In some people with inherited bone marrow failure, a small number of cells naturally undergo ‘spontaneous reversion’ – the correction of the genetic cause of disease. This can result in milder symptoms and, in some cases, ‘cure’ bone marrow failure. The goal of this project was to investigate this phenomenon in patients and find strategies to encourage reverted bone marrow cells to grow and replace defective ones. A second stream of this study describes a mouse model for bone marrow failure that can be used to research the potential for transplantation of corrected cells as a therapy for Bone Marrow Failure Syndromes
2022-2025 (Fellowship): Dissecting immune dysregulation in acquired Bone Marrow Failure Syndromes to identify new therapeutic leads. Associate Professor Rachel Koldej, ACRF Translational Research Laboratory, Melbourne Health. Bone Marrow Failure ...
Read more2017-2021 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 ...
Read more2018-2021 (Doctoral Degree Fellowship): Clinical, genomic and molecular determinants of outcome in patients with Bone Marrow Failure Syndromes, Dr Lucy Fox, The University of Melbourne. This project is the first scholarship in Australia to ...
Read more2023-2025 (Fiona Riewoldt Nursing/Allied Health Fellowship): Utility of a neurobehavioural assessment for treatment planning, educational, and family support for children undergoing bone marrow transplant for non-malignant ...
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