Targeted alternative splicing: a common therapeutic platform to treat inherited diseases [ 2018 - 2020 ]

Research Grant

Researchers: Prof Stephen Wilton (Principal investigator) ,  Prof Frank Mastaglia ,  Prof Minghao Zheng ,  Prof Sue Fletcher

Brief description Genes consist of exons (protein coding domains) separated by introns (non-coding intervening sequences). It is now evident that not every exon need be included in the gene message to direct synthesis of a functional gene product. This application seeks to identify which exons are essential for gene function and those exons that are "dispensable". Such information will allow personalized therapies to be developed based on splice switching, as we have done for Duchenne muscular dystrophy.

Funding Amount $AUD 808,325.31

Funding Scheme Project Grants

Notes Standard Project Grant