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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Curation of the DIAAMOND-based Aplastic Anaemia Biobank

2021-2023 (Grant-in-Aid): Curation of the DIAAMOND-based Aplastic Anaemia Biobank. Associate Professor Stephen Ting, Monash University. This project (the DIAAMOND biobank) is a sub-study of the DIAAMOND clinical trial and will collect, process ...

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Genome editing of haematopoietic stem and progenitor cells to uncover novel therapeutics for Aplastic Anaemia and other Bone Marrow Failure Syndromes. 

2017-2021 Flavorite Fellowship, Genome editing of haematopoietic stem and progenitor cells to uncover novel therapeutics for Aplastic Anaemia and other Bone Marrow Failure Syndromes, Dr Yih-Chih Chan, The University of Melbourne and Peter ...

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Establishing an in vivo humanised mouse model for telomere related bone marrow failure syndromes.

2019-2021 (Grant-in-Aid): Establishing an in vivo humanised mouse model for telomere related Bone Marrow Failure Syndromes. Professor Tracy Bryan, Children’s Medical Research Institute. Human genetic information is packaged into discrete bundles ...

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