Journal article

Isolation of LMX1a Ventral Midbrain Progenitors Improves the Safety and Predictability of Human Pluripotent Stem Cell-Derived Neural Transplants in Parkinsonian Disease

Isabelle R de Luzy, Jonathan C Niclis, Carlos W Gantner, Jessica A Kauhausen, Cameron PJ Hunt, Charlotte Ermine, Colin W Pouton, Lachlan H Thompson, Clare L Parish

Journal of Neuroscience | SOC NEUROSCIENCE | Published : 2019


Human pluripotent stem cells (hPSCs) are a promising resource for the replacement of degenerated ventral midbrain dopaminergic (vmDA) neurons in Parkinson's disease. Despite recent advances in protocols for the in vitro generation of vmDA neurons, the asynchronous and heterogeneous nature of the differentiations results in transplants of surprisingly low vmDA neuron purity. As the field advances toward the clinic, it will be optimal, if not essential, to remove poorly specified and potentially proliferative cells from donor preparations to ensure safety and predictable efficacy. Here, we use two novel hPSC knock-in reporter lines expressing GFP under the LMX1A and PITX3 promoters, to selecti..

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Awarded by National Health and Medical Research Council Australia

Funding Acknowledgements

C.L.P. was supported by Viertel Charitable Foundation (Australia) Senior Medical Research Fellowship. I.R.d.L. was supported by University of Melbourne International Scholarship(Australia). C.W.G. was supported by Australian Postgraduate Award. This work was supported by National Health and Medical Research Council Australia Project Grant APP1102704, and Stem Cells Australia. The Florey Institute of Neuroscience and Mental Health acknowledges the strong support from the Victorian Government and in particular the funding from the Operational Infrastructure Support Grant. We thank Mong Tien for expert technical assistance; and Melbourne Brain Centre flow cytometry facility for support.