Targeted alternative splicing: a common therapeutic platform to treat inherited diseases

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/GNT1144791

Funding Status
Closed

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Funded Activity Summary

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.

Funded Activity Details

Start Date: 01-01-2018

End Date: 01-01-2020

Funding Scheme: Project Grants

Funding Amount: $797,165.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

Molecular Medicine

ANZSRC Socio-Economic Objective (SEO)

There are no SEO codes available for this funding activity

Other Keywords

antisense oligonucleotides | disease mechanisms | genetic disorders | splicing | treatment strategies

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