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| 2024-01-10 13:06:58 | onclick： | Virus When Vector Pilot Editing System Can Restore Partial Vision in Animals |
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Pilot editing is a generic form of gene editing that corrects most known disease-causing gene mutations.Scientists at MIT and Harvard's Broad Institute have designed virus-like particles to deliver the pilot editor to mouse cells with high enough efficiency to treat genetic diseases.The results of the study were published in the journal Nature Biotechnology on the 8th.
The team used their previously designed engineered virus-like particles (eVLP) to carry the base editor.The base editor is another precise gene editor that makes single-letter changes in DNA.
The researchers redesigned eVLP and some of the lead editing proteins and RNA mechanisms.Compared to eVLP, which previously provided a base editor, the new system improves editing efficiency in human cells by 170 times.The team corrected mutations in two disease-causing genes in mice models that inherited blindness, partially restoring their vision.They also transmitted the pilot editor to the mouse brain without detecting any off-target editing.
Liu Ruqian, senior author of the study and professor at Harvard University and director of the Melkin Institute for Healthcare Transformation Technology, said the study is a protein-RNA complex delivery that was first used to achieve therapeutic editing in animals.
The researchers improved the way the pilot-edited drug is packaged in eVLP, enabling it to be separated from the transport vector and delivered to the nucleus of the target cell.
When particles form, the lead editor drug must be effectively packaged into eVLP and effectively released after the target cell enters, the researchers said.All these steps must be carefully planned.
The team has tested the system in mice, correcting two different genetic mutations in the mice's eyes.In the case of two genetic mutations, eVLP corrected mutations in up to 20 percent of animal retinal cells, partially restoring their vision.
The study also showed that eVLP, equipped with a pilot editing mechanism, can effectively edit genes in the brains of live mice, and nearly half of the cells in the mouse cerebral cortex that receive editing show editing "traces."
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