New Generation of Genetic Editing can Repair 90% of Genetic Diseases

A recent study published in the journal Nature reported that a gene called the “prime editor” was invented. Editing technology is more accurate than the CRISPR that was born in 2012, and the editing scope is wider. Researchers say they can “repair 89% of known human genetic diseases.”

CRISPR gene editing has a number of limitations, sometimes changing the genes outside the target to cause unexpected consequences. David Liu, a chemist at the Broad Institute of MIT and Harvard, one of the lead authors of the new study, said: “If Crispr-Cas9 is like a pair of scissors, the basic editor like pencils, prime editors are like word processors.”

According to Liu Ruqian’s calculations, this new gene editing technology can solve 89% of the known genetic diseases in humans. Their published study edited 175 genetic disease genes in human cell culture dishes, including anemia, cystic fibrosis, and family Tay-sachs.

The traditional CRISPR gene editing method consists of two parts: the biochemical enzyme Cas9 for cutting DNA, and the RNA that directs the biochemical enzyme to the target cleavage site. Other enzymes can perform different tasks, such as attaching to a gene to turn it off, or opening a letter in a DNA replacement.

The enzyme used in the new method is a combination of a molecule that acts as a cutting knife and another reverse transcriptase that turns RNA into DNA. The new method uses a different RNA, not only responsible for finding the target editing location, but also carrying a version for editing. When it finds the target DNA, it makes a small mark on the surface, and then the transcriptase adds the correct DNA sequence to the version brought by the RNA one by one, just like “the typewriter knocks out the letters one by one.” The result is two copies of DNA, one original (marked), and one edited. Finally, the cell repair mechanism is initiated, the original fragment is excised, and the edited version is “permanently left there.”

Gaetan Burgio, a geneticist at the Australian National University (ANU), who did not participate in the study, said the study “has great potential for cell editing and may be revolutionary.”

Bourgeois also mentioned that one of the big problem with this new approach is that the “editor” is too large to fit into the virus at the cellular level, and researchers often use viruses to send editing components into cells. . Such large volumes may also block needles for microinjection, which makes it very difficult to inject into mice, or human embryos.

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New Generation of Genetic Editing can Repair 90% of Genetic Diseases - /10

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A recent study published in the journal Nature reported that a gene called the "prime editor" was invented.

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