Projects
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Precision gene editing for the treatment of Fanconi Anaemia. 

2021-2024 (Grant-in-Aid): Precision gene editing for the treatment of Fanconi Anaemia. Dr Lorna McLeman, St Vincent’s Institute of Medical Research.

Fanconi Anaemia is the most common cause of inherited bone marrow failure with a median onset of only eight years of age. Bone marrow failure is fatal in Fanconi Anaemia except for one life saving therapy: a bone marrow transplant.  However, transplants have risks to children, including life threatening toxicities and increased risk of developing secondary cancers. Such risks would be substantially reduced by using gene therapy, which corrects the genetic mutation that causes the bone marrow failure. Recent published clinical trials have proven gene therapy as a potential curative treatment for bone marrow failure in these patients. This project will investigate a new gene editing technology (Prime Editing), with the aim of efficiently correcting Fanconi mutations. If successful, this would provide a new alternative therapeutic option for preventing bone marrow failure in this patient group.

Related Projects

Identifying therapeutics which can be repurposed for the treatment of Myelodysplastic Syndromes and other bone marrow failure disorders.

2018-2021 (Grant-in-Aid): Identifying therapeutics which can be repurposed for the treatment of Myelodysplastic Syndromes and other bone marrow failure disorders. Associate Professor Amee George, The Australian National University. There are ...

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Predicting malignant transformation of Bone Marrow Failure Syndromes using longitudinal targeted sequencing of peripheral blood and cell-free DNA (cfDNA).

2018-2020 (Grant-in-Aid): Predicting malignant transformation of Bone Marrow Failure Syndromes using longitudinal targeted sequencing of peripheral blood and cell-free DNA (cfDNA). Associate Professor Piers Blombery, Peter MacCallum Cancer ...

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Influences of clonal haematopoiesis in allogeneic bone marrow transplantation.

2018-2020 (Grant in aid): Influences of clonal haematopoiesis in allogeneic bone marrow transplantation. Dr Paul Yeh and Professor Mark Dawson, The University of Melbourne. Dr Paul Yeh’s research focusses on using genetic testing to study clonal ...

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Microenvironmental determinants of Aplastic Anaemia progression to MDS / AML.

2019-2021 (Grant-in-Aid): Microenvironmental determinants of Aplastic Anaemia progression to MDS / AML. Associate Professor Rachel Koldej, ACRF Translational Research Laboratory, Melbourne Health. Aplastic Anaemia is a disorder where the body ...

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