Some babies are born without properly functioning immune systems, leaving them vulnerable to life-threatening infections, autoimmunity and cancer – yet treatment options remain limited. By developing a highly sensitive stem cell differentiation platform, we can model these rare conditions in the laboratory and rapidly test gene therapies before they reach clinical trials. This brings us closer to curative treatments, offering real hope for patients with inherited immune disorder
Impact
This research will provide an efficient platform for necessary new gene editing techniques in stem cells to treat a wide range of blood disorders. Furthermore, given the vast number of primary immunodeficiencies, many of which are quite rare, these studies will contribute to understanding the pathogenic mechanism of the modelled disorders, potentially revealing further therapeutic opportunities.
reNEW research
We have established a rapid and robust stem cell differentiation platform, in which we can mature blood stem cells into eight immune cell lineages (and counting!), all of which require different cocktails of cytokines, growth factors and conditions. We are now primed to start testing clinically relevant gene editing techniques, initially with stem cells modelling four different primary immunodeficiency disorders.
Image description
Blue fluorescent haematopoietic stem cells are seen emerging from embryoid bodies. In this picture, the cells have naturally formed a ‘possum-like’ tail from which the stem cells are maturing.
Credits
Dr Kat Goodall, Senior Research Officer, Raymond Yow, Research Assistant, Elefanty Lab, reNEW Melbourne.
