Modelling consequences of cell abundance, heterogeneity and origin for autologous cell therapy in genetic Bone Marrow Failure Syndromes.

2020-2023 (Fellowship): Modelling consequences of cell abundance, heterogeneity and origin for autologous cell therapy in genetic Bone Marrow Failure Syndromes. Dr Parvathy Venugopal, Centre for Cancer Biology / University of South Australia.

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