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Patient iPSCs: a new discovery tool for Smith-Lemli-Opitz syndrome

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A new study with patient stem cell–based modeling of Smith-Lemli-Opitz syndrome (SLOS) shows that the accumulation of a specific cholesterol precursor dysregulates the Wnt/b-catenin pathway, which in turn leads to precocious neural differentiation.

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Figure 1: iPSCs from individuals with SLOS as a discovery tool for disease research.

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Correspondence to Hongjun Song.

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Wen, Z., Song, H. & Ming, Gl. Patient iPSCs: a new discovery tool for Smith-Lemli-Opitz syndrome. Nat Med 22, 343–344 (2016). https://doi.org/10.1038/nm.4081

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