MSF preclinical platform: The components, models, assays and preclinical tools are listed. Patient-derived IPSCs include both variants SMAD4 p.R496C and SMAD4 p.I500V and their non-affected siblings of same sex, generated from PBMC, using reprogramming with Sendai virus, by iXCells Biotechnologies USA, Inc. for MSF. The Generation of vascular endothelial cells and microglial cells from the patient-derived IPSCs and the cellular assays standardization and validation, and screening performed by NeuCyte Inc. for MSF. The mouse models are generated by Ozgene Pty Ltd for MSF; and the phenotyping assays and screening performed by PsychoGenics for MSF.

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