Disease Modeling
Delivering phenotypically relevant disease models
Overview
DefiniGEN's induced pluripotent stem cell (iPSC) OptiDIFF platform enables the generation of high-quality wild-type and disease cell models. We can produce mature cells which recapitulate a range of disease phenotypes and are being adopted for preclinical evaluation studies.
We have developed various disease models for a wide range of rare metabolic indications such as:
DefiniGEN offer the ability to develop custom disease models for screening or characterizing lead compounds for indications that have previously been difficult to study.
Disease Modeling Services
Whether you need CRISPR gene-edited disease models, or patient-derived models, our scientists can deliver.
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CRISPR ModelingOur custom cell model service is an integrated, end-to-end package that includes iPSC line or donor selection, the complete CRISPR editing workflow, and differentiation into the target cell typeLearn More -
Patient Derived ModelsDefiniGEN's patient-derived iPSC custom cell model development service covers the entire process from patient sample sourcing and iPSC generation to differentiation, validation, and cryopreservation.Learn More -
IPSC DifferentiationWe've transformed the "difficult" into the "routine" and have successfully differentiated multiple iPSC lines into a range of target cell types that fully recapitulate the mature cell phenotype.Learn More
CRISPR Modeling Workflow
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iPSC reprogrammingWe can start your service with any of our comprehensively characterized in-house iPSC lines, you can supply your own iPSC lines, or you can provide material derived from patient donors. -
Gene EditingWe work in collaboration with you to design sgRNAs and use a footprint-free CRISPR/Cas9 system to edit the iPSCs. We optimize transfection conditions before full-scale editing, and then screen the edited populations, seed single cells, and sanger sequence the clones. You select which clones to move forward with. -
iPSC DifferentiationWe use our world-leading OptiDIFF platform to differentiate your edited iPSCs.
CRISPR Modeling
- Rapid turnaround time. Our optimized protocols and standardized workflows ensure fast, efficient project completion.
- High success rates. Our deep expertise handling challenging iPSCs easily translates into successful CRISPR editing of iPSCs.
- Collaborative project management. We conduct every project with complete transparency and in close collaboration with client teams to ensure we deliver what you need.
Patient-derived Model
- Access to rare and metabolic disease samples. Our established relationships with well-regarded cell banks can streamline patient sample sourcing for your project.
- High success rates. Our optimized iPSC generation and differentiation workflows and years of experience directly translate into higher probabilities for success.
- Collaborative project management. We conduct every project with complete transparency and in close collaboration with client teams to ensure we deliver what you need.
Case Studies
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CRISPR Model - NAFLDDefiniGEN disease modelled NAFLD iPSC-derived hepatocytes represent an optimized model for drug discovery applications and a principal tool for elucidating the underlying mechanisms of the disease.Learn More -
Patient derived model - A1ATDDefiniGEN provide disease modelled alpha-1 antitrypsin deficiency hepatocytes which are highly functional patient-derived human hepatocytes.Learn more -
Custom model developmentFrom a basic frameshift knock-out mutation to complex knock-in genetic changes, our dedicated scientific team will work collaboratively with you to design the right tools to accelerate your research programs.Discover
Frequently Asked Questions
Disease Modeling
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It depends on the complexity of the project but generally we deliver the edited cell line in 8 weeks. If differentiation into a specific cell type is required, additional time will be necessary and will depend on the requested scale and cell type of interest.
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The entire gene editing process takes between 8-10 weeks depending on the complexity of the project. We will advise you of the lead time when we generate your quote.
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We work as a research partner offering collaborative services. We offer bi-weekly calls with our clients and interim emails with project reports. Our team is always available to provide technical support which can include experimental design and training on best practices for handling cells.
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To generate a quote, we need your name, the name of your organization, your email address and details on the gene, mutation of interest, and cell type to edit. We also recommend contacting our team to go through project requirements in more detail. Contact us today >
