Editing beneficial single nucleotide polymorphisms into the genome: A new approach to genetic disease [ 2016 - 2018 ]

Research Grant

Researchers: Prof Merlin Crossley (Principal investigator)

Brief description ?-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.

Funding Amount $AUD 665,264.73

Funding Scheme Project Grants

Notes Standard Project Grant