CIB products suitable for drug development

    One of the most important features of the iPSC technology is that it enables researchers to obtain iPSCs from patient samples and re-differentiate the patient-derived iPSCs into somatic cells that manifest disease phenotype in vitro. In addition, iPSC technology combined with gene editing technology allows researchers to create isogenic iPSC lines that contain genetic mutations discovered in patients and thus mimic the naturally occurring disease phenotypes. Numerous reports have demonstrated the utility of iPSC in disease modeling and drug development. 

    1) iPSC lines for Parkinson’s Disease (PD):  in past a couple of years, we have combined iPSC and gene editing technologies to create a panel of isogenic iPSC lines that contain defective genes found in some Parkinson patients. We also derived neural stem cells (NSCs) and neuronal progenitor cells (NPCs) from these iPSCs and demonstrated that loss of function of some genes cause abnormal phenotype in neurons (Figure 1). Our PD panel includes NSCs and NPCs from two iPSC lines harboring defective Park7 and Pink1 genes, loss of function of these two genes are associated with mitochondrial dysfunction and PD. These products offer a unique model for studying mechanism of PD and a platform for screening novel therapeutics for treatment of PD.

    2) iPSC lines for Wilson Disease (WD):  this panel consists of two iPSC lines derived from patients with Wilson disease, a liver disease caused by a single nucleotide mutation in the gene ATP7B. Hepatocytes derived from the patient iPSC line showed abnormality in ATP7B protein function, illustrated by loss of interaction with protein P230 (Figure 2). The ATP7B-P230 protein-protein interaction is essential for liver function.

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