OVER £4m of funding has been awarded to the University of Dundee for gene editing research that could help treat cancer, neurodegeneration and other diseases.
The £4.4m of funding has been awarded by UK Research and Innovation (UKRI) for the UNLEASH project, whilst a matching amount was also granted by the European Research Council (ERC) for collaborators.
Researchers David Gray and Angus Lamond will work on the UNLEASH project with colleagues in Germany and Spain with the aim of developing small molecules that can control alternative splicing (AS), the process responsible for disease development.
Genes are pieces of DNA that contain the information required to make different types of proteins that build and maintain living organisms.
In order to instruct the cell how to build proteins, DNA must first be copied into a molecule called RNA.
Regions which are not essential for coding are spliced out to create mRNA, which assembles the information required to produce specific proteins.
The importance of mRNA molecules and how to engineer them was demonstrated by Covid-19 vaccines.
AS causes the information contained in human genes to ‘split’ by allowing a single gene to code for multiple proteins.
In medical terms, it enables the production of different messenger RNAs and proteins from a single gene.
The aim of the research is to video-edit our genes in order to manipulate this process accurately.
If successful, this project can help pharmaceutical companies develop new therapeutical approaches for treating human diseases.
Professor Lamond said: “The gene can be seen as the raw video footage that is captured, while the mRNA product that delivers the instructions for proteins is like edited video footage, where the editor has removed unwanted regions to form the final video story.
“Just as a skilled video editor can edit their raw footage in different ways to create more than one alternative video sequence, so too can human genes be edited differently, thus creating alternative final mRNA products that code for different types of proteins.
“AS holds the key to understanding how our genes function and for the development of new therapeutics for a host of human diseases, many of which are known to alter the splicing process.”
Professor Gray said: “In principle, there are a wide array of cancers and neurodegenerative diseases we can treat if we can understand how to manipulate alternative splicing events.
“We know that alternative splicing is highly relevant to disease, but there is a knowledge gap that must be addressed if we are to develop these drugs.”
UNLEASH combines complementary expertise in chemical, structural, molecular, cellular and systems biology, and Artificial Intelligence to be found at Dundee, the Helmholtz Center in Munich, and Barcelona’s Center for Genomic Regulation.
