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

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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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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Towards targeted treatments for Fanconi Anaemia 

2017 – 2019 Fellowship / 2017 Grant-in-Aid, Towards targeted treatments for Fanconi Anaemia, Associate Professor Wayne Crismani, St Vincents Institute for Medical Research Fanconi Anaemia is an inherited disorder which can lead to bone ...

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Identification of microRNA biomarkers predictive of clinical outcomes in Aplastic Anaemia and Myelodysplastic Syndrome

2016 – 2019 (Grant-in-Aid) Identification of microRNA biomarkers predictive of clinical outcomes in Aplastic Anaemia and Myelodysplastic Syndrome, Dr Lynette Chee, Melbourne Health. DNA is the genetic material which provides the information that ...

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