Could Pharmacological Restoration of Mecp2 levels be of Therapeutic Value in Rett syndrome?

Funding Activity

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Funded Activity Summary

Rett syndrome is one of the commonest single gene cause of developmental disability in girls; 1:8500 Australian females are affected by the age of 12. There is currently no cure. Rett syndrome most commonly results from a fault in one copy of the Mecp2 gene on the X chromosome. The proposed research aims to identify medicines, already known to be safe in children and adults, which specifically improve the functioning of the Mecp2 gene. This should rapidly lead to targeted clinical treatments for this condition.

Funded Activity Details

Start Date: 01-01-2012

End Date: 01-01-2014

Funding Scheme: NHMRC Postgraduate Scholarships

Funding Amount: $.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

Gene Expression (incl. Microarray and other genome-wide approaches)

ANZSRC Socio-Economic Objective (SEO)

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Other Keywords

Rett syndrome | child health | drug discovery | drug therapy | gene expression | intellectual disability | stem cells | targeted therapy