Enhanced Prime Editing System Creates Gene-Sized Edits in Human Cells
June 13, 2024| |
Researchers from the Broad Institute of MIT and Harvard and partners developed a system that can insert or substitute genes in human cells. Their study demonstrates great potential for therapeutic applications of human diseases.
Current methods of inserting genes in mammalian genomes have low programmability, efficiencies, or specificities. Due to this, it is difficult to treat genetic diseases, such as cystic fibrosis and phenylketonuria.
To help with this issue, the scientists combined prime editing with newly developed recombinase enzymes to produce the eePASSIGE system. This versatile method can create gene-sized edits that are more efficient compared to other similar techniques.
"This system offers promising opportunities for cell therapies where it can be used to precisely insert genes into cells outside of the body before administering them to patients to treat disease, among other applications," said Smriti Pandey, one of the authors of the study.
Read the article on Nature Biomedical Engineering for more information.
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