Projects
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Using induced pluripotent stem cells to find causes and cures for bone marrow failure in children and young adults

2018-2023 (Grant-in-Aid): Using induced pluripotent stem cells to find causes and cures for bone marrow failure in children and young adults. Professor Andrew Elefanty, Murdoch Children’s Research Institute.

Bone Marrow Failure Syndromes may be caused by inherited mutations. In cases where mutated genes are not identified, it is believed that an abnormal immune system is the cause. Induced pluripotent stem cells (iPSCs) are a powerful research tool in understanding the mechanisms behind a disease, as they can be grown into many different cell types, including blood cells.

The goal of this project is to create iPSCs from patients with bone marrow failure and their relatives to study the cause of bone marrow failure. To date, iPSCs have been made from 5 patients with unknown cause of bone marrow failure and their family members and one patient with a known inherited mutation.

By examining the differences in blood cells grown from these iPSCs, the team can better understand abnormalities in blood cells that lead to bone marrow failure and explore new treatment options.

Related Projects

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 ...

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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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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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Outcomes of Haematopoietic Stem Cell Transplantation for Paediatric Patients with Severe Aplastic Anaemia (SAA) and Bone Marrow Failure (BMF) Syndromes.

2021-2022 (Grant-in-Aid): Outcomes of Haematopoietic Stem Cell Transplantation for Paediatric Patients with Severe Aplastic Anaemia (SAA) and Bone Marrow Failure (BMF) Syndromes. Dr Steven Keogh, Australasian Bone Marrow Transplant Recipient ...

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