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.
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 ...
Read more2018-2020 (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. ...
Read more2019-2021 (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 ...
Read more2020-2022 (Grant-in-Aid): Using whole genome sequence analysis to find answers for unsolved cases of inherited Bone marrow Failure Syndrome. Associate Professor Piers Blombery, The University of Melbourne. Inherited Bone Marrow Failure Syndromes ...
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