New genome editing technology

Verve Therapeutics has proposed a new genome editing technology. Unlike the well-known CRISSPR gene editing system, it is also often called "molecular scissors", the proposed method does not cut DNA vertically along the chain, but makes correction at one "base" or a letter in the genome: without affecting nucleotides, one letter is replaced by another.

Two studies have been performed. In a monkey trial the experts have proved that this method disables PCSK9 or ANGPTL3 genes, which are associated with cardiovascular pathology. The studies have shown the preservation of the genetic code in non-target body cells and the absence of side effects. The CEO of Verve Therapeutics said that they were able to develop a concept for a new method to prevent strokes and heart attacks. In the long term, this treatment method, even with a single use, will contribute to the regular control of low cholesterol and triglyceride levels. Clinical trials are scheduled to be released in 2022 or 2023.

SOURCE: FIERCE BIOTECH