According to a recent report on the website of the British journal Nature, at least three RNA editing therapies are currently being approved or have entered clinical trials. Supporters believe that this technology may be safer and more flexible than genome editing techniques such as CRISPR. RNA, which is both fragile and powerful, is a fragile and unstable molecule that rapidly decomposes, resulting in a short lifespan. But it has a wide range of uses and is crucial for human survival. RNA editing technology compensates for harmful mutations by altering RNA sequences, allowing normal protein synthesis. RNA editing can also increase the production of beneficial proteins. Unlike CRISPR genome editing, RNA editing does not alter genes or produce permanent changes. Joshua Rosenthal, a neurobiologist at the Biological Laboratory of the Woods Hole Institute of Oceanography in the United States, pointed out that this transience may bring a safety advantage. One risk of CRISPR therapy, which can "cleave" DNA, is off target effects. If off target effects occur within RNA, the risk is much smaller. Single base editing is a common RNA editing method that utilizes an enzyme within the cell, adenosine deaminase (ADAR), which acts on RNA. This enzyme replaces the base adenine in the RNA sequence with the base inosine. Wave Life Sciences in the United States is exploring single base editing technology to treat a genetic disease—— α- 1. Antitrypsin deficiency. This disease will reduce the production of protective protein AAT. AAT can protect the lungs from damage caused by polluted air or other irritants. The company's product is a short chain nucleotide that guides naturally occurring ADAR enzymes to alter specific bases in each mRNA molecule to correct mutations that affect AAT production. Mouse experiments have shown that the drug edits about 50% of the target mRNA in liver cells, which is sufficient to produce therapeutic effects. The company began clinical trials of this drug in the UK and Australia in December last year. Another method of exon editing is called RNA exon editing. It can alter thousands of genetic bases in RNA molecules at once. This technology is particularly important for treating diseases caused by multiple gene mutations. This technology targets forward messenger ribonucleic acid (pre mRNA). Pre mRNA includes exons and introns. Exons are the parts of RNA transcripts that contain instructions for manufacturing proteins, while introns do not contain such instructions. The RNA splicing mechanism removes introns from pre mRNA and connects them with exons to form the final mRNA, which is then translated into proteins. Ascidian Medical, located in Boston, is using RNA splicing processes to remove exons containing mutations and replace them with healthy exon genes. In January of this year, the company obtained approval from the US Food and Drug Administration to conduct clinical trials of exon editors to treat Sturger's disease, which causes vision loss. This is a juvenile macular degeneration disease. Ascidian's therapy relies on a DNA fragment that has been genetically processed and delivered
Edit：GuoGuo Responsible editor：FangZhiYou
Source：People.cn
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