Researchers develop new methods to correct disease-causing gene mutations to make gene therapy safer

The top academic journal "Cell" recently published an important paper online.

"This technological advancement can be used to accelerate the establishment of animal disease models.

Professor Feng Guoping’s team is committed to studying the mechanisms of neurodevelopment and mental illness.

The application of gene editing tool CRISPR has greatly helped the creation of such animal disease models.

However, the researchers pointed out that using this method to construct disease models may still take months or even years.

In order to improve the efficiency of the gene editing process, in this research, Professor Feng Guoping's team initially envisioned adding a DNA repair protein RAD51 to the standard CRISPR gene editing tool to allow the target cell-mouse fertilized eggs to be produced as desired.

The researchers tested the autism-related gene Chd2 and inserted a point mutation into the gene.

"Generally speaking, it is very rare to edit two chromosomes at the same time," explained Dr.

As a result, the research team turned to focus on studying the role of RAD51 in gene editing.

▲Research diagram: The addition of RAD51 significantly increases the ratio of repairs between homologs (picture source: reference [1])

"Previous studies on IHR have shown that the efficiency of IHR in most cells is very low," Dr.

"HDR-based strategies still have the problem of low efficiency, and there is also the risk of unnecessary integration of foreign DNA in the entire genome," Professor Feng added.

This research team is still looking for other DNA repair-related proteins that can stimulate IHR, gain insight into the mechanism of IHR, and finally apply this technology to develop a safer method for gene therapy.

Reference materials:

[1] Jonathan J.

[2] McGovern lab develops new technique for correcting disease-causing mutations.