Open a new strategy for gene therapy!

A few days ago, Professor David Liu, a well-known scientist in the field of gene editing, led a team to publish a paper in "Nature-Biotechnology", releasing a new version of the gene editing system "prime editing"

This breakthrough once again expands the capabilities of precise gene editing and brings us an important step toward safer gene therapy, with the potential to insert therapeutic genes in a safer and more targeted manner, replacing disease-causing mutations or missing genes, thereby achieve treatment goals

Two years ago, Professor Liu Ruqian's team published a blockbuster paper in the journal "Nature", bringing a new gene editing system called "lead editing"

In a variety of human cells, the researchers demonstrated the broad editing capabilities of lead editing, not only precisely modifying individual bases, but also inserting or deleting specific DNA segments at specific locations in the genome

▲ Schematic diagram of the pilot editing technology (Image source: Susanna Hamilton, Broad Institute)

However, the application of this system also encountered an important bottleneck, that is, the length of editable DNA

The new version of the pilot editing system published this time breaks through this limitation

▲The optimized twinPE system can insert large fragments of complete genes into the genome of human cells (Image source: Reference [2]; Credit: Ricardo Job-Reese, Broad Communications)

The research team dubbed the new version twinPE

To further edit larger fragments, the research team also used the twinPE system in combination with site-specific recombinases, which catalyze the integration of DNA into specific sites in the genome

The research team said they are also testing different recombinases in hopes of further improving the efficiency of twinPE by introducing longer gene sequences

It is worth mentioning that just two months ago, Professor Liu Ruqian's team just completed another optimization of the lead editor in the journal "Cell", which made the editing efficiency 2.

Note: The original text has been deleted

References:

[1] Andrew V.

[2] New prime editing system inserts entire genes in human cells.