Developing exon replacement gene therapy to cure Rett syndrome: an innovative model for neurodevelopmental disorders

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/2001536

Funding Status
Status not available

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Funded Activity Summary

There is no cure for neurodevelopmental disorders such as Rett syndrome which is caused by mutations in the MECP2 gene. Gene therapy is ineffective due to the 'Goldilocks' effect where too little, as well as too much expression of MECP2 causes disease. Here a gene editing therapy will be tested in patient cells and mouse model that will maintain the normal cellular expression of MECP2 by cutting out the mutated regions of the gene (exons) and replacing them with repaired copies.

Funded Activity Details

Start Date: 01-01-2020

End Date: End date not available

Funding Scheme: Ideas Grants

Funding Amount: $475,105.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

ANZSRC Socio-Economic Objective (SEO)

There are no SEO codes available for this funding activity

Other Keywords

central nervous system (cns) | developmental disorders | gene therapy | neurology

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