DefiniGEN has world-leading expertise in the area of iPSC production and metabolic disease modelling. The company has a unique platform technology for generating phenotypically validated human cell disease models for a range of rare metabolic and liver diseases, to optimize preclinical drug discovery. DefiniGEN’s proprietary OptiDIFF platform generates terminally differentiated human cells of endodermal lineage from iPSCs. We provide iPSC-derived hepatocytes, pancreatic cells, and intestinal organoids from healthy and diseased donors which closely resemble human primary cells.
Off-the shelf products include:
The application of these technologies in drug discovery provides pharmaceutical companies with more predictive in vitro human cell products enabling safer and more effective treatments. This technology platform can be combined with our cutting edge CRISPR gene-editing service to produce a wide range of bespoke validated disease model cell products enabling pharmaceutical companies to effectively reprofile and reposition their drug libraries.
Gene-editing of iPSCs allows researchers to create isogenic cell models containing key disease driving mutations to achieve mechanistic understanding, without background genetic variability allowing determination of causative relationships between genotype and phenotype. We offer a collaborative partnership with our clients to deliver models in a timely and efficient manner.
Service types available:
Indel for gene disruption
Large cassettes integration
Service includes: targeting reagent design & construction, cell line engineering, cell line validation + differentiation to specific cell type.
Highly characterized/validated edited human cell lines (clonal or pool)
Isogenic (parental) controls to remove any phenotypic effects driven by factors intrinsic to the particular mutation of interest
DefiniGEN is highly experienced in using CRISPR/Cas9 gene editing technology to manipulate the genome of iPSC to generate disease models.
Our expertise can help design the guide RNA’s (gRNA) in silico for maximum efficiency, then optimize the electroporation of the cell line in question to best establish ideal conditions for the gene edit. Our team then methodically sequence a number of clones to confirm the precise genetic manipulation has been successful.
Service types available:
• Indel for gene disruption
• Large deletions
• Multiple deletions
• Point mutations/SNPs
• Large cassettes integration
• Tag Reporter
Service includes: targeting reagent design & construction, cell line engineering, cell line validation + differentiation to specific cell type if required.
• Highly characterized/validated edited human cell lines (clonal or pool)
• Isogenic (parental) controls to remove any phenotypic effects driven by factors intrinsic to the particular mutation of interest
DefiniGEN’s core technology platform has focussed on the development of differentiation protocols which increases the success of generating viable, functional and mature hepatocytes from either patient material (generally fibroblasts and PBMCs) or existing iPSC lines.
With DefiniGEN’s differentiation service, we deliver high-quality bespoke cell models derived from patient donors or existing iPSC lines. As an alternative to the provision of client cells we can source donor material from our extensive network.
iPSC reprogramming or sourcing: donor material sourced through DefiniGEN's hospital network or provided by our clients are reprogrammed to iPSC using multiple methodologies
Expansion in culture system: expansion of iPSC line to generate start material and master cell bank
Small scale differentiation: optimization of best differentiation parameters at small-scale in preparation for large scale
Large scale differentiation: differentiate from iPSC through definitive endoderm to differentiated cell type followed by cryopreservation
Product validation: thaw and recovery with % viability confirmation by monolayer assessment. Cell morphology, marker verification sequence and/or gene expression analysis confirmation
Delivery: cryopreserved cells shipped to client with high viability post-thaw
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