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Early Detection of Phenotypes in GD1 Neurons

Gaucher disease type 1 (GD1) associates with an increased risk of developing Parkinson's disease. While induced pluripotent stem cells (iPSCs) derived from GD1 patients can be useful for pathological analyses, the commonly used method for neuron differentiation remains a complex undertaking that requires 35 to 60 days to form mature neurons. In a new STEM CELLS Translational Medicine article, researchers led by Tomohiko Akiyama (Keio University School of Medicine, Tokyo) and Hidehisa Iwata (Takeda Pharmaceutical Company Limited, Fujisawa, Japan) report that the synthetic mRNA‐based expression of transcription factors enhances iPSC differentiation and enables the early detection (10 days) of a Parkinson's disease risk factor. Overall, these results suggest that synthetic mRNA‐induced differentiation can accelerate the analyses of the pathological mechanisms associated with Parkinson's disease in GD1 patients.