Splice correction as a treatment for rare diseases [ 2015 - 2018 ]

Research Grant

Researchers: Prof Stephen Wilton (Principal investigator) ,  Prof Jack Goldblatt ,  Prof Matthew Bellgard ,  Prof Sue Fletcher

Brief description 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.

Funding Amount $AUD 824,316.94

Funding Scheme Project Grants

Notes Standard Project Grant