of new therapies for human genetic diseases.

The DNA double helix consists of four chemical bases, namely adenine (A), guanine (G), cytosine (C), and thymine (T), where C and G are paired, and A and T are paired. In 2016, David Liu, a scientist at the Bode Institute, and colleagues described a modified base-based editing method based on CRISPR-Cas9. Base editing is effective because sometimes only a certain pair of bases mutate in the long strand of DNA. This phenomenon is called "point mutation." This improved method is popular because it corrects single-base mutations more efficiently than in the past, and is less prone to unintended DNA modification, and does not cause random deletion or insertion in the genome to cause DNA double-strand breaks (CRISPR- A common consequence of methods such as Cas9). But at the time, researchers could only convert G·C base pairs into T·A base pairs. In this latest study, the novel adenine base editor (ABE) has been able to convert A·T base pairs into G·C base pairs.

About half of all known disease-related single base pair mutations in humans involve the conversion of wild-type G·C base pairs into mutant A·T base pairs, so the base editor can recover a large number of such mutations. . It has been shown that it is effective against both bacterial cells and human cell DNA. In human cells, they are capable of introducing expected genetic mutations over a wide range of target regions with an efficiency of approximately 50%, which is higher than the efficiency of any other current genome editing method, and has almost no adverse side effects, allowing random insertion and deletion of other mutations. .

The new "base editor" rearranges the atoms of a DNA base into another base in the genome of a living cell, enabling efficient and selective replacement of all four DNA bases individually, without Causes any DNA double-strand breaks. It will be used to correct single-base mutations that cause genetic diseases, introduce single-base mutations that inhibit disease, and help to enhance human understanding of genetic diseases, and to promote humans to find new treatments for genetic diseases.

Source: Technology Daily

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