Scientists Restore Vision in Mice, Achieve 170-Fold Gene Editing Boost

Technology News ,Medical science :- Scientists from MIT and Harvard have discovered a more effective way to correct genetic mistakes using tiny, virus-like particles. These particles, called engineered virus-like particles (eVLPs), carry prime editors into mouse cells with great success, fixing genetic disorders.

Prime editing is a powerful technique for fixing genetic mutations that cause diseases. The researchers improved the editing process in human cells by 170 times by modifying these eVLPs. In tests on mice with genetic eye problems, they corrected mistakes and partially restored their vision.

The breakthrough is seen as a promising treatment for genetic disorders in living animals. Importantly, when the same method was used to edit genes in the mouse brain, there were no unintended changes. This study marks the first time that therapeutic prime editing has been successfully delivered in an animal using protein-RNA complexes, according to David Liu, the senior author of the study.

Scientists have been exploring gene editing to address genetic issues causing diseases. Prime editing, introduced in 2019, allows precise and diverse changes in DNA. However, delivering this editing system into living animal cells has been challenging.

Different methods have been used, including lipid nanoparticles and viruses, with virus-like particles (VLPs) showing promise. VLPs are made of viral proteins and carry cargo but do not contain viral genetic material. Although VLPs have had some success, they require specific engineering for each cargo type.

In their study, Liu and his team used their system to fix two different genetic problems in the eyes of mice. One issue causes a disease called retinitis pigmentosa, leading to gradual vision loss, and the other is linked to blindness in a condition called Leber congenital amaurosis (LCA) in humans.

Using their eVLPs, they corrected the mutations in about 20 percent of the animals" retina cells, partially restoring their vision. The researchers made improvements to how the prime editing cargo was packaged, separated from the delivery vehicle, and entered the target cells" nuclei. These coordinated enhancements led to a 100-fold increase in efficiency, making the technique potentially suitable for therapeutic use in animals.

"This kind of improvement in efficiency should be enough to give us therapeutically relevant levels of prime editing," said Liu. "But we did not know for sure until we tested it in animals."