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Jan 10, 2013
 |  BC Innovations  |  Tools & Techniques

Scaling up iPS cells

A Memorial Sloan-Kettering Cancer Center team has conducted one of the first large-scale screens of a small molecule library using patient-derived induced pluripotent stem cells.1 The approach was applied to neuronal cells derived from patients with familial dysautonomia and provides early evidence of its potential for identifying new leads.

Separately, a public-private partnership led by Roche and the University of Oxford is aiming to establish a repository comprising 1,500 induced pluripotent stem (iPS) cells-triplicate samples from 500 patients with diabetes or a wide range of neurological diseases-in an effort to provide a tool for finding new targets and therapeutic leads.

Patient-derived iPS cells are generated by reprogramming skin fibroblasts. These cells can subsequently serve as the starting material for obtaining disease-relevant cell types through redifferentiation. The goal is to obtain cells from patients that will display phenotypes characteristic of the disease and thus can be used to explore disease mechanisms or screen for small molecules that reverse the disease phenotype.

"For the first time, we have human cells from patients and can differentiate them into different cell types in a dish. If we can identify disease-relevant phenotypes, this has huge potential," said Martin Graf, head of the stem cell platform at Roche.

However, there are many practical challenges in using iPS cells to characterize disease, including identifying strong disease-associated phenotypes and patient-to-patient genetic variations.

For high throughput screening, there is the added need to have a large number of relatively pure cells and an assay that can be scaled up. Because it is difficult to conduct small molecule screens with iPS cells, initial screens to look for compounds that modulate a target or phenotype typically have been conducted with standard, non-patient-derived human cell lines.

"The...

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