Menu

CRISPR Modelling

CRISPR Service

The CRISPR-Cas9 system is able to achieve highly specific and precise targeting for the genetic manipulation of cells and cell lines, transforming the field of genome engineering and presenting a wealth of new possibilities for R&D. DefiniGEN have optimized conditions for CRISPR Cas-9 genome editing on multiple patient-derived iPSC’s, and we have built up considerable experience in using the system for genomic manipulation in a range of other cell lines.

We can offer our clients an integrated end-to-end package including sgDNA design, transfection, optimisation, and clone picking/sequencing to generate a library of gene edits for client selection. From 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.

 


Workflow

We have developed a complete workflow for the knockout of an endogenous gene. Our workflow consists of footprint-free CRISPR/Cas9-mediated editing, single-cell cloning of the edited population, and characterization of expanded clonal cell lines to identify positive clones. The edited iPSC cells maintain pluripotency and have normal, stable karyotypes, which are important for testing downstream applications such as directed differentiation.

Diagram of iPSC CRISPR Cas9 gene editing service

 

  • Physiologically relevant disease models for complex diseases
  • iPSC gene-editing using healthy/diseased donors
  • Bespoke deliverables: homozygous/heterozygous mutations, point mutations, with/without silent mutations
  • Isogenic control lines available

 


CRISPR/Cas9 Cell Line Service Timeline

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. Our service can be specifically tailored to your requirements.

To help you generate your particular disease models, we offer gene editing services to knock out a target gene or knock in a disease specific mutation.

ServiceTimeline
Cell line characterization2 weeks
Cell line engineering4 weeks
Cell line validation2-3 weeks
Targeting, reagent design & construction2 weeks
Total11 weeks

Let us know your project specific requirements

Let our team of experts create custom edited clonal cell lines for your research. Contact us today so we can get started on your project.

  • Contents

Potential disease model examples

Disease of interest 
Crigler NajjarAlagille syndrome
Gaucher's diseasePrimary hyperoxaluria type 1
Wilson's diseaseUrea cycle disorders (except ASL)
Genetic cholestasis (PFIC)Acute intermittent porphyria
Hereditary hemochromatosisArginiosuccinic aciduria (ASL)
Phenylketonuria (PKU)Atypical haemolytic uremic syndrome-1
Ornithine transcarbamylase deficiency (OTC)Cystic Fibrosis
Tyrosinemia type 1Erythoropoietic protoporphyria
Glycogen storage disease (GSD) type 0-XIIFamilial amyloid polyneuropathy
Organic acidurias