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Novel stem cell approach restores motor function in Parkinson’s Disease

In a remarkable approach to combatting neurodegeneration using stem cells, Florey researchers have achieved preclinical evidence that stem cells grafts can restore motor function in animals with Parkinson's Disease.

Led by Professor Clare Parish, Head of the Florey’s Stem cell and neurodevelopment laboratory, the team investigated whether combined use of stem cells and gene therapy could repair the brain in Parkinson’s disease. Their first-of-its-kind research unearthed incredible results.

“By transplanting human stem cells into the brain along with viral delivery of a growth promoting protein called GDNF, we were able to restore motor function in animal models of Parkinson’s disease,” explained Professor Parish. 

Professor Clare Parish

Image: Professor Clare Parish, Head of the Florey’s Stem cell and neurodevelopment laboratory

In Parkinson’s disease, the progressive loss of dopamine neurons in the brain gives rise to the weakening motor function. The use of stem cell-derived dopamine transplants in Parkinson’s disease is not a new concept, but one that has presented researchers with significant challenges. Transplanted dopamine cells have been found to survive poorly and fail to adequately integrate or ‘knit’ into the brain.

The novel technique developed by Parish and her team could change that. Results of their study demonstrate increased survival, maturity and integration of transplanted dopamine cells, ultimately leading to an improvement in motor function. 

“We now hope to advance this stem cell approach to clinical trials in the coming years,” added Parish.

Image: Delivery of GDNF increased survival of dopamine cells (brown staining) within transplanted grafts.

The research has been published in Cell Stem Cell (DOI: 10.1016/j.stem.2020.01.010). Click to access the full research paper here.

Media enquiries to Claire Smoorenburg, Media and Communications Manager, The Florey Institute of Neuroscience & Mental Health M: +61 438 090 208 E: claire.smoorenburg@florey.edu.au

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