A New Approach to Gene Therapy

When listening to the Chalk-Talks this week, there was one that I remember immediately seeing the potential real-world/medical applications to and that was Nico Rey’s talk. Nico’s project provides a potential new mechanism through which gene therapy can be carried out. As spoken about in his chalk talk, the older model through which this was done was to introduce a subject to a viral vector that carries a functional form of the targeted gene so that cellular function  can be restored through the production of a functional form of the otherwise dysfunctional protein.

Rather than maintaining the use of a viral vector to introduce entire new genes, Nico’s project utilizes the splicing machinery native to the cells to replace the mutated/dysfunctional exons of the pre-mRNA from the target gene with a functional form of the exons thus rendering the protein and cellular function normal again.

Leave a Reply

Your email address will not be published. Required fields are marked *