iPSC Gene-Editing Service

Horizon’s leading cell line engineering service now available in human pluripotent stem cells

Induced Pluripotent Stem Cells (iPSC) provide researchers with translational in vitro models that allows study of ‘disease in a dish’ for difficult to represent diseases and to discover novel drug targets with increased predictability of their safety and efficacy.

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.

iPS cell uses:

  • Knockout a gene of interest
  • Knockin a disease-associated point mutation
  • Reversion to wildtype in disease-derived iPS line
  • Tag a gene of interest with a choice of reporters

iPSC projects:

  • Cells: iPSCs or ESCs provided by the customer
  • Estimated timeline: 17-27 weeks
  • Deliverables: Edited iPSCs or ESCs and a summary report

Our service can be tailored to your requirements. Please complete our Custom iPS Gene-Editing Service form if you would like to discuss your requirements further.

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To provide an integrated gene-edting and iPSC offering, Horizon has partnered with two key providers of iPSC services. The benefits of our iPS cell-derived disease models include:

  • Isogenic trios provided - precisely edited at endogenous loci and parental un-edited controls are available to remove issue of background variability
  • Heterozygous and Homozygous genotypes - Allows investigation of 'dose-dependent' effect of genotype
  • Functional cell models - key phenotypes are confirmed in the gene-edited differentiated cell types

Axol BioscienceAxol Bioscience

Horizon has partnered with Axol Bioscience, bringing together Horizon’s precision genome editing capability and Axol’s expertise in iPSC reprogramming and differentiation, in order to provide isogenic cell lines for neurodegenerative disease modelling.

By combining our areas of expertise, we have generated an off-the shelf collection of isogenic, functional gene-edited iPS cell-derived neural stem cells, providing a more translational and predictive model for neurodegenerative diseases including Parkinson's disease, Alzheimer's disease and Frontotemporal Dementia.

Mutations available off the shelf are MAPT R406W, P301L and V337M plus LRRK2 G2019S.

Download our recent poster 'Modelling neurological disease: in vitro gene editing and iPSC differentiation combine to create powerful new tools' for more information on these models.



Horizon has partnered with DefiniGEN, a leading provider for liver, pancreas, intestinal and lung cells derived from iPSCs utilising their proprietary OptiDiff platform. Together, we have created a selection of iPSC-derived models that represent metabolic diseases that had a genetic association. Gene-edited iPSC-derived pancreatic cells are available carrying either a HNF1A P291insC or KCNJ11 R201H mutation as a disease model for MODY (maturity onset diabetes of the young) and Neonatal Diabetes.

In addition, iPSC-derived hepatocytes have been genetically engineered carrying the LDLR E101K mutation, which has been associated with autosomal dominant inheritance of familial hypercholesterolemia. To find out more about the use of this model, download our recent poster 'Modelling familial hypercholesterolemia using human iPSCs'.


How to order:

If you are interested in placing an order for our off-the-shelf gene-edited iPSC-derived isogenic cell models, please contact our Customer Services team via the Live Chat option on the page or via our Contact us page:

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