Harnessing RNA interference in gene therapy vectors for ?-thalassaemia

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/1147867

Funding Status
Closed

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Funded Activity Summary

There is an urgent need to develop safe and effective treatments for ?-thalassaemia. We anticipate that ?-globin-specific RNAi sequences will synergise with ?-globin transgene expression to achieve balanced ?-/?-globin ratio in a clinical setting. Given that one of the major issues with current gene therapy vectors is achieving high levels of expression, we believe this will be a more effective gene therapy strategy than ?-globin transgene expression alone.

Funded Activity Details

Start Date: 01-01-2018

End Date: 01-01-2020

Funding Scheme: Project Grants

Funding Amount: $719,188.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

Haematology

ANZSRC Socio-Economic Objective (SEO)

There are no SEO codes available for this funding activity

Other Keywords

RNA interference | gene therapy | lentiviral vector | mouse models | thalassaemia

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