Projects
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Steps toward generating new molecular therapies for Bone Marrow Failure Syndromes

2020 – 2024 (Alex Gadomski Scholarship): Steps toward generating new molecular therapies for Bone Marrow Failure Syndromes, Ariel Simpson, Menzies Institute for Medical Research and University of Tasmania.

The only established treatment for Bone Marrow Failure Syndromes is a bone marrow transplant, which has a high risk of complications. Patients with inherited bone marrow failure also commonly experience symptoms outside the bone marrow that cannot be improved by a transplant.

This project takes multiple steps towards developing new therapies for Bone Marrow Failure Syndromes to improve the safety of transplants and address complications that occur outside the bone marrow: 

1) achieve correction of changes (mutations) in the DNA that cause bone marrow failure using new ‘gene editing’ technology,

2) uncover how blood cells develop from bone marrow, and

3) develop a strategy for the generation of a ‘universal donor cell line’ – a product that could be used safely for transplantation instead of donor bone marrow.

This work may give rise to new therapies which are sorely needed to improve the lives of patients and their families.

Related Projects

High resolution definition of the mechanisms of poor graft function following allogeneic haematopoietic stem cell transplantation

2023-2028 (Maddie Riewoldt’s Vision/Haematology Society of Australia and New Zealand Scholarship) High resolution definition of the mechanisms of poor graft function following allogeneic haematopoietic stem cell transplantation, Dr Daniel ...

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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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Discovering new genes and mutations that cause failure of bone marrow neutrophil production. 

2018-2020 (Grant in aid): Discovering new genes and mutations that cause failure of bone marrow neutrophil production. Professor Graham Lieschke, Australian Regenerative Medicine Institute/Monash University and The Royal Melbourne Hospital. ...

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A preclinical trial of next generation gene editing for the prevention of bone marrow failure in Fanconi Anaemia

2023- 2025 (Fellowship): The inaugural Captain Courageous Fellowship. A preclinical trial of next generation gene editing for the prevention of bone marrow failure in Fanconi Anaemia. Dr Astrid Glaser at the Genome Stability Unit of St Vincent’s ...

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