MIT Researchers Develop Enhanced Gene Editing Technique for Human Cells

MIT and Broad Institute researchers have advanced gene editing, enabling efficient insertion or replacement of entire genes in human cells. Led by David Liu, this development could revolutionize treatments for diseases like cystic fibrosis, where multiple gene mutations are involved.

The new method, eePASSIGE, utilizes prime editing and a novel recombinase to insert large DNA segments directly into the genome's native locations. This approach surpasses traditional methods, which often require multiple edits for each mutation.

Liu's team enhanced the efficiency of gene integration by evolving a more effective version of the Bxb1 recombinase. The improved eePASSIGE system can integrate up to 30% of a gene's size, significantly more than previous methods.

This breakthrough could lead to a single gene therapy capable of treating a wide range of genetic disorders, regardless of specific mutations. By preserving the surrounding DNA sequence, the therapy aims to ensure proper gene regulation, avoiding over- or under-expression.

Liu's ongoing work focuses on integrating eePASSIGE with delivery systems like engineered virus-like particles (eVLPs) to overcome barriers in therapeutic gene delivery. This could pave the way for effective in vivo gene editing treatments.