Platform Screens Cell Lines Derived from Human Embryonic Stem Cells for Muscular Dystrophy Research

Investigators are using a new stem cell screening platform to automate high content screening of small molecules for muscular dystrophy therapeutic research. The research shows the utility of the system in performing automated screens on cell lines derived from human embryonic stem cells.

In the new study, researchers from the Institute for Stem cell Therapy and Exploration of Monogenic Diseases (I-STEM; Evry, France) used a BioCel 1800 platform, developed by Agilent Automation Solutions (Santa Clara, CA, USA).

The most common adult neuromuscular disease, the type 1 muscular dystrophy (DM1), is an autosomal monogenic disease characterized by the aggregation of mutated mRNA in structures called foci within cells' nuclei. The researchers screened the Prestwick library (Illkirch, France) of U.S. Food And Drug Administration- (FDA)-approved compounds in a cell-based assay using an Agilent Automation Solutions BioCel 1800, looking for molecules targeting foci structure using a high content screening (HCS) strategy. Mesenchymal stem cells derived from the Vub03 mutated human embryonic stem cell line were selected as a model for this research as they display foci when labeled by fluorescent in situ hybridization (FISH) in 96-well plates. The BioCel platform allows researchers to handle cell lines with confidence and precision and is now considered a critical part of I-STEM's stem cell screening strategy.

To avoid any risk of contamination to the cell suspensions, the BioCel 1800 Platform was configured with ULPA filtered environmental venting and temperature control. The reported results demonstrate how the Biocel 1800 provide a high level of liquid-handling reproducibility. Moreover, the researchers developed methodology for screening compounds using cell-based assays and two Agilent vertical pipetting stations for both cell culture management and compound library management.

The Institute for Stem Cell Therapy and Exploration of Monogenic diseases (I-STEM) is a center for research and development, dedicated to the development of treatments based on the potential offered by stem cells and applicable to rare diseases of genetic origin. The activity of I-STEM extends from basic biologic research and pathologic processes up to the transfer of new therapies to clinical research.

From stand-alone units to fully integrated systems, Agilent Automation Solutions combines sample preparation and walk-away automation approaches with customized customer service to provide laboratory systems.

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