Editing beneficial single nucleotide polymorphisms into the genome: A new approach to genetic disease

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/GNT1098391

Funding Status
Closed

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Funded Activity Summary

?-thalassaemia and sickle cell anaemia are red blood cell diseases caused by deficiencies in adult ?-globin, a component of oxygen-carrying haemoglobin. Interestingly there is a rare group of patients whose symptoms are reduced by naturally occurring mutations that upregulate another globin gene to compensate for defective ?-globin. We are investigating how these beneficial mutations elevate globin levels with a view to engineering them into cells of affected patients to treat these disorders.

Funded Activity Details

Start Date: 01-01-2016

End Date: 01-01-2018

Funding Scheme: Project Grants

Funding Amount: $646,979.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

Biochemistry and Cell Biology not elsewhere classified

ANZSRC Socio-Economic Objective (SEO)

There are no SEO codes available for this funding activity

Other Keywords

gene regulation | genetic disorders | genetic engineering | thalassaemia | transcription factor

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