Splice correction as a treatment for rare diseases

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/1086311

Funding Status
Closed

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Funded Activity Summary

We propose that a strategy of bringing effective and personalised treatments to amenable patients with rare genetic diseases, though ambitious, is readily achievable and opportune. Importantly, a consensus approach will facilitate expediting potentially curative treatments for many patients with rare diseases that would be unlikely to be commercially viable if considered individually.

Funded Activity Details

Start Date: 01-01-2015

End Date: 01-01-2018

Funding Scheme: Project Grants

Funding Amount: $824,316.00

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

RNA processing | antisense technology | genetic disorders | splicing | targeted therapy

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