Target Validation Assay
Transient & Stable genome edition
TARGET DISCOVERY AND VALIDATION USING GENOME EDITING TECHNOLOGIES
Combine a unique translational human model and a powerful gene editing toolbox
APPLICATIONS
Take advantage of the unique capacity of EndoC-βH5® to generate valuable translational data on your target through a full range of genome editing techniques for gene knock-out and knock-down. In parallel, we propose associated precise and predictive evaluation using combination of readouts.
Human Cell Design can provide both constitutive and transient edition as well as a complete characterization of the impact of the target on beta cell loss, function and metabolism. Transient edition can be efficient for a rapid proof of concept of your targeted gene validation, while a constitutive edition focus more on a cell model development (see ADAPT® platform), providing you with a unique human cell model in infinite quantity for accelerating your drug discovery program.
KEY ADVANTAGES
TAILORED MODEL FOR YOUR NEEDS
RAPID & EFFECTIVE TRANSCIENT INVALIDATION
STABLE CONSTITUTIVE APPROACH
DATA CONTINUITY
A versatile toolbox to invalidate your targeted gene
Access to a rapid and robust proof of concept
ASSAY CONCEPT
Target gene invalidation
Human Cell Design’s target validation service ensures a complete and rigorous evaluation of therapeutic targets, facilitating the development of effective diabetes treatments.
| Target gene invalidation | Molecular tools | Target's expression validation | Cell lines |
|---|---|---|---|
| Transient Knock-Down | siRNA | qPCR | EndoC-βH1® |
| miRNA | Western Blot | EndoC-βH3® | |
| Lentiviral shRNA | Live-cells Cytometry | EndoC-βH5® | |
| Immunofluorescence | HLA-A2 & GLTx EndoC-βH5® |
Comprehensive assessment using multiple readouts
Evaluate the impact of your target on a wide range of readouts developed to meet your needs: from phenotype studies to stress response or complete profiling of cell signaling.
| Applications | Readouts | Tools |
|---|---|---|
| Phenotype | Beta Cell Loss | Viability |
| Beta Cell Function | Insulin secretion | |
| Beta Cell Differentiation | Beta cell markers’ FACS expression | |
| Omics profiling | Transcriptomic | RNA sequencing, metabolic pathway regulation, core network analysis, gene signature |
| Proteomic | LC-MS sequencing on secreted and intracellular content | |
| Metabolomic | ||
| Stress response | Inflammatory Stress Response | Inflammatory markers’ expression, SASP, cytokines secretion |
| Oxydatif Stress Response | Stress markers’ expression, ROS accumulation, ER stress | |
| Mitochondrial Stress Response | Mitochondrial stress markers’ expression, mitochondrial respiration, membrane potential, mitochondrial mass |
HAVE A PROJECT IN MIND?
Human Cell Design team of experts can offer you tailored assays and EndoC-βH5® derived cell models.
