An exciting new gene editing technology called prime editing now has an effective way to deliver its molecular machinery to cells inside living animals. Researchers at MIT and Harvard have engineered specialized virus-like particles that can carry prime editing components to cells and fix disease-causing genetic mutations.
Gene editing tools work by precisely editing DNA sequences. They have potential to permanently correct genetic defects that lead to many diseases. Prime editors are a particularly versatile form of gene editor. Unlike other popular editors CRISPR and base editors, prime editors can make virtually any change in DNA, not just small tweaks. This means prime editors could theoretically fix most known mutations that cause genetic diseases.
However, delivering these molecular editing machines to target cells in the body has been a major obstacle. The researchers overcame this by extensively redesigning both the virus-like delivery particles and parts of the prime editors themselves. The redesigned particles, called eVLPs, can now carry prime editing proteins and RNAs and deliver them to cells at very high efficiency.
In tests in mice, the researchers used the souped-up eVLPs to correct mutations causing blindness. Up to 20% of retina cells had mutations fixed, partially restoring vision. They also delivered prime editors to brain cells. The results demonstrate the technology works well enough inside living animals to have real therapeutic potential for treating genetic diseases.
Prime editing is still early in development but has tremendous possibilities. Being able to edit DNA precisely opens the door to permanently curing diseases caused by genetic errors. The delivery advance brings this a step closer to reality. There is still much work needed to advance the technology toward clinical use. But it’s an exciting development in the quest to make gene editing technologies safe and practical for treating patients.
See more at Broad Institute.