Scientists use lipid nanoparticles to precisely target liver gene editing

IMAGE: Researchers have developed nanoparticles that carry a gene-editing package specifically to the liver in mice, successfully reducing levels of “bad” LDL cholesterol in the blood. With previous studies showing… see more

Credit: Tufts University

CRISPR genome editing technology has emerged as a powerful new tool that can change the way we treat disease. The challenge in altering the genetics of our cells, however, is how to do it safely, effectively, and specifically targeting the gene, tissue, and organ that require treatment. Scientists at Tufts University and the Broad Institute at Harvard and MIT have developed unique nanoparticles made up of lipids – fat molecules – capable of conditioning and delivering gene-editing machines specifically to the liver. In a study published today in the Proceedings of the National Academy of Sciences, they showed that they could use lipid nanoparticles (LNPs) to efficiently deliver the CRISPR machinery into the livers of mice, resulting in specific genome modification and reduction of blood. cholesterol levels up to 57% – a reduction that can last at least several …

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