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Target Engagement and MOA |
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Gene manipulation of in vitro and in vivo |
Gene target in contexts of disease model |
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• Target knockdown, co-knockdown of isoforms, & target overexpression |
• Pathway dissecting & phenotypic study |
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• Conventional knockout, knock-in; conditional regional, organ, or cell type specific knockout in mice |
• In vitro functional readout & evaluation |
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or isogenic cell lines |
• MOA & signaling cascade |
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• Region specific or cell type specific inducible/reversible transgenic mice |
• Targeting specific tissues for MOA study |
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• BAC/YAC transgenic mice for humanized animal models |
• In vivo phenotypic characterizations in various disease models |
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• Evaluation of target functional roles |
• shRNA or sgRNA library screening for target id |
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• Characterization of target activities & correlations in cell lines with positive and negative effects |
• IHC, FACS, WB, qPCR for target engagement |
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• Rescue study for target specificity confirmation |
Translational Predictive Biomarkers |
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Genetic approaches |
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• Multiple marker classes including DNA, RNA, protein & |
• Viral-based or nonviral-based vectors in specific cell, tissue, organ or systemic gene delivery |
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microRNA markers |
& manipulation in vitro & in vivo |
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• Genetically defined platform including both in vitro & in vivo |
• Transgenic technology for model generation; genotyping; breeding & characterization |
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disease models |
• Targeted genome editing platform (CRISPR/cas 9) for knockout, knock-in of genome manipulation |
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• Assay development using both mouse & human samples |
at cell types of interest |
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• Bioinformatics supports |
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